Wyszukaj badanie kliniczne
185 Wyniki
Badanie kliniczne z otwartą rekrutacją
= 
; Badanie kliniczne w toku
= 
; Terminated trial = 
; Ufundowane przez członka IRDiRC = 
; MISSING CONTENT = 
Krajowe badania kliniczne
AUSTRIA
TIROL
INNSBRUCK
LCI-OUT: Lung Clearance Index as an OUTcome parameter to detect the efficacy of Aztreonam Lysine Inhalation in cystic fibrosis patients with near normal spirometry - an observational proof-of concept study
Medizinische Universität Innsbruck
Universitätsklinik für Pädiatrie III
AUSTRIA
TIROL
INNSBRUCK
A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis - AT
Medizinische Universität Innsbruck
Universitätsklinik für Pädiatrie III
AUSTRIA
WIEN
ADDRESS: NOT PROVIDED - AT
A Phase 3, Open-label, Rollover Study to Evaluate the Safety and Efficacy of Long Term Treatment With VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation - AT
Institution: Information not provided - AT
AUSTRIA
WIEN
ADDRESS: NOT PROVIDED - AT
CHELATE STUDY: Trientine Tetrahydrochloride (TETA 4HCL) for the Treatment of Wilson's Disease (Phase 3) - AT
Institution: Information not provided - AT
AUSTRIA
WIEN
ADDRESS: NOT PROVIDED - AT
ALPINE2: Randomized, Double-Blind, Phase 3B Trial to Evaluate the Safety and Efficacy of 2 Treatment Regimens of Aztreonam 75 mg Powder and Solvent for Nebulizer Solution / Aztreonam for Inhalation Solution (AZLI) in Pediatric Subjects With Cystic Fibrosis (CF) and New Onset Respiratory Tract Pseudomonas Aeruginosa (PA) Infection/Colonization - AT
Institution: Information not provided - AT
AUSTRIA
WIEN
ADDRESS: NOT PROVIDED - AT
A Phase 3, Randomized, Rater-Blinded, Multi-Center Study To Evaluate the Efficacy and Safety of ALXN1840 Administered For 48 Weeks Versus Standard of Care in Patients With Wilson Disease Aged 12 Years and Older With an Extension Period of Up To 60 Months - AT
Institution: Information not provided - AT
AUSTRIA
WIEN
ADDRESS: NOT PROVIDED - AT
A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation - AT
Institution: Information not provided - AT
AUSTRIA
WIEN
ADDRESS: NOT PROVIDED - AT
A Randomized, Double Blind, Dose-finding Study of Inhaled Oligosaccharide (OligoG) vs Placebo in Patients With Cystic Fibrosis (Phase II-III) - AT
Institution: Information not provided - AT
AUSTRIA
WIEN
ADDRESS: NOT PROVIDED - AT
A Phase 2, Single-arm, Pathologist-blinded Study Using Liver Biopsy Specimens to Assess Copper Concentration and Histopathologic Changes in Patients With Wilson Disease Who Are Treated With ALXN1840 for 48 Weeks Followed by an Extension Treatment Period With ALXN1840 for up to an Additional 48 Weeks - AT
Institution: Information not provided - AT
AUSTRIA
WIEN
ADDRESS: NOT PROVIDED - AT
SEQUOIA: A Placebo-Controlled, Multi-dose, Phase 2 Study to Determine the Safety, Tolerability and Pharmacodynamic Effect of ARO-AAT in Patients With Alpha-1 Antitrypsin Deficiency (AATD) - AT
Institution: Information not provided - AT
AUSTRIA
WIEN
ADDRESS: NOT PROVIDED - AT
A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-121 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive CFTR Mutation and No F508del Mutation - AT
Institution: Information not provided - AT
AUSTRIA
WIEN
ADDRESS: NOT PROVIDED - AT
ESTRELLA: A Phase 2a, Randomized, Double-blind, Placebo-controlled, Safety, Tolerability, Pharmacokinetic and Pharmacodynamic Study of Belcesiran in Patients with PiZZ Alpha-1 Antitrypsin Deficiency - AT
Institution: Information not provided - AT
AUSTRIA
WIEN
WIEN
A Pilot Open Label, Multi-dose, Phase 2 Study to Assess Changes in a Novel Histological Activity Scale in Response to ARO-AAT in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD) - AT
Allgemeines Krankenhaus der Stadt Wien
Klinische Abteilung für Gastroenterologie und Hepatologie
AUSTRIA
WIEN
WIEN
A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis - AT
Allgemeines Krankenhaus der Stadt Wien
Klinische Abteilung für Pädiatrische Pulmologie, Allergologie und Endokrinologie
BELGIA
ANTWERPEN
ANTWERPEN
Een fase 2-onderzoek van Galicaftor/Navocaftor/ABBV-119 combinatietherapie bij proefpersonen met cystische fibrose die homozygoot of heterozygoot zijn voor de F508del-mutatie - BE
GZA Ziekenhuizen campus Sint-Vincentius
Muco-Referentiecentrum Antwerpen
BELGIA
ANTWERPEN
EDEGEM (ANTWERPEN)
Functionele respiratoire beeldvorming om het kortetermijneffect van het product ORKAMBI (Lumacaftor / Ivacaftor) te beoordelen op de longfunctie bij ORKAMBI naive-patiënten met cystische fibrose homozygoot voor Phe508del
Antwerp University Hospital - UZA
Muco-Referentiecentrum (kinderen afdeling)
BELGIA
ARRONDISSEMENT BRUSSELS-CAPITAL
ADDRESS: NOT PROVIDED - BE
Étude de phase 1b, randomisée, en double aveugle, contrôlée par placebo visant à évaluer l'innocuité, la tolérabilité et la pharmacocinétique du QR-010 chez des sujets atteints de fibrose kystique porteurs homozygotes de la mutation DeltaF508 - BE
Institution: Information not provided - BE
BELGIA
ARRONDISSEMENT BRUSSELS-CAPITAL
ADDRESS: NOT PROVIDED - BE
Ship-CT: Een Fase 3 gerandomiseerde, dubbelblinde, gecontroleerde studie van geïnhaleerde 7% hypertone zoutoplossing versus 0,9% isotone zoutoplossing gedurende 48 weken op longstructuur bij 3-6 jaar oud kinderen met Cystic Fibrosis, parallel aan de Noord-Amerikaanse SHIP klinische studie, gemeten met computertomografie (CT) - BE
Institution: Information not provided - BE
BELGIA
ARRONDISSEMENT BRUSSELS-CAPITAL
ADDRESS: NOT PROVIDED - BE
MERGE - MRX-800: Une étude visant à évaluer la sécurité à long terme du maralixibat, un inhibiteur du transporteur d'acide biliaire dépendant du sodium apical (ASBTi), dans le traitement de la maladie hépatique cholestatique chez des sujets qui ont déjà participé à une étude sur le maralixibat - BE
Institution: Information not provided - BE
BELGIA
ARRONDISSEMENT BRUSSELS-CAPITAL
BRUSSELS
RISE/MRX-801: Étude ouverte de phase 2 visant à évaluer l'innocuité et la tolérabilité du maralixibat dans le traitement des nourrissons atteints de maladies hépatiques cholestatiques, y compris la cholestase intrahépatique familiale progressive et le syndrome d'Alagille - BE
Cliniques universitaires Saint-Luc - UCLouvain
Service de gastro-entérologie et hépatologie pédiatrique
BELGIA
ARRONDISSEMENT BRUSSELS-CAPITAL
BRUSSELS
Une étude ouverte de phase 3 évaluant l'innocuité et l'efficacité à long terme de la thérapie combinée VX-445 chez des sujets atteints de fibrose kystique qui sont homozygotes ou hétérozygotes pour la mutation F508del - BE
Cliniques universitaires Saint-Luc - UCLouvain
Centre de référence de la mucoviscidose
BELGIA
ARRONDISSEMENT BRUSSELS-CAPITAL
BRUSSELS
Effets de l'ajout du dispositif SIMEOX sur le drainage autogène chez des patients atteints de mucoviscidose
Cliniques universitaires Saint-Luc - UCLouvain
Service de pneumologie
BELGIA
ARRONDISSEMENT BRUSSELS-CAPITAL
BRUSSELS
ALPINE2: Essai de phase 3B, randomisé, en double aveugle visant à évaluer l'innocuité et l'efficacité de 2 schémas thérapeutiques d'Aztreonam 75 mg en poudre et solvant pour solution de nébuliseur / Aztreonam pour solution pour inhalation (AZLI) chez des sujets pédiatriques atteints de mucoviscidose et à début d' infection/colonisation des voies respiratoires par Pseudomonas Aeruginosa - BE
Hôpital Universitaire des Enfants Reine Fabiola - HUDERF
Service de Pneumologie-Allergologie
BELGIA
ARRONDISSEMENT BRUSSELS-CAPITAL
BRUSSELS
MACRODUCT-3710: Efficacité d'un nouveau système de collecte de la sueur: impact sur le dépistage néonatal et le traitement précoce de la mucoviscidose
Université Catholique de Louvain (UCL)
Louvain centre for Toxicology and Applied Pharmacology
BELGIA
VLAAMS BRABANT
LEUVEN
Une étude multicentrique de phase 3, randomisée, en aveugle visant à évaluer l'efficacité et l'innocuité de l'ALXN1840 administré pendant 48 semaines par rapport au traitement standard chez des patients atteints de la maladie de Wilson âgés de 12 ans et plus avec une période de prolongation pouvant aller jusqu'à 60 mois - BE
UZ Leuven - Campus Gasthuisberg
Dienst maag-, darm- en leverziekten (gastro-enterologie en hepatologie)
BELGIA
VLAAMS BRABANT
LEUVEN
Een fase 3b open-label studie ter evaluatie van de veiligheid van combinatietherapie met elexacaftor/tezacaftor/ivacaftor bij personen met cystische fibrose - BE
UZ Leuven - Campus Gasthuisberg
Mucocentrum
BELGIA
VLAAMS BRABANT
LEUVEN
Een fase 3, open-label, rollover-onderzoek om de veiligheid en werkzaamheid van langdurige behandeling met VX-661 in combinatie met ivacaftor te evalueren bij proefpersonen van 12 jaar en ouder met cystische fibrose, homozygoot of heterozygoot voor de F508del-CFTR-mutatie - BE
UZ Leuven - Campus Gasthuisberg
Mucocentrum
BELGIA
VLAAMS BRABANT
LEUVEN
Een open-label fase 3b-onderzoek om het effect van Elexacaftor/Tezacaftor/Ivacaftor op glucosetolerantie te beoordelen bij personen met cystische fibrose met abnormaal glucosemetabolisme - BE
UZ Leuven - Campus Gasthuisberg
Mucocentrum
BELGIA
VLAAMS BRABANT
LEUVEN
Une étude d'extension de phase 3 visant à évaluer l'innocuité et l'efficacité du traitement à long terme par Lumacaftor en association avec l'Ivacaftor chez des sujets âgés de 12 ans et plus atteints de fibrose kystique homozygote ou hétérozygote pour la mutation F508del-CFTR - BE
UZ Leuven - Campus Gasthuisberg
UZ Leuven
KANADA
Québec
ADDRESS: NOT PROVIDED - CA
A Phase 3, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation - CA
Institution: Information not provided - CA
KANADA
Québec
ADDRESS: NOT PROVIDED - CA
Long Term Safety of Alpha1-Proteinase Inhibitor in Subjects With Alpha1 Antitrypsin Deficiency (SPARTA-OLE)-CA
Institution: Information not provided - CA
KANADA
Québec
ADDRESS: NOT PROVIDED - CA
A Phase 2, Proof-of-concept, Multicentre, Double-blind, Randomised, Dose-ascending, Sequential Group, Placebo-controlled Study to Evaluate the Mechanistic Effect, Safety, and Tolerability of 12 Weeks Twice Daily Oral Administration of Alvelestat (MPH966) in Participants With Alpha-1 (PiZZ or Null) Antitrypsin Deficiency-CA
Institution: Information not provided - CA
KANADA
Québec
ADDRESS: NOT PROVIDED - CA
A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF) - CA
Institution: Information not provided - CA
KANADA
Québec
ADDRESS: NOT PROVIDED - CA
A Phase 2, Randomized, Double-blind, Controlled Study to Evaluate the Safety and Efficacy of VX-440 Combination Therapy in Subjects Aged 12 Years and Older With Cystic Fibrosis-CA
Institution: Information not provided - CA
DANIA
Jylland
ADDRESS: NOT PROVIDED - DK
Evaluation of the effect of Triheptanoin on fatty acid oxidation and exercise tolerance in patients with debrancher deficiency, glycogenin-1 deficiency and phosphofructokinase deficiency at rest and during exercise. A randomized, double-blind, placebo-controlled, cross-over study.
Institution: Information not provided - DK
ESTONIA
Tartu
TARTU
EDIT : A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Phase III Study in Cystic Fibrosis (CF) Subjects to Assess Efficacy, Safety and Pharmacokinetics of Tobramycin Inhalation Powder from a Modified Manufacturing Process (TIPnew)
Children's Clinic - Tartu University Hospital
Allergy Centre for Children and Teenagers
ESTONIA
Tartu
TARTU
An Open-Label, Multicenter Study to Evaluate the Long-term Safety of Weekly Intravenous Alpha1-Proteinase Inhibitor (Human), Modified Process 60 mg/kg in Subjects With Pulmonary Emphysema Due to Alpha1-Antitrypsin Deficiency - EE
Lung Clinic - Tartu University Hospital
Lung Clinic
FRANCJA
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
A Randomized, Double-Blind, Placebo-Controlled, Crossover Multi-Center Study to Assess the Efficacy and Safety of Inhaled Tobramycin Nebuliser Solution (TOBI®) for the Treatment of Early Infections of P. aeruginosa in Cystic Fibrosis Subjects Aged from 3 Months to less than 7 years - FR
Institution: Information not provided - FR
FRANCJA
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Crossover Study to Evaluate the Efficacy and Safety of Ivacaftor and VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation, and a Second Allele With a CFTR Mutation Predicted to Have Residual Function
Institution: Information not provided - FR
FRANCJA
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Etude roll-over de phase III visant à évaluer l'innocuité et l'efficacité du traitement à long terme par Lumacaftor en association avec l'Ivacaftor chez des sujets âgés de 12 ans et plus atteints de Mucoviscidose homozygote ou hétérozygote pour la mutation F508del-CFTR - FR
Institution: Information not provided - FR
FRANCJA
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
MERGE - MRX-800: A Long-Term Safety Study of Maralixibat, an Apical Sodium Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study - FR
Institution: Information not provided - FR
FRANCJA
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
A Phase 3b Open-label Study to Assess the Effect of Elexacaftor/Tezacaftor/Ivacaftor on Glucose Tolerance in Cystic Fibrosis Subjects with Abnormal Glucose Metabolism - A Study to Assess the Effect of ELX/TEZ/IVA on Glucose Tolerance in Subjects With Cystic Fibrosis-FR
Institution: Information not provided - FR
FRANCJA
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
RISE: Open-Label, Phase 2 Study to Evaluate the Safety and Tolerability of Maralixibat in the Treatment of Infants with Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome. - Maralixibat Infant Safety Evaluation -FR
Institution: Information not provided - FR
FRANCJA
AUVERGNE-RHONE-ALPES
BRON
Ship-CT: Etude de phase 3, randomisée, contrôlée, en double aveugle évaluant l'effet d'un traitement de 48 semaines de sérum salé hypertonique (SSH) à 7% en comparaison d'une solution non hypertonique à 0,9% chez des enfants de 3 à 6 ans inclus atteints de mucoviscidose - FR
CHU de Lyon HCL - GH Est-Hôpital Femme Mère Enfant
Service de pneumologie et allergologie pédiatrique, mucoviscidose
FRANCJA
HAUTS-DE-FRANCE
LILLE
TOBRAMUC : Evaluation de l'efficacité d'une cure associant un (ou plusieurs) antibiotique(s) avec 14 jours de tobramycine (Nebcine®) en injection intra-veineuse versus la même cure d'antibiotique(s) associée à seulement 5 jours de tobramycine (Nebcine®) en injection intra-veineuse suivi d'aérosols de tobramycine (Tobi®) pendant 9 jours dans le cadre de la mucoviscidose (Phase III)
CHRU de Lille - Hôpital Albert Calmette
Clinique de Pneumologie - CRC mucoviscidose
FRANCJA
OCCITANIE
TOULOUSE
MUCOLAX : Effet d'un traitement par polyéthylène glycol sur l'inflammation intestinale associée a la mucoviscidose chez l'enfant
CHU de Toulouse - Hôpital des Enfants
Service de Pédiatrie - Pneumologie et allergologie
NIEMCY
Baden-Württemberg
HEIDELBERG
CHELATE STUDY: Trientin-Tetrahydrochlorid (TETA 4HCL) zur Behandlung der Wilson-Krankheit (Phase 3)
Universitätsklinikum Heidelberg
NIEMCY
Baden-Württemberg
HEIDELBERG
ANAKIN: Phase IIa, randomisierte, Placebo-kontrollierte, doppel-blinde, cross-over Studie zur Untersuchung von Sicherheit und Wirksamkeit der subkutanen Verabreichung von Anakinra bei Patienten mit Cystischer Fibrose (CF)
Universitätsmedizin Heidelberg
Zentrum für Seltene Erkrankungen (ZSE) Heidelberg
NIEMCY
Bayern
MÜNCHEN
CHELATE STUDY: Trientin-Tetrahydrochlorid (TETA 4HCL) zur Behandlung der Wilson-Krankheit (Phase 3)
LMU Klinikum der Universität München - Campus Großhadern
LMU Klinikum
NIEMCY
Berlin
ADDRESS: NOT PROVIDED - DE
Eine Phase III Rollover-Studie zur Untersuchung der Sicherheit und Wirksamkeit von langfristig angewendetem Lumacaftor in Kombination mit Ivacaftor bei Patienten mit Mukoviszidose ab einem Alter von 6 Jahren und mit homozygoter F508del-CFTR-Mutation -DE-
Institution: Information not provided - DE
NIEMCY
Berlin
ADDRESS: NOT PROVIDED - DE
Eine explorative Phase 2, 2-teilige, randomisierte, doppelblinde, Placebo-kontrollierte Studie mit einer langfristigen, offenen Labelperiode zur Untersuchung der Auswirkungen von Lumacaftor/Ivacaftor auf den Krankheitsverlauf in Probanden im Alter von 2 bis 5 Jahren mit Mukoviszidose, homozygot für F508del
Institution: Information not provided - DE
NIEMCY
Berlin
ADDRESS: NOT PROVIDED - DE
Eine offene Phase-3-, 2-armige Studie zur Evaluierung der Sicherheit und Pharmakodynamik der langfristigen Ivacaftor-Behandlung bei Patienten mit zystischer Fibrose, die bei Behandlungsbeginn jünger als 24 Monate sind und eine bestätigte Ivacaftor-responsive Mutation aufweisen
Institution: Information not provided - DE
NIEMCY
Berlin
ADDRESS: NOT PROVIDED - DE
Phase 1b, Randomisierte, doppelblinde, Placebo-kontrollierte, Dosis-Eskalationsstudie zur Beurteilung der Sicherheit, Verträglichkeit und Pharmakokinetik von QR-010 bei CF-Patienten mit homozygoter deltaF508-Mutation
Institution: Information not provided - DE
NIEMCY
Berlin
ADDRESS: NOT PROVIDED - DE
VX19-445-115: Eine offene Phase-3b-Studie zur Evaluierung der Sicherheit der Kombinationstherapie Elexacaftor/Tezacaftor/Ivacaftor bei Mukoviszidose
Institution: Information not provided - DE
NIEMCY
Rheinland-Pfalz
MAINZ
OrkambiKIDS: Einfluss von CFTR-Modulation mit Lumacaftor/Ivacaftor auf das intestinal und respiratorische Mikrobiom und mikrobielle Metabolite bei Kindern im Alter von 2- 12 Jahren
Universitätsmedizin Mainz
Zentrum für Kinder- und Jugendmedizin
IRLANDIA
County Cork
CORK
IMPACTT (PsAer-IgY): Prospective, randomized, placebo-controlled, double blind, multicenter phase III study to evaluate clinical efficacy and safety of Avian Polyclonal Anti-Pseudomonas Antibodies (IgY) in prevention of recurrence of Pseudomonas Aeruginosa infection in Cystic Fibrosis patients - IE
Cork University Hospital
Department of Respiratory Medicine
IRLANDIA
County Dublin
ADDRESS: NOT PROVIDED - IE
A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445/TEZ/IVA Combination Therapy in Subjects With Cystic Fibrosis Who Are 6 Years of Age and Older-IE
Institution: Information not provided - IE
IRLANDIA
County Dublin
ADDRESS: NOT PROVIDED - IE
A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-121 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive CFTR Mutation and No F508del Mutation - IE
Institution: Information not provided - IE
IRLANDIA
County Dublin
ADDRESS: NOT PROVIDED - IE
A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation - IE
Institution: Information not provided - IE
IRLANDIA
County Dublin
ADDRESS: NOT PROVIDED - IE
A Phase 2, Randomized, Double-blind Study to Evaluate the Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis-IE
Institution: Information not provided - IE
WŁOCHY
EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT
Studio di fase III di Ataluren (PTC124) in soggetti mutazione non senso affetti da Fibrosi Cistica - IT
Institution: Information not provided - IT
WŁOCHY
EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT
Studio esplorativo per valutare la tollerabilità di amfotericina B in complesso lipidico (Abelcet) mediante aerosol in pazienti con Fibrosi cistica e Aspergillosi broncopolmonare allergica - CRCFC-ABLC003
Institution: Information not provided - IT
WŁOCHY
LAZIO
ROMA
Studio sperimentale, randomizzato, in aperto, per valutare l'effetto della supplementazione di acido docosaesaenoico (DHA) verso 5 -metiltetraidrofolato (5-MTHF) + vitamina B12 e DHA, in pazienti con fibrosi cistica e insufficienza pancreatica.
IRCCS Ospedale Pediatrico Bambino Gesù - SEDE GIANICOLO
U.O.C. di Fibrosi Cistica
WŁOCHY
LAZIO
ROMA
Studio randomizzato di fase III per valutare efficacia e la sicurezza di Aztreonam Lisina inalazione vs Tobramicina Soluzione nebulizzazione, nel contesto di un regime antibiotico aerosolizzato intermittente in pazienti con fibrosi cistica.
IRCCS Ospedale Pediatrico Bambino Gesù - SEDE GIANICOLO
U.O.C. di Fibrosi Cistica
HOLANDIA
Limburg
MAASTRICHT
Does Arginine Enhance Galactose Oxidative Capacity in Classic Galactosemia: A Pilot Study
Maastricht UMC+
Afdeling Kindergeneeskunde
HOLANDIA
Utrecht
ADDRESS: NOT PROVIDED - NL
A Phase 3b Open-label Study to Assess the Effect of Elexacaftor/Tezacaftor/Ivacaftor on Glucose Tolerance in Cystic Fibrosis Subjects with Abnormal Glucose Metabolism - NL
Institution: Information not provided - NL
HOLANDIA
Utrecht
UTRECHT
The TRIO study: Genistein as an add-on treatment for CF?
UMC Utrecht - Universitair Medisch Centrum Utrecht
Afdeling Longziekten voor kinderen
HOLANDIA
Zuid-Holland
ROTTERDAM
Ship-CT: A Phase 3 randomised, double-blind, controlled trial of inhaled 7% hypertonic saline versus 0.9% isotonic saline for 48 weeks in patients with Cystic Fibrosis at 3-6 years of age in parallel with the North American SHIP clinical trial - NL
Erasmus MC - Erasmus Medisch Centrum
Afdeling Kinderlongziekten
POLSKA
Poznań
POZNAŃ
Otwarte, wieloośrodkowe, randomizowane, skrzyżowane badanie porównawcze skuteczności i bezpieczeństwa PANZYTRATU 25 000 i KREONU 25 000 w kontroli biegunki tłuszczowej u pacjentów w wieku 7 lat i starszych chorujących na mukowiscydozę i zewnątrzwydzielniczą niewydolność trzustki
Szpital Kliniczny im. K. Jonschera
Klinika Gastroenterologii Dzieciecej I Chorob Metabolicznych
SŁOWACJA
Bratislavsky kraj
ADDRESS: NOT PROVIDED - SK
A Phase 2 Study of Galicaftor/Navocaftor/ABBV-119 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation -SK
Institution: Information not provided - SK
HISZPANIA
Madrid
ADDRESS: NOT PROVIDED - ES
Estudio de fase III, multicéntrico, randomizado y con enmascaramiento del evaluador, para evaluar la eficacia y la seguridad de WTX101 administrado durante 48 semanas frente al tratamiento de referencia en pacientes con enfermedad de Wilson de 18 años en adelante con una fase de extensión de hasta 60 meses -ES
Institution: Information not provided - ES
HISZPANIA
Madrid
ADDRESS: NOT PROVIDED - ES
Estudio en fase III, abierto, para evaluar la eficacia y la seguridad a largo plazo del tratamiento de combinación VX-445 en sujetos con fibrosis quística que son heterocigóticos para la mutación F508del y una mutación de activación o de función residual (genotipos F/A y F/FR) -ES
Institution: Information not provided - ES
HISZPANIA
Madrid
ADDRESS: NOT PROVIDED - ES
Estudio de fase II, de un solo grupo y cegado para el patólogo utilizando muestras de biopsia hepática para evaluar la concentración de cobre y los cambios histopatológicos en pacientes con enfermedad de Wilson que reciben tratamiento con ALXN1840 durante 48 semanas, seguido de un período de extensión de tratamiento con ALXN1840 durante un máximo de 48 semanas adicionales -ES
Institution: Information not provided - ES
HISZPANIA
Madrid
ADDRESS: NOT PROVIDED - ES
Estudio en fase IIIb, abierto, para evaluar la eficacia y seguridad a largo plazo del tratamiento combinado de elexacaftor/tezacaftor/ivacaftor en pacientes con fibrosis quística de 6 años de edad en adelante heterocigotos para la mutación F508del y una mutación mínima de la función (F/MF) -ES
Institution: Information not provided - ES
HISZPANIA
Madrid
ADDRESS: NOT PROVIDED - ES
Estudio abierto en fase IIIb para evaluar la seguridad de un tratamiento combinado con elexacaftor/tezacaftor/ivacaftor en sujetos con fibrosis quística -ES
Institution: Information not provided - ES
HISZPANIA
Madrid
ADDRESS: NOT PROVIDED - ES
ESTRELLA: Estudio de fase 2a aleatorizado, doble ciego y controlado con placebo de la seguridad, la tolerabilidad, la farmacocinética y la farmacodinamia de Belcesiran en pacientes con déficit de alfa-1 antitripsina PiZZ
Institution: Information not provided - ES
HISZPANIA
Madrid
ADDRESS: NOT PROVIDED - ES
Estudio multicéntrico, aleatorizado, controlado, abierto y ciego para el evaluador para evaluar la eficacia, la seguridad, la farmacocinética y la farmacodinámica de ALXN1840 en comparación con el tratamiento habitual en participantes pediátricos con enfermedad de Wilson -ES
Institution: Information not provided - ES
HISZPANIA
Madrid
ADDRESS: NOT PROVIDED - ES
Estudio en fase III doble ciego, aleatorizado, controlado con placebo, para evaluar la eficacia y la seguridad de ELX/TEZ/IVA en sujetos con fibrosis quística de 6 años o más con una mutación de CFTR no F508del que responde a ELX/TEZ/IVA -ES
Institution: Information not provided - ES
HISZPANIA
Madrid
ADDRESS: NOT PROVIDED - ES
Estudio clínico aleatorizado, doble ciego, controlado con placebo, multicéntrico, ininterrumpido, adaptativo, de seguridad y búsqueda de dosis en fase III de la transferencia génica mediada por VAA con UX701 para el tratamiento de la enfermedad de Wilson
Institution: Information not provided - ES
HISZPANIA
Madrid
ADDRESS: NOT PROVIDED - ES
Estudio en fase III, aleatorizado, doble ciego, controlado, para evaluar la eficacia y la seguridad de la terapia combinada con VX-121 en sujetos con fibrosis quística que son heterocigotos para la mutación F508del y una mutación de función mínima (F/MF) -ES
Institution: Information not provided - ES
HISZPANIA
Madrid
ADDRESS: NOT PROVIDED - ES
Estudio abierto en fase III que evalúa la eficacia y la seguridad a largo plazo de Elexacaftor/Tezacaftor/Ivacaftor en pacientes con fibrosis quística con genotipos que no son CFTR F508del
Institution: Information not provided - ES
HISZPANIA
Madrid
ADDRESS: NOT PROVIDED - ES
Estudio de fase 3b, multicéntrico y abierto, de la seguridad, tolerabilidad y eficacia del tolvaptán en lactantes, niños y adolescentes de 28 días a menos de 18 años de edad con poliquistosis renal autosómica recesiva (ARPKD) -ES
Institution: Information not provided - ES
HISZPANIA
Madrid
MADRID
Estudio de fase I/II, primero en el ser humano, de dos partes, para evaluar la seguridad, la tolerabilidad y la farmacocinética de dosis únicas ascendentes (Parte 1: sin enmascaramiento) y dosis repetidas (Parte 2: aleatorizada, con doble enmascaramiento y controlada con placebo) de UX053 en pacientes con glucogenosis tipo III
Hospital Universitario 12 de Octubre
HISZPANIA
Madrid
MADRID
SZWECJA
Region Stockholm
ADDRESS: NOT PROVIDED - SE
A Phase 3, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor in Subjects Aged 6 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation - SE
Institution: Information not provided - SE
SZWECJA
Region Stockholm
ADDRESS: NOT PROVIDED - SE
A Phase 1/2 Ascending Dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered DCR-A1AT in Healthy Adult Volunteers and Patients With Alpha1-Antitrypsin-Deficiency-Associated Liver Disease - SE
Institution: Information not provided - SE
ZJEDNOCZONE KRÓLESTWO
Greater Manchester
ADDRESS: NOT PROVIDED - UK
VX11-661-101: A Phase 2, Multicenter, Double Blinded, Placebo Controlled Study to Evaluate Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of VX-661 Monotherapy and VX-661/Ivacaftor Cotherapy in Subjects with Cystic Fibrosis, Homozygous or Heterozygous for the F508del CFTR Mutation - UK
Institution: Information not provided - UK
ZJEDNOCZONE KRÓLESTWO
Merseyside
LIVERPOOL
'The cystic fibrosis (CF) anti-staphylococcal antibiotic prophylaxis trial (CF START); a randomised registry trial to assess the safety and efficacy of flucloxacillin as a longterm prophylaxis agent for infants with CF'
Alder Hey Children's Hospital
ZJEDNOCZONE KRÓLESTWO
Nottinghamshire
NOTTINGHAM
Gut Imaging for Function & Transit in Cystic Fibrosis Study 2 (GIFT-CF2
University of Nottingham Research Innovation Services
University of Nottingham (UK)
ZJEDNOCZONE KRÓLESTWO
West Midlands
BIRMINGHAM
ASTRAEUS: A Phase 2, Proof-of-concept, Multicentre, Double-blind, Randomised, Dose-ascending, Sequential Group, Placebo-controlled Study to Evaluate the Mechanistic Effect, Safety, and Tolerability of 12 Weeks Twice Daily Oral Administration of Alvelestat (MPH966) in Participants With Alpha-1 (PiZZ or Null) Antitrypsin Deficiency-GB
Birmingham Children's Hospital NHS Foundation Trust
ZJEDNOCZONE KRÓLESTWO
West Midlands
BIRMINGHAM
ASTRAEUS: A Phase 2, Proof-of-concept, Multicentre, Double-blind, Randomised, Dose-ascending, Sequential Group, Placebo-controlled Study to Evaluate the Mechanistic Effect, Safety, and Tolerability of 12 Weeks Twice Daily Oral Administration of Alvelestat (MPH966) in Participants With Alpha-1 (PiZZ or Null) Antitrypsin Deficiency-GB
University of Birmingham
University of Birmingham HQ
ZJEDNOCZONE KRÓLESTWO
West Yorkshire
LEEDS
HFNT During Exercise in CF (Cystic Fibrosis)
Leeds Children's Hospital
Leeds Regional Paediatric Cystic Fibrosis Centre
STANY ZJEDNOCZONE AMERYKI
Connecticut
CHESHIRE
A Phase 2, open-label study to assess copper and molybdenum balance in participants with Wilson disease treated with ALXN1840
Alexion Pharmaceuticals, Inc.
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
CHELATE STUDY: Trientine tetrahydrochloride (TETA 4HCl) for the treatment of Wilson's disease -GB
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
Proteostasis PTI428: A Phase I/II Multi-center, Randomized, Placebo-Controlled, Study Designed to Assess the Safety, Tolerability, and Pharmacokinetics of PTI-428 in Subjects with Cystic Fibrosis - US
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
A Double Blind, Placebo Controlled, Dose Escalation Trial of Glycerol Phenylbutyrate Corrector Therapy for Cystic Fibrosis
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
A Multicenter Extension Study to Evaluate the Long-Term Safety and Durability of the Therapeutic Effect of LUM001, an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome
Institution: Information not provided - US
AUSTRIA
WIEN
ADDRESS: NOT PROVIDED - AT
MARCH-PFIC: Randomized Double-blind Placebo-controlled Phase 3 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects With Progressive Familial Intrahepatic Cholestasis - AT
Institution: Information not provided - AT
AUSTRIA
WIEN
ADDRESS: NOT PROVIDED - AT
MRX-503: An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC) - AT
Institution: Information not provided - AT
BELGIA
ARRONDISSEMENT BRUSSELS-CAPITAL
BRUSSELS
GLYCO-1B: Évaluation de l'innocuité et de l'efficacité de l'administration d'Empagliflozine en tant que traitement de la neutropénie chez des patients présentant une glycogénose de type 1b et déficients en G6PC3
Cliniques universitaires Saint-Luc - UCLouvain
Cliniques Universitaires Saint-Luc
BELGIA
ARRONDISSEMENT BRUSSELS-CAPITAL
BRUSSELS
MRX-503: Etude d'extension en ouvert évaluant la sécurité et l'efficacité de Maralixibat dans le traitement de sujets atteints de cholestase intrahépatique progressive familiale - BE
Cliniques universitaires Saint-Luc - UCLouvain
Service d'hépato-gastro-entérologie
BELGIA
ARRONDISSEMENT BRUSSELS-CAPITAL
BRUSSELS
MRX-502/MARCH-PFIC: Étude de phase 3, randomisée, en double aveugle, contrôlée contre placebo visant à évaluer l'efficacité et l'innocuité du maralixibat dans le traitement des sujets atteints de cholestase intrahépatique familiale progressive - BE
Cliniques universitaires Saint-Luc - UCLouvain
Service d'hépato-gastro-entérologie
BELGIA
VLAAMS BRABANT
LEUVEN
Een langetermijnonderzoek om de voortdurende veiligheid en werkzaamheid van recombinant humaan zuur-sfingomyelinase te beoordelen bij patiënten met zure sfingomyelinase-deficiëntie - BE
UZ Leuven - Campus Gasthuisberg
Centrum metabole ziekten - UZ Leuven
FRANCJA
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
MRX-503: Etude d'extension en ouvert évaluant la sécurité et l'efficacité de Maralixibat dans le traitement de sujets atteints de Cholestase intrahépatique progressive familiale (PFIC)
Institution: Information not provided - FR
FRANCJA
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
MRX-502: Randomized Double-blind Placebo-controlled Phase 3 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC) - MARCH-PFIC - N/A -FR
Institution: Information not provided - FR
NIEMCY
Berlin
ADDRESS: NOT PROVIDED - DE
MRX-503: Eine offene Erweiterungsstudie zur Evaluierung der langfristigen Sicherheit und Wirksamkeit von Maralixibat bei der Behandlung von Patienten mit progressiver familiärer intrahepatischer Cholestase (PFIC)
Institution: Information not provided - DE
NIEMCY
Niedersachsen
HANNOVER
MRX-502/MARCH-PFIC: Randomisierte, doppelblinde, placebokontrollierte Phase-3-Studie zur Bewertung der Wirksamkeit und Sicherheit von Maralixibat bei der Behandlung von Patienten mit progressiver familiärer intrahepatischer Cholestase
Medizinische Hochschule Hannover
Klinik für Pädiatrische Pneumologie, Allergologie und Neonatologie
NIEMCY
Rheinland-Pfalz
MAINZ
ASCEND: Eine multizentrische , randomisierte, doppelblinde , Placebo-kontrollierte Phase 2/3-Wiederholungsdosis-Studie zur Untersuchung der Wirksamkeit, Sicherheit, Pharmakodynamik und Pharmakokinetik von Olipudase alfa bei Patienten mit Säure-Sphingomyelinase-Mangel
Universitätsmedizin Mainz
Villa Metabolica - Schwerpunktbereich angeborene Stoffwechselerkrankungen
NIEMCY
Sachsen
CHEMNITZ
ENVISION: Eine randomisierte, doppelblinde, placebokontrollierte multizentrische Phase-3- Studie mit offener Verlängerung zur Evaluierung der Wirksamkeit und Sicherheit von Givosiran bei Patienten mit akuter hepatischer Porphyrie
Klinikum Chemnitz gGmbH
Zentrum für Innere Medizin II
WŁOCHY
EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT
MRX-503: studio di estensione in aperto per valutare la sicurezza e l'efficacia a lungo termine di maralixibat nel trattamento di soggetti con colestasi intraepatica familiare progressiva (PFIC) - IT
Institution: Information not provided - IT
HOLANDIA
Noord-Holland
AMSTERDAM
ASCEND: A Phase 2/3, Multicenter, Randomized, Double-blinded, Placebo-controlled, Repeat-dose Study to Evaluate the Efficacy, Safety, Pharmacodynamics, and Pharmacokinetics of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency - NL
Amsterdam UMC, locatie AMC
Afdeling Erfelijke Stofwisselingsziekten
HISZPANIA
Madrid
ADDRESS: NOT PROVIDED - ES
Estudio de Fase 3, Aleatorizado, Doble Ciego y Controlado con Placebo de la Transferencia del Gen de la Glucosa-6-fosfatasa Mediada por el Virus Adenoasociado de serotipo 8 en Pacientes con Glucogenosis de Tipo Ia
Institution: Information not provided - ES
HISZPANIA
Madrid
MADRID
ASCEND: Estudio en fase II/III, multicéntrico, aleatorizado, doble ciego, controlado con placebo, con dosis repetidas y de comparación de dosis para evaluar la eficacia, seguridad, farmacodinámica y farmacocinética de olipudasa alfa en pacientes con deficiencia de esfingomielinasa ácida -ES
Hospital Universitario Ramón y Cajal
Servicio de Hematología y Hemoterapia
ZJEDNOCZONE KRÓLESTWO
Greater London
LONDON
A Long-Term Study to Assess the Ongoing Safety and Efficacy of Recombinant Human Acid Sphingomyelinase in Patients With Acid Sphingomyelinase Deficiency - UK
GOSH NHS Foundatin Trust
Great Ormond Street Hospital
STANY ZJEDNOCZONE AMERYKI
Georgia
ATLANTA
STANY ZJEDNOCZONE AMERYKI
New York
NEW YORK
ASCEND-Peds: A Phase 1/2, Multi-Center, Open-Label, Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Exploratory Efficacy of Olipudase Alfa in Pediatric Patients Aged <18 Years With Acid Sphingomyelinase Deficiency - US
Icahn School of Medicine at Mount Sinai
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
Safety and Efficacy of Panhematin for Prevention of Acute Attacks of Porphyria
Institution: Information not provided - US
BELGIA
VLAAMS BRABANT
LEUVEN
PEDFIC 1: Etude de phase 3, en double-aveugle, randomisée, contrôlée par placebo visant à évaluer l'efficacité et la sécurité d'administration de l'A4250 chez des enfants atteints de cholestase familale intra-hépatique de types 1 et 2 - BE
UZ Leuven - Campus Gasthuisberg
Dienst Kindergeneeskunde/Department of Pediatrics
JAPONIA
JAPAN
ADDRESS : NOT PROVIDED - JP
A clinical trial of efficacy and safety of sodium phenylbutyrate in progressive familial intrahepatic cholestasis type 2
Institution: Information not provided - JP
Wieloośrodkowe badania kliniczne
AUSTRALIA
Victoria
ADDRESS: NOT PROVIDED - AU
A randomised, multicentre, double-blind, placebo-controlled, crossover trial determining the efficacy of dry powder Mannitol in improving lung function in subjects with Cystic Fibrosis aged six to seventeen years (Phase II)
Institution: Information not provided - AU
AUSTRALIA
Victoria
ADDRESS: NOT PROVIDED - AU
A Phase 3b Open-label Study to Assess the Effect of Elexacaftor/Tezacaftor/Ivacaftor on Glucose Tolerance in Cystic Fibrosis Subjects with Abnormal Glucose Metabolism - A Study to Assess the Effect of ELX/TEZ/IVA on Glucose Tolerance in Subjects With Cystic Fibrosis
Institution: Information not provided - AU
BELGIA
ARRONDISSEMENT BRUSSELS-CAPITAL
ADDRESS: NOT PROVIDED - BE
SAF 001: A long-term safety follow-up study of patients suffering from Urea Cycle disorders (UCD) or Crigler-Najjar Syndrome (CN) having received infusions of HepaStem.
Institution: Information not provided - BE
BELGIA
ARRONDISSEMENT BRUSSELS-CAPITAL
ADDRESS: NOT PROVIDED - BE
FALCON: Assessment of Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Oral Doses of the Combination of GLPG2451 and GLPG2222, With or Without GLPG2737, in Adult Subjects With Cystic Fibrosis.
Institution: Information not provided - BE
BELGIA
ARRONDISSEMENT BRUSSELS-CAPITAL
ADDRESS: NOT PROVIDED - BE
A phase IIa, randomized, double-blind, placebo-controlled study to evaluate GLPG2222 in ivacaftor-treated subjects with Cystic Fibrosis harbouring one F508del CFTR mutation and a second gating (class III) mutation
Institution: Information not provided - BE
FRANCJA
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
A Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Study of VX-809 to Evaluate Safety, Pharmacokinetics, and Pharmacodynamics of VX-809 in Cystic Fibrosis Subjects Homozygous for the DeltaF508-CFTR Gene Mutation (Phase II) (coordination)
Institution: Information not provided - FR
FRANCJA
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
CareCN: A Phase I/II, Open Label, Escalating Dose Study to Evaluate Safety and Efficacy of an Intravenous Injection of GNT0003 (AAV Vector Expressing the UGT1A1 Transgene) in Patients With Severe Crigler-Najjar Syndrome Requiring Phototherapy.
Institution: Information not provided - FR
FRANCJA
OCCITANIE
LANGLADE
NIEMCY
Berlin
ADDRESS: NOT PROVIDED - DE
A Randomised, Double-blind, Placebo-controlled Parallel-group Trial to Confirm the Efficacy After 12 Weeks and the Safety of Tiotropium 5 Mcg Administered Once Daily Via the Respimat® Device in Patients With Cystic Fibrosis (Phase III)
Institution: Information not provided - DE
NIEMCY
Berlin
ADDRESS: NOT PROVIDED - DE
Evaluation of the PneumRx, Inc. Lung Volume Reduction Coil for the Treatment of Emphysema in Alpha-1 Antitrypsin Deficient Patients
Institution: Information not provided - DE
WŁOCHY
EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT
Pharmacokinetic study of BRAMITOB® administered for inhalation by PARI EFLOW® rapid electronic nebulizer vs PARI LC® PLUS nebulizer coupled with the PARI TURBO BOY® compressor in cystic fibrosis patients infected with Pseudomonas aeruginosa
Institution: Information not provided - IT
WŁOCHY
EMILIA ROMAGNA
PARMA
A multicentre, multinational, open-label, randomised, parallel group clinical trial of Tobrineb®/Actitob®/Bramitob® (Tobramycine solution for nebulisation, 300 mg twice daily in 4 mL unit dose vials) compared to TOBI® in the treatment of patients with cystic fibrosis and chronic infection with Pseudomonas aeruginosa (Phase III) (coordination)
Chiesi Farmaceutici S.p.A
CHIESI Farmaceutici S.p.A.
HOLANDIA
Utrecht
ADDRESS: NOT PROVIDED - NL
Open-Label, Exploratory Study to Evaluate the Effects of QR-010 on Nasal Potential Difference in Subjects With CF With the deltaF508 CFTR Mutation
Institution: Information not provided - NL
HOLANDIA
Utrecht
ADDRESS: NOT PROVIDED - NL
Phase 1b, Randomized, Double-blind, Placebo-controlled, Dose Escalation Study to Evaluate the Safety, Tolerability and Pharmacokinetics of QR-010 in Subjects With Homozygous deltaF508 Cystic Fibrosis
Institution: Information not provided - NL
NORWEGIA
Østlandet
ADDRESS: NOT PROVIDED - NO
A Double-blind, Randomized, Placebo-controlled Cross Over Study of Inhaled Alginate Oligosaccharide (OligoG) Administered for 28 Days in Subjects With Cystic Fibrosis
Institution: Information not provided - NO
SZWECJA
Region Stockholm
ADDRESS: NOT PROVIDED - SE
Taste and Palatability of Orfadin Suspension. An Open, Non-controlled 3 Day Study in Pediatric Patients With Hereditary Tyrosinemia Type 1 Treated With Orfadin.
Institution: Information not provided - SE
SZWECJA
Region Uppsala
UPPSALA
IMPACTT: Immunoglobulin IgY pseudomonas A clinical trial for cystic fibrosis treatment
Uppsala Universitet
Uppsala University
SZWAJCARIA
Suisse Romande
ADDRESS: NOT PROVIDED - CH
EARLY: A Randomized, double-blind, placebo-controlled, crossover multicenter study to assess the efficacy and safety of inhaled Tobramycin Nebuliser Solution (TOBI®) for the treatment of early infections of pseudomonas aeruginosa in Cystic Fibrosis subjects aged from 3 months to less than 7 years (Phase III)
Institution: Information not provided - CH
ZJEDNOCZONE KRÓLESTWO
Greater London
ADDRESS: NOT PROVIDED - GB
ENVISION : A Phase 3, 2 Part, Randomized, Double-Blind, Placebo Controlled, Parallel Group Study to Evaluate the Pharmacokinetics, Efficacy and Safety of VX 770 in Subjects Aged 6 to 11 Years with Cystic Fibrosis and the G551D Mutation (coordination) - Terminated
Institution: Information not provided - GB
ZJEDNOCZONE KRÓLESTWO
Greater London
ADDRESS: NOT PROVIDED - GB
Open-Label Phase 3 Trial to Evaluate the Safety of Aztreonam 75 mg Powder and Solvent for Nebuliser Solution/Aztreonam for Inhalation Solution (AZLI) in Children With Cystic Fibrosis (CF) and Chronic Pseudomonas Aeruginosa (PA) in the Lower Airways (Coordination)
Institution: Information not provided - GB
ZJEDNOCZONE KRÓLESTWO
Greater London
ADDRESS: NOT PROVIDED - GB
ALPINE: Open-Label Phase 2 Trial to Evaluate the Safety and Efficacy of Aztreonam 75 mg Powder and Solvent for Nebuliser Solution/Aztreonam for Inhalation Solution (AZLI) in Pediatric Patients With Cystic Fibrosis (CF) and New Onset Lower Respiratory Tract Culture Positive for Pseudomonas Aeruginosa (PA)
Institution: Information not provided - GB
ZJEDNOCZONE KRÓLESTWO
Greater London
ADDRESS: NOT PROVIDED - GB
Open multicenter phase III study to asses safety and efficacy of inhalatory Aztreonam Lisine versus Tobramycin solution for nebulization, in the context of a intermittent antibiotic aerosol regimen in patients with cycstic fibrosis.
Institution: Information not provided - GB
ZJEDNOCZONE KRÓLESTWO
Greater London
ADDRESS: NOT PROVIDED - GB
A Phase 2, Randomized, Double-blind, Placebo-controlled, Repeat-dose Study of KB001-A in Subjects With Cystic Fibrosis Infected With Pseudomonas Aeruginosa
Institution: Information not provided - GB
ZJEDNOCZONE KRÓLESTWO
Greater London
ADDRESS: NOT PROVIDED - GB
ASTRAEUS: A Phase 2, Proof-of-concept, Multicentre, Double-blind, Randomised, Dose-ascending, Sequential Group, Placebo-controlled Study to Evaluate the Mechanistic Effect, Safety, and Tolerability of 12 Weeks Twice Daily Oral Administration of Alvelestat (MPH966) in Participants With Alpha-1 (PiZZ or Null) Antitrypsin Deficiency
Institution: Information not provided - GB
STANY ZJEDNOCZONE AMERYKI
California
FOSTER CITY
ALPINE2: Randomized, Double-Blind, Phase 3B Trial to Evaluate the Safety and Efficacy of 2 Treatment Regimens of Aztreonam 75 mg Powder and Solvent for Nebulizer Solution / Aztreonam for Inhalation Solution (AZLI) in Pediatric Subjects With Cystic Fibrosis (CF) and New Onset Respiratory Tract Pseudomonas Aeruginosa (PA) Infection/Colonization
GILEAD SCIENCES, INC.
Gilead Sciences, Inc
STANY ZJEDNOCZONE AMERYKI
California
FOSTER CITY
MERGE - MRX-800: A Long-Term Safety Study of Maralixibat, an Apical Sodium Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study
Mirum Pharmaceuticals, Inc.
STANY ZJEDNOCZONE AMERYKI
Connecticut
CHESHIRE
A Phase 3, Randomized, Rater-Blinded, Multi-Center Study To Evaluate the Efficacy and Safety of ALXN1840 Administered For 48 Weeks Versus Standard of Care in Patients With Wilson Disease Aged 12 Years and Older With an Extension Period of Up To 60 Months
Alexion Pharmaceuticals, Inc.
STANY ZJEDNOCZONE AMERYKI
Florida
ALACHUA
A Multiple-Site, Phase 2, Safety and Efficacy Trial of a Recombinant Adeno-associated Virus Vector Expressing Alpha-1 Antitrypsin (rAAV1-CB-hAAT) in Patients With Alpha-1 Antitrypsin Deficiency
Applied Genetic Technologies Corp
Applied Genetic Technologies Corp.
STANY ZJEDNOCZONE AMERYKI
Illinois
LAKE FOREST
STANY ZJEDNOCZONE AMERYKI
Massachusetts
CAMBRIDGE
ENVISION: A Phase 3 Randomized, Double-blind, Placebo-Controlled Multicenter Study With an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients With Acute Hepatic Porphyrias
Alnylam Pharmaceuticals, Inc.
STANY ZJEDNOCZONE AMERYKI
Massachusetts
CAMBRIDGE
A Phase 2, Randomized, Double-blind, Controlled Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects Aged 18 Years and Older With Cystic Fibrosis
Vertex Pharmaceuticals Inc.
STANY ZJEDNOCZONE AMERYKI
Massachusetts
CAMBRIDGE
A Phase 2, Randomized, Double-blind Study to Evaluate the Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis
Vertex Pharmaceuticals Inc.
STANY ZJEDNOCZONE AMERYKI
New Jersey
SOUTH PLAINFIELD
A Phase 3 Efficacy and Safety Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis (coordination)
PTC Therapeutics Inc.
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
TRAFFIC: A phase 3, randomized, double blind, placebo controlled, parallel group study to evaluate the efficacy and safety of Lumacaftor in combination with Ivacaftor in patients aged 12 years and older with Cystic Fibrosis, homozygous for the F508del CFTR mutation
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
A phase III, randomized, double blind, placebo controlled, parallel group study to evaluate the efficacy and safety of Lumacaftor in combination with Ivacaftor in subjects aged 12 Years and older with Cystic Fibrosis, homozygous for the F508del CFTR mutation
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
A phase 3 extension study of ataluren (PTC124) in subjects with nonsense mutation-mediated cystic fibrosis
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
A Phase 2, Multicenter, Double-Blind, Placebo-Controlled, Multiple-Dose Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Lumacaftor Monotherapy, and Lumacaftor and Ivacaftor Combination Therapy in Subjects With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
A phase II, multicenter, double-blind, placebo controlled, 3-part study to evaluate safety, efficacy, pharmacokinetics, and pharmacodynamics of VX 661 monotherapy, VX 770 monotherapy, and VX 661 in combination with VX 770 in subjects with Cystic Fibrosis, homozygous for the F508del-CFTR mutation
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
TRANSPORT: A phase III, randomized, double blind, placebo controlled, parallel group study to evaluate the efficacy and safety of Lumacaftor in combination with Ivacaftor in subjects aged 12 Years and older with Cystic Fibrosis, homozygous for the F508del CFTR mutation
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
A Phase III, rollover study to evaluate the safety and efficacy of long-term treatment with Lumacaftor in combination with Ivacaftor in subjects aged 12 years and older with Cystic Fibrosis, homozygous or heterozygous for the F508del-CFTR mutation
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
A Phase 3 Efficacy and Safety Study of Ataluren (PTC124®) in Patients with Nonsense Mutation Cystic Fibrosis
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
A Phase 3, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects Aged 6 Through 11 Years With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
A Phase 3, Two-Part, Randomized, Double-Blind, Placebo-Controlled, Crossover Study With an Open-Label Period to Evaluate the Efficacy and Safety of Ivacaftor in Subjects With Cystic Fibrosis Who Have a Non-G551D CFTR Gating Mutation
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Ivacaftor in Subjects With Cystic Fibrosis Who Have the R117H-CFTR Mutation
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
A Phase 1 Non-Randomized, Open-Label Study to Assess the Safety and Pharmacokinetics of VX-770 in Subjects With Moderate Hepatic Impairment and in Matched Healthy Subjects
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
Ship-CT: A Phase 3 randomised, double-blind, controlled trial of inhaled 7% hypertonic saline versus 0.9% isotonic saline for 48 weeks on lung structure in patients with Cystic Fibrosis at 3-6 years of age, in parallel with the North American SHIP clinical trial, as measured by computed tomography (CT)
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
A Double-Blind, Placebo-Controlled, Dose-Escalating, Phase 1 Study to Determine the Safety, Tolerability, Pharmacokinetics and Effect of Circulating Alpha-1 Antitrypsin Levels of ARC-AAT in Healthy Volunteer Subjects and in Patients With Alpha-1 Antitrypsin Deficiency (AATD) (prematurely ended)
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
CHELATE STUDY: Trientine tetrahydrochloride (TETA 4HCl) for the treatment of Wilson's disease
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
Phase 3b, Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Assess the Safety, Efficacy, and Tolerability of Tezacaftor/Ivacaftor (TEZ/IVA) in an Orkambi-experienced Population Who Are Homozygous for the F508del-CFTR Mutation
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
A Phase 3, Open-label, Rollover Study to Evaluate the Safety and Efficacy of Long Term Treatment With VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF).
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
SPARTA-OLE: An Open-Label, Multicenter Study to Evaluate the Long-term Safety of Weekly Intravenous Alpha1-Proteinase Inhibitor (Human), ModifiedProcess 60 mg/kg in Subjects With Pulmonary Emphysema Due to Alpha1-Antitrypsin Deficiency
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
A Phase 1/2 Study of VX-121 in Healthy Subjects and in Subjects With Cystic Fibrosis
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
A Phase 3b, 2-part, Randomized, Double-blind, Placebo-controlled Crossover Study With a Long term Open-label Period to Investigate Ivacaftor in Subjects With Cystic Fibrosis Aged 3 Through 5 Years Who Have a Specified CFTR Gating Mutation
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
A Phase 1/2 Study of VX-445 in Healthy Subjects and Subjects With Cystic Fibrosis
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
A Phase 2, Randomized, Double-blind, Controlled Study to Evaluate the Safety and Efficacy of VX-440 Combination Therapy in Subjects Aged 12 Years and Older With Cystic Fibrosis
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
A Pilot Open Label, Multi-dose, Phase 2 Study to Assess Changes in a Novel Histological Activity Scale in Response to ARO-AAT in Patients with Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD)
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
A Phase 3b Open-label Study Evaluating the Safety of Elexacaftor/Tezacaftor/Ivacaftor Combination Therapy in Cystic Fibrosis Subjects
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
RISE: Open-Label, Phase 2 Study to Evaluate the Safety and Tolerability of Maralixibat in the Treatment of Infants with Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome. - Maralixibat Infant Safety Evaluation.
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
A Phase 3b, Randomized, Placebo-Controlled Study Evaluating the Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects 6 Through 11 Years of Age Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
A Phase 2 Study of Galicaftor/Navocaftor/ABBV-119 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation.
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
California
FOSTER CITY
MRX-503: An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC)
Mirum Pharmaceuticals, Inc.
STANY ZJEDNOCZONE AMERYKI
Massachusetts
CAMBRIDGE
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
MRX-502: Randomized Double-blind Placebo-controlled Phase 3 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC) MARCH-PFIC - N/A
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)
Institution: Information not provided - US
SZWECJA
Region Stockholm
ADDRESS: NOT PROVIDED - SE