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Nationale klinische studie(s)
BELGIË
VLAAMS BRABANT
LEUVEN
CORAL: een multicenter, gerandomiseerd, gecontroleerd versus placebo, dubbelblind, 4 parallelle armen, dosisbereik onderzoek om de werkzaamheid, veiligheid, verdraagbaarheid en aanvaardbaarheid van herhaalde doses van ADV7103 na 7 dagen behandeling te evalueren bij patiënten met cystinurie en een exploratief onderzoek om de werkzaamheid en veiligheid bij de jongste kinderen te evalueren - BE
UZ Leuven - Campus Gasthuisberg
Referentiecentrum voor kindernefrologie
BELGIË
VLAAMS BRABANT
LEUVEN
Open label, multicentre study, evaluating the safety, tolerability, efficacy, compliance and the acceptability of alkalising treatments at long-term in patients with cystinuria - BE
UZ Leuven - Campus Gasthuisberg
Referentiecentrum voor kindernefrologie
DUITSLAND
Baden-Württemberg
HEIDELBERG
SELICA II: Open, prospective, uncontrolled, multicentre study to evaluate the safety and efficacy of multiple applications of liver cell suspension into the portal vein in newborns with Urea Cycle Disorders (UCDs) (Phase II)
Zentrum für Kinder- und Jugendmedizin - Angelika-Lautenschläger-Klinik
Klinik für Kinderheilkunde I - Sektion für Neuropädiatrie und Stoffwechselmedizin
ITALIË
LAZIO
ROMA
Medical treatment of cystinuria
IRCCS Ospedale Pediatrico Bambino Gesù - SEDE GIANICOLO
Dipartimento di Nefrologia e Urologia
OOSTENRIJK
WIEN
ADDRESS: NOT PROVIDED - AT
A Randomised, Controlled, Open-Label Parallel Arm Study of Safety, PK and Ammonia Control of RAVICTI® (Glycerol Phenylbutyrate) Oral Liquid and Sodium Phenylbutyrate in Phenylbutyrate Treatment Naïve Patients With Urea Cycle Disorders (Phase 4) - AT
Institution: Information not provided - AT
OOSTENRIJK
WIEN
ADDRESS: NOT PROVIDED - AT
PEACE (Pegzilarginase Effect on Arginase 1 Deficiency Clinical Endpoints): A Randomized, Double-blind, Placebo-controlled Phase 3 Study of the Efficacy and Safety of Pegzilarginase in Children and Adults With Arginase 1 Deficiency - AT
Institution: Information not provided - AT
SPANJE
Andalucía
MÁLAGA
A Randomised, Controlled, Open-Label Parallel Arm Study of Safety, PK and Ammonia Control of RAVICTI® (Glycerol Phenylbutyrate) Oral Liquid and Sodium Phenylbutyrate in Phenylbutyrate Treatment Naïve Patients With Urea Cycle Disorders (Phase IV) - ES
Hospital Regional Universitario de Málaga - Hospital Materno-Infantil
Unidad de Gastroenterología y Nutrición Infantil
SPANJE
Cataluña
BARCELONA
A Randomised, Controlled, Open-Label Parallel Arm Study of Safety, PK and Ammonia Control of RAVICTI® (Glycerol Phenylbutyrate) Oral Liquid and Sodium Phenylbutyrate in Phenylbutyrate Treatment Naïve Patients With Urea Cycle Disorders (Phase IV) - ES
Hospital Universitari Vall d'Hebron
SPANJE
Galicia
SANTIAGO DE COMPOSTELA
A Randomised, Controlled, Open-Label Parallel Arm Study of Safety, PK and Ammonia Control of RAVICTI® (Glycerol Phenylbutyrate) Oral Liquid and Sodium Phenylbutyrate in Phenylbutyrate Treatment Naïve Patients With Urea Cycle Disorders (Phase IV) - ES
Hospital Clínico Universitario de Santiago
SPANJE
Madrid
ADDRESS: NOT PROVIDED - ES
Open label, multicentre study, evaluating the safety, tolerability, efficacy, compliance and the acceptability of alkalising treatments at long-term in patients with cystinuria (Phase III) - ES
Institution: Information not provided - ES
SPANJE
Madrid
ADDRESS: NOT PROVIDED - ES
CAPtivate: A Phase 1/2, Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults with Late-Onset OTC Deficiency - ES
Institution: Information not provided - ES
SPANJE
Madrid
ADDRESS: NOT PROVIDED - ES
CAPtivate: A Long-Term Follow-up Study to Evaluate the Safety and Efficacy of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults with Late-Onset OTC Deficiency - ES
Institution: Information not provided - ES
SPANJE
Madrid
ADDRESS: NOT PROVIDED - ES
UNLOCKED: A Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of Care - ES
Institution: Information not provided - ES
SPANJE
País Vasco
BARAKALDO
A Randomised, Controlled, Open-Label Parallel Arm Study of Safety, PK and Ammonia Control of RAVICTI® (Glycerol Phenylbutyrate) Oral Liquid and Sodium Phenylbutyrate in Phenylbutyrate Treatment Naïve Patients With Urea Cycle Disorders (Phase IV) - ES
Hospital Universitario Cruces
Unidad de Trastornos Congénitos del Metabolismo
USA
Colorado
AURORA
Oxidative Stress Markers In Inherited Homocystinuria And The Impact Of Taurine (Phase 1/2) - US
Children's Hospital Colorado
Department of Genetics
VERENIGD KONINKRIJK
Greater Manchester
ADDRESS: NOT PROVIDED - UK
An exploratory, randomised, 3-way crossover study to determine the effect of paracetamol on urinary cystine excretion in cystinuria (Phase II)
Institution: Information not provided - UK
VERENIGD KONINKRIJK
Greater Manchester
ADDRESS: NOT PROVIDED - UK
A multicentre, randomised, controlled versus placebo, double-blinded, 4 parallel arms, dose-ranging main study, to evaluate the efficacy, safety and tolerability and acceptability of repeated doses of ADV7103, after 7 days of treatment, in patients with cystinuria, and an efficacy and safety exploratory study in the youngest children-GB
Institution: Information not provided - UK
VERENIGD KONINKRIJK
Greater Manchester
ADDRESS: NOT PROVIDED - UK
Open label, multicentre study, evaluating the safety, tolerability, efficacy, compliance and the acceptability of alkalising treatments at long-term in patients with cystinuria-GB
Institution: Information not provided - UK
CANADA
Québec
MONTRÉAL
Phase 3b Open-Label Study to Evaluate the Effect of Kuvan® on Neurocognitive Function, Maintenance of Blood Phenylalanine Concentrations, Safety, and Population Pharmacokinetics in Young Children With Phenylketonuria (PKU-015) - CA
Glen / McGill Univeristy Health Centre - Centre Universitaire de santé McGill
Medical Genetics - Génétique Médicale
FRANKRIJK
ILE-DE-FRANCE
PARIS
SONIA 2: An international, multicenter, randomized, evaluator-blinded, no-treatment controlled, parallel-group study to assess the efficacy and safety of once daily nitisinone in patients with alkaptonuria after 12 months of treatment, followed by an additional 36-month treatment period (phase III) - FR
Hôpital Necker-Enfants Malades
Service des Maladies Métaboliques pédiatriques
VERENIGD KONINKRIJK
Merseyside
LIVERPOOL
SONIA 2: An international, multicenter, randomized, evaluator-blinded, no-treatment controlled, parallel-group study to assess the efficacy and safety of once daily nitisinone in patients with alkaptonuria after 12 months of treatment, followed by an additional 36-month treatment period (phase III) - UK
Royal Liverpool University Hospital NHS
The Robert Gregory Alkaptonuria Centre (NAC)
IERLAND
County Dublin
DUBLIN 2
KOGNITO: A Phase IV open-label, single-cohort study of the long-term neurocognitive outcomes in 4 to 5 year-old children with Phenylketonuria treated with Sapropterin Dihydrochloride (Kuvan®) for 7 years - DE
BioMarin International Limited
SPANJE
Cataluña
ESPLUGUES DE LLOBREGAT
KOGNITO: A Phase 4 Open-Label, Single-Cohort Study of the Long-Term Neurocognitive Outcomes in 4 to 5 Year-Old Children With Phenylketonuria Treated With Sapropterin Dihydrochloride (Kuvan®) for 7 Years - ES
Hospital Sant Joan de Déu Barcelona
Unidad de Enfermedades Metabólicas Congénitas
VERENIGD KONINKRIJK
Avon
BRISTOL
Treatment of Barth Syndrome by CARDIOlipin MANipulation (CARDIOMAN): A randomised placebo-controlled pilot trial conducted by the nationally commissioned Barth Syndrome Service
Bristol Royal Infirmary
University Hospitals Bristol NHS Foundation Trust HQ
VERENIGD KONINKRIJK
Greater London
LONDON
KOGNITO - A Phase IV Open-Label, Single-Cohort Study of the Long-Term Neurocognitive Outcomes in 4 to 5 Year-Old Children with Phenylketonuria Treated with Sapropterin Dihydrochloride (Kuvan®) for 7 Years - UK
National Hospital for Neurology and Neurosurgery
Metabolic Office
Multinationale klinische studie(s)
BELGIË
ARRONDISSEMENT BRUSSELS-CAPITAL
BRUSSELS
HEP 001: A prospective, open label, multicenter, partially randomised, safety study of one cycle of Promethera HepaStem in Urea Cycle Disorders (UCD) and Crigler-Najjar Syndrome (CN) paediatric patients (Terminated)
Cliniques universitaires Saint-Luc - UCLouvain
Service de gastro-entérologie et hépatologie pédiatrique
USA
Illinois
LAKE FOREST
VERENIGD KONINKRIJK
Merseyside
LIVERPOOL
An international, multicentre, randomised, open-label, no-treatment controlled, parallel group, dose-response study to investigate the effect of once daily nitisinone on 24-hour urinary homogentisic acid excretion in patients with alkaptonuria after 4-weeks treatment (Phase II) - Coordination
Royal Liverpool University Hospital NHS
The Robert Gregory Alkaptonuria Centre (NAC)
VERENIGD KONINKRIJK
Merseyside
LIVERPOOL
DEVELOPAKURE: Clinical Development of Nitisinone for Alkaptonuria
Royal Liverpool University Hospital NHS
The Robert Gregory Alkaptonuria Centre (NAC)
VERENIGD KONINKRIJK
Merseyside
LIVERPOOL
SONIA 1: An international, multicentre, randomised, open-label, no-treatment controlled, parallel group, dose-response study to investigate the effect of once daily nitisinone on 24-hour urinary homogentisic acid excretion in patients with alkaptonuria after 4-weeks treatment (Phase II)
Royal Liverpool University Hospital NHS
Linda McCartney Centre
VERENIGD KONINKRIJK
Merseyside
LIVERPOOL