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Krajowe badania kliniczne
AUSTRIA
WIEN
ADDRESS: NOT PROVIDED - AT
TALAPRO-1: A phase 2, open-label, response rate study of talazoparib in men with DNA repair defects metastatic castration-resistant prostate cancer who previously received taxane-based chemotherapy and progressed on at least 1 novel hormonal agent (enzalutamide and/or abiraterone acetate/prednisone) - AT
Institution: Information not provided - AT
AUSTRIA
WIEN
ADDRESS: NOT PROVIDED - AT
Phase III Clinical Trial for CPX-351 in Myeloid Leukemia in Children with Down Syndrome 2018 - AT
Institution: Information not provided - AT
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
KANADA
Québec
ADDRESS: NOT PROVIDED - CA
MOVE Trial: A Phase 3, Efficacy and Safety Study of Oral Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP) -GB
Institution: Information not provided - CA
KANADA
Québec
ADDRESS: NOT PROVIDED - CA
A Phase 3, Randomized, Double-Blind Trial of Two Formulations of Setmelanotide (Daily and Weekly) With a Crossover to Open-Label Once Weekly Setmelanotide in Patients With Specific Gene Defects in the Melanocortin-4 Receptor Pathway Who Are Currently on a Stable Dose of the Once Daily Formulation -CA
Institution: Information not provided - CA
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
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
ADDRESS: NOT PROVIDED - FR
Essai de phase 3, randomisé, en double aveugle, évaluant deux formulations de setmelanotide (quotidienne et hebdomadaire) suivi du setmelanotide hebdomadaire en ouvert chez les patients présentant des anomalies génétiques spécifiques de la voie du récepteur de la mélanocortine-4 et traités actuellement avec une dose stable de la formulation quotidienne -FR
Institution: Information not provided - FR
FRANCJA
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Étude de phase 2 randomisée, en double aveugle, contrôlée par placebo, multicentrique, en deux parties, évaluant l'efficacité, l'innocuité et la tolérance du RGH-706 chez les patients atteints du syndrome de Prader-Willi -FR
Institution: Information not provided - FR
FRANCJA
BOURGOGNE-FRANCHE-COMTE
DIJON
SESAM : Essai de phase II en double aveugle, multicentrique et contrôlé par placebo, évaluant l'efficacité et la tolérance de l'alpelisib (BYL719) chez les enfants et les adultes atteints du syndrome de mégalencéphalie-malformation capillaire-polymicrogyrie (MCAP) -FR
CHU Dijon Bourgogne - Hôpital François Mitterrand
Centre Hospitalier Universitaire Dijon
FRANCJA
ILE-DE-FRANCE
PARIS
FancoMob : Etude pilote évaluant la faisabilité de la mobilisation sanguine et de la collection des cellules CD34+ après traitement par G-CSF et plérixafor chez des patients atteints d'Anémie de Fanconi en vue d'un traitement ulterieur par thérapie génique (Phase I-II)
Hôpital Necker-Enfants Malades
Service d'Hématologie adulte
FRANCJA
ILE-DE-FRANCE
PARIS
A Phase 2 Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of a RARy-Specific Agonist (Palovarotene) in the Treatment of Preosseous Flare-ups in Subjects With Fibrodysplasia Ossificans Progressiva (FOP) (Phase II) - FR
Hôpital Necker-Enfants Malades
Service de Génétique Moléculaire
FRANCJA
ILE-DE-FRANCE
PARIS
Prolongation d'une étude de phase 2, en ouvert, évaluant l'éfficacité et la sécurité d'une étude agoniste spécifique de RARy (Palovarotene) dans le traitement des poussées pré-osseuse chez des sujets atteints de Fibrodysplasie ossifiante progressive (FOP)- FR
Hôpital Necker-Enfants Malades
Service de Génétique Moléculaire
FRANCJA
ILE-DE-FRANCE
PARIS
Etude de phase 2, en ouvert, évaluant l'éfficacité et la sécurité d'une étude agoniste spécifique de RARy (Palovarotene) pour la prévention de l'ossification hétérotopique chez des sujets atteints de Fibrodysplasie ossifiante progressive (FOP)
Hôpital Necker-Enfants Malades
Service de Génétique Moléculaire
FRANCJA
ILE-DE-FRANCE
PARIS
ACTHYF: Evaluation de efficacité d'un traitement systématique par acide folinique et hormone thyroîdienne sur le développement psychomoteur du jeune enfant trisomique 21 - FR
Institut Jérôme Lejeune
FRANCJA
OCCITANIE
TOULOUSE
OXYJEUNE : Effets d'administrations intranasales d'ocytocine sur l'anxiété, les troubles du comportement et l'hyperphagie chez des enfants âgés de 3 à 12 ans présentant un syndrome de Prader-Willi (Phase III)
CHU de Toulouse - Hôpital des Enfants
Centre de référence du syndrome de Prader-Willi
FRANCJA
OCCITANIE
TOULOUSE
Étude interventionnelle de suivi à long terme jusqu'à l'âge de 4 ans des enfants atteints du syndrome de Prader-Willi inclus dans l'essai clinique OTBB3 et comparaison avec une cohorte non traitée d'enfants atteints du syndrome de Prader-Willi. -FR
CHU de Toulouse - Hôpital des Enfants
Centre de référence du syndrome de Prader-Willi
NIEMCY
Bayern
GARMISCH-PARTENKIRCHEN
STOPFOP: Saracatinib-Studie zur Vorbeugung von FOP. Eine 6-monatige randomisierte, kontrollierte Doppelblindstudie mit AZD0530 im Vergleich zu Placebo, gefolgt von einer 12-monatigen Open-Label-Verlängerungsphase
Klinikum Garmisch-Partenkirchen GmbH
Abteilung für Kinder- & Jugendmedizin
NIEMCY
Bayern
MÜNCHEN
SynCoRAS: Verbesserung der synaptischen Plastizität und der kognitiven Funktion bei Störungen des RAS-Signalweges (GeNeRARe-Studie)
TUM Fakultät für Medizin
NIEMCY
Bayern
MÜNCHEN
SynCoRAS: Verbesserung der synaptischen Plastizität und der kognitiven Funktion bei Störungen des RAS-Signalweges (GeNeRARe-Studie)
kbo-Kinderzentrum München gemeinnützige GmbH
kbo-Kinderzentrum München
NIEMCY
Berlin
ADDRESS: NOT PROVIDED - DE
MOVE-Studie: Eine Phase 3, Wirksamkeits- und Sicherheitsstudie von oralem Palovaroten zur Behandlung der Fibrodysplasie Ossificans Progressiva (FOP)
Institution: Information not provided - DE
NIEMCY
Berlin
ADDRESS: NOT PROVIDED - DE
Eine randomisierte Phase-3-Doppelblindstudie mit zwei Setmelanotid-Formulierungen (täglich und wöchentlich) mit einem Crossover zu einmal wöchentlichem Setmelanotid mit offener Zulassung bei Patienten mit spezifischen Gendefekten im Melanocortin-4-Rezeptor-Signalweg, die derzeit eine stabile Dosis der einmal täglichen Formulierung erhalten
Institution: Information not provided - DE
WŁOCHY
EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT
Studio in aperto per valutare l'efficacia e la tollerabilità di Rosiglitazone somministrato per via orale in pazienti affetti da Fibrodisplasia Ossea Progressiva (FOP)
Institution: Information not provided - IT
WŁOCHY
EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT
Follow-up di studio di fase I/II sull'impiego di cellule T CaspaCide derivate da donatore familiare parzialmente compatibile sottoposto a procedura di T deplezione alfa-beta, in pazienti pediatrici affetti da disordini ematologici dopo trapianto aploidentico - IT
Institution: Information not provided - IT
WŁOCHY
EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT
Studio randomizzato, in doppio cieco, controllato con placebo, multicentrico, in 2 parti, di fase 2, per valutare l'efficacia, la sicurezza e la tollerabilità di RGH-706 nella sindrome di Prader-Willi
Institution: Information not provided - IT
WŁOCHY
SICILIA
MESSINA
Studio randomizzato, controllato con placebo, cross-over, in doppio cieco di una terapia di supporto metabolico con Ubichinolo Q10 e un complesso multivitaminico B ed E in due coorti di pazienti con autismo idiopatico e sindromico (sindrome di Phelan-McDermid)
Università degli Studi di Messina
JAPONIA
JAPAN
ADDRESS : NOT PROVIDED - JP
Multicenter randomized double-blind comparison test followed by open-label continuous administration test of NPC-12T for Fibrodysplasia Ossificans Progressiva
Institution: Information not provided - JP
HOLANDIA
Zuid-Holland
ROTTERDAM
Dutch Growth Hormone Cohort study in children with Prader-Willi syndrome: Long-term effects on growth, body composition, activity level and psychosocial development
Stichting Kind en Groei
HOLANDIA
Zuid-Holland
ROTTERDAM
Behandeling met N-acetylcysteïne voor skin picking gedrag in kinderen en jongvolwassenen met PWS: een gerandomiseerde, gecontroleerde, cross-over studie
Stichting Kind en Groei
HISZPANIA
Madrid
ADDRESS: NOT PROVIDED - ES
FANCOLEN-II: Ensayo clínico en Fase II para evaluar la eficacia de la infusión de células autólogas CD34+ transducidas con un vector lentiviral portador del gen FANCA (Medicamento Huérfano) en pacientes con anemia de Fanconi subtipo A -ES
Institution: Information not provided - ES
HISZPANIA
Madrid
ADDRESS: NOT PROVIDED - ES
FALKON: Estudio de fase II, de dos partes, con doble enmascaramiento y grupos paralelos, controlado con placebo, para evaluar la eficacia y la seguridad de 2 pautas posológicas de IPN60130 por vía oral para tratar la fibrodisplasia osificante progresiva en participantes de ambos sexos a partir de 5 años de edad
Institution: Information not provided - ES
HISZPANIA
Madrid
ADDRESS: NOT PROVIDED - ES
Seguimiento a largo plazo: estudio clínico de fase I/II para evaluar la seguridad y la eficacia de la infusión de células CD34+ autólogas transducidas con un vector lentiviral portador del gen FANCA (medicamento huérfano) en pacientes con anemia de Fanconi subtipo A
Institution: Information not provided - ES
HISZPANIA
Madrid
ADDRESS: NOT PROVIDED - ES
EPIK-P2: Estudio de fase II doble ciego con un periodo inicial aleatorizado, controlado con placebo y de 16 semanas de duración para evaluar la eficacia, la seguridad y la farmacocinética de alpelisib (BYL719) en pacientes pediátricos y adultos con síndrome de sobrecrecimiento asociado a PIK3CA -ES
Institution: Information not provided - ES
HISZPANIA
Madrid
ADDRESS: NOT PROVIDED - ES
Estudio en fase II, aleatorizado, doble ciego, controlado con placebo, multicéntrico y en 2 partes para evaluar la eficacia, la seguridad y la tolerabilidad de RGH-706 en el síndrome de Prader-Willi -ES
Institution: Information not provided - ES
HISZPANIA
Madrid
ADDRESS: NOT PROVIDED - ES
Progress: Estudio de fase II, aleatorizado, doble ciego y controlado con placebo para evaluar la eficacia, la seguridad y la tolerabilidad de INCB000928 en participantes con fibrodisplasia osificante progresiva -ES
Institution: Information not provided - ES
HISZPANIA
Madrid
MADRID
Ensayo clínico abierto, Fase II para evaluar la eficacia y seguridad del uso de eltrombopag en niños y adolescentes con Anemia de Fanconi
Hospital Infantil Universitario Niño Jesús
Servicio de Hematología y Hemoterapia
HISZPANIA
Madrid
MADRID
SZWECJA
Region Stockholm
ADDRESS: NOT PROVIDED - SE
MOVE Trial: A Phase 3, Efficacy and Safety Study of Oral Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP) - SE
Institution: Information not provided - SE
ZJEDNOCZONE KRÓLESTWO
Greater London
ADDRESS: NOT PROVIDED - GB
A Phase 3, Randomized, Double-Blind Trial of Two Formulations of Setmelanotide (Daily and Weekly) With a Crossover to Open-Label Once Weekly Setmelanotide in Patients With Specific Gene Defects in the Melanocortin-4 Receptor Pathway Who Are Currently on a Stable Dose of the Once Daily Formulation -CA
Institution: Information not provided - GB
ZJEDNOCZONE KRÓLESTWO
Greater London
ADDRESS: NOT PROVIDED - GB
A Phase 3, Randomized, Double-Blind Trial of Two Formulations of Setmelanotide (Daily and Weekly) With a Crossover to Open-Label Once Weekly Setmelanotide in Patients With Specific Gene Defects in the Melanocortin-4 Receptor Pathway Who Are Currently on a Stable Dose of the Once Daily Formulation -GB
Institution: Information not provided - GB
ZJEDNOCZONE KRÓLESTWO
Greater London
LONDON
Follow-up of Phase 1/2 Study of CaspaCIDe T Cells (BPX-501) From an HLA-partially Matched Family Donor After Negative Selection of TCR α β+T Cells in Pediatric Patients Affected by Hematological Disorders-GB
GOSH NHS Foundatin Trust
Great Ormond Street Hospital
ZJEDNOCZONE KRÓLESTWO
Merseyside
LIVERPOOL
Effects of exenatide on appetite and ghrelin levels in patients with Prader-Willi Syndrome
Fazakerley Hospital
University Hospital Aintree - Diabetes and Endocrinology
ZJEDNOCZONE KRÓLESTWO
West Midlands
BIRMINGHAM
Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of Obesity
University Hospital Birmingham
Marshfield Clinic
STANY ZJEDNOCZONE AMERYKI
Ohio
CINCINNATI
Quercetin in Children With Fanconi Anemia; a Pilot Study (Phase 1) - US
Cincinnati Children's Hospital Medical Center
Department of Pediatry
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
Phase 2 Study: Intranasal Oxytocin vs. Placebo for the Treatment of Hyperphagia in Children and Adolescents With Prader-Willi Syndrome
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
Evaluating the Neurophysiologic and Clinical Effects of Single-Dose Acamprosate, Lovastatin, Minocycline and Placebo in Fragile X Syndrome
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
Effects of AFQ056 on Language Learning in Young Children With Fragile X Syndrome (FXS)
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
Wieloośrodkowe badania kliniczne
KANADA
Québec
ADDRESS: NOT PROVIDED - CA
MOVE Trial: A Phase 3, Efficacy and Safety Study of Oral Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP)
Institution: Information not provided - CA
WĘGRY
Dél-Dunántúl
ADDRESS: NOT PROVIDED - HU
A Randomized, Double-blind, Placebo-controlled, Multi-center, 2-part, Phase 2 Study to Evaluate Efficacy, Safety, and Tolerability of RGH-706 in Prader-Willi Syndrome.
Institution: Information not provided - HU
HOLANDIA
Utrecht
ADDRESS: NOT PROVIDED - NL
STOPFOP: Saracatinib Trial TO Prevent FOP
Institution: Information not provided - NL
HOLANDIA
Utrecht
ADDRESS: NOT PROVIDED - NL
Global Growth Hormone Study in Adults With Prader-Willi Syndroom
Institution: Information not provided - NL
SZWAJCARIA
Suisse Romande
ADDRESS: NOT PROVIDED - CH
Sequential, Two-period Study to Assess the Pharmacokinetics, Safety & Tolerability of Single and Multiple Oral Doses of AFQ056 in Patients With FXS (Fragile X Syndrome) Aged 5-11 Years (Cohort 1) and 3-4 Years (Cohort 2) (Phase I) (Coordination)
Institution: Information not provided - CH
SZWAJCARIA
Suisse Romande
ADDRESS: NOT PROVIDED - CH
An Open-label Study to Evaluate the Long-term Safety and Tolerability of AFQ056 in Adolescent Patients With Fragile X Syndrome (Phase II-III) (Coordination)
Institution: Information not provided - CH
SZWAJCARIA
Suisse Romande
ADDRESS: NOT PROVIDED - CH
A Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate the Efficacy and Safety of AFQ056 in Adolescent Patients With Fragile X Syndrome (Phase II-III) (Coordination)
Institution: Information not provided - CH
SZWAJCARIA
Suisse Romande
ADDRESS: NOT PROVIDED - CH
An Open-label Study to Evaluate the Long-term Safety, Tolerability and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome (Phase II) (Coordination)
Institution: Information not provided - CH
SZWAJCARIA
Suisse Romande
ADDRESS: NOT PROVIDED - CH
A Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate AFQ056 in Adult Patients With Fragile X Syndrome (Phase II) (Coordination)
Institution: Information not provided - CH
SZWAJCARIA
Suisse Romande
ADDRESS: NOT PROVIDED - CH
A Randomized, Double-blind, 12-week, Parallel Group, Placebo-controlled Study of Efficacy and Safety of RO4917523 in Patients With Fragile X Syndrome (Phase II) (Coordination)
Institution: Information not provided - CH
ZJEDNOCZONE KRÓLESTWO
Greater London
LONDON
ZEPHYR: A Phase 2b/3 Study to Evaluate the Safety, Tolerability, and Effects of Livoletide (AZP-531), an Unacylated Ghrelin Analog, on Food-related Behaviors in Patients With Prader-Willi Syndrome
Millendo Therapeutics Ltd
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
Texas
HOUSTON
Follow-up of Phase 1/2 Study of CaspaCIDe T Cells (BPX-501) From an HLA-partially Matched Family Donor After Negative Selection of TCR α β+T Cells in Pediatric Patients Affected by Hematological Disorders
Bellicum Pharmaceuticals, Inc.
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
Phase III Clinical Trial for the Treatment of Myeloid Leukemia in Children with Down Syndrome 2018 - ML-DS 2018
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US
EPIK-P2: A Phase II Double-blind Study With an Upfront, 16-week Randomized, Placebo-controlled Period, to Assess the Efficacy, Safety and Pharmacokinetics of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum (PROS)
Institution: Information not provided - US
STANY ZJEDNOCZONE AMERYKI
Washington
ADDRESS: NOT PROVIDED - US