Orphanet: Pesquisa simples
x

Pesquisar um ensaio clínico

* (*) campo(s) de preenchimento obrigatório

297 Resultado(s)

Ordenados por

Ensaio em fase de recrutamento = Ensaio em fase de recrutamento; Ensaio a decorrer = Ensaio a decorrer; Financiado por um membro do IRDiRC =

Ensaio (s) clínico (s) nacional (is)

CANADA

Québec
MONTRÉAL

Ensaio em fase de recrutamentoEnsaio a decorrerLongitudinal Natural History Study of Patients With Peroxisome Biogenesis Disorders (PBD)
Montreal Children's Hospital Research Institute - McGill University
Montreal Children's Hospital Research Institute

REINO UNIDO

Tyne & Wear
NEWCASTLE-UPON-TYNE

ALEMANHA

Baden-Württemberg
HEIDELBERG

Ensaio a decorrerSELICA 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

ALEMANHA

Berlin
BERLIN

Ensaio em fase de recrutamentoEnsaio a decorrerA Multi-center, Open-label, Uncontrolled, Single-arm, Extension Study to Determine the Long-term Safety and Tolerability of Oral Lucerastat in Adult Subjects With Fabry Disease (Phase 3) -DE-
Charité - Universitätsmedizin Berlin (CVK)
Medizinische Klinik mit Schwerpunkt Nephrologie und Internistische Intensivmedizin

ALEMANHA

Hamburg
HAMBURG

Ensaio em fase de recrutamentoEnsaio a decorrerFinanciado por um membro do IRDiRCCOMPASS: CNS Unmet Medical Need in Mucopolysaccharidosis: A Phase 2 Safety and Pharmacokinetics Study of Ataluren - DE
UKE - Universitätsklinikum Hamburg-Eppendorf
Klinik und Poliklinik für Kinder- und Jugendmedizin

ALEMANHA

Rheinland-Pfalz
MAINZ

BELGICA

VLAAMS BRABANT
LEUVEN

Ensaio a decorrerImmunization against oxLDL in patients with lysosomal lipid diseases and associated metabolic disorders - BE
University Hospitals Leuven - Gasthuisberg
Centrum metabole ziekten - UZ Leuven

ESTADOS UNIDOS

Colorado
AURORA

FRANCA

AUVERGNE-RHONE-ALPES
BRON

Ensaio a decorrerIMPROVES: A Phase IIa Study to Investigate Safety, Pharmacokinetics, and Efficacy of Odiparcil in Patients 16 Years and Above With Mucopolysaccharidosis (MPS) Type VI - FR
CHU de Lyon HCL - GH Est-Hôpital Femme Mère Enfant
Service d'endocrinologie et de diabétologie pédiatriques et maladies héréditaires du métabolisme

FRANCA

AUVERGNE-RHONE-ALPES
CLERMONT-FERRAND

IRLANDA

County Dublin
DUBLIN

Ensaio em fase de recrutamentoEnsaio a decorrerMPSVI CSP: MPS VI Clinical Surveillance Program (CSP) (Phase IV) - IE
Children's University Hospital
National Centre for Inherited Metabolic Disorders

ITALIA

LAZIO
ROMA

Ensaio a decorrerMedical treatment of cystinuria
IRCCS Ospedale Pediatrico Bambino Gesù - SEDE GIANICOLO
Dipartimento di Nefrologia e Urologia

ITALIA

LAZIO
ROMA

Ensaio a decorrerA Long-Term, Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients With Cystinosis (phase III) - IT
IRCCS Ospedale Pediatrico Bambino Gesù - SEDE SAN PAOLO
Laboratorio di Nefrologia - Divisione di Nefrologia e Dialisi

ITALIA

LOMBARDIA
MILANO

Ensaio em fase de recrutamentoEnsaio a decorrerFinanciado por um membro do IRDiRCA Phase I/II Clinical Trial of Hematopoietic Stem Cell Gene Therapy for the Treatment of Metachromatic Leukodystrophy
Istituto San Raffaele Telethon per la Terapia Genica - TIGET
Unità di Ricerca Clinica

PAISES BAIXOS

Gelderland
NIJMEGEN

POLONIA

Warszawa
WARSAW

REINO UNIDO

Greater London
LONDON

Ensaio a decorrerTEETPIM:Trial of Erythrocyte Encapsulated Thymidine Phosphorylase In Mitochondrial Neurogastrointestinal Encephalomyopathy-GB
St George's University of London
Molecular and Clinical Sciences Reseach Institute

REINO UNIDO

Greater Manchester
ADDRESS: NOT PROVIDED - UK

REINO UNIDO

Greater Manchester
ADDRESS: NOT PROVIDED - UK

REINO UNIDO

Greater Manchester
MANCHESTER

Ensaio em fase de recrutamentoEnsaio a decorrerMPSVI CSP: MPS VI Clinical Surveillance Program (CSP) (Phase IV) - UK
St Mary's Hospital
Manchester Cente for Genomic Medicine

REINO UNIDO

South Glamorgan
CARDIFF

REINO UNIDO

Tyne & Wear
NEWCASTLE UPON TYNE

REPUBLICA CHECA

Capital City Prague
PRAHA

Ensaio a decorrerFIELD : A Study of Two Fabrazyme (agalsidase beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms (Phase IIIb) - CZ
Vseobecna fakultni nemocnice a 1. lekarska fakulta UK
General Teaching Hospital and 1st Faculty of Medicine

ALEMANHA

Baden-Württemberg
HEIDELBERG

Ensaio a decorrerA Phase 2, Multicenter, Open-label, Study to Evaluate the Efficacy and Safety of WTX101 Administered for 24 Weeks in Newly Diagnosed Wilson Disease Patients Aged 18 and Older - DE
Zentrum für Innere Medizin (Krehl-Klinik)
Abteilung Innere Medizin IV - Gastroenterologie, Infektionskrankheiten, Vergiftungen

ALEMANHA

Baden-Württemberg
HEIDELBERG

ALEMANHA

Bayern
MÜNCHEN

Ensaio em fase de recrutamentoEnsaio a decorrerCHELATE STUDY: Trientine Tetrahydrochloride (TETA 4HCL) for the Treatment of Wilson's Disease (Phase 3) - DE
Ludwig-Maximilians-Universität München
Klinikum der Universität München

ALEMANHA

Hamburg
HAMBURG

ALEMANHA

Hamburg
HAMBURG

ALEMANHA

Hamburg
HAMBURG

ALEMANHA

Hessen
GIEßEN

Ensaio a decorrerOpen-label study to evaluate efficacy and safety of Cystadane for the treatment of aspartylglucosaminuria -FI
Justus-Liebig-Universität Gießen
Institut für Biochemie - AG Prof. Dr. A. Bindereif

AUSTRIA

WIEN
ADDRESS: NOT PROVIDED - AT

BELGICA

ANTWERPEN
ANTWERPEN-EDEGEM

Ensaio em fase de recrutamentoEnsaio a decorrerFinanciado por um membro do IRDiRCEmbolden: A Global, Multicenter, Open-label, Matched Historical Control Study of Intrathecal SHP611 in Subjects With Late Infantile Metachromatic Leukodystrophy - BE
University Hospital of Antwerp - UZA
Department of pediatric metabolic diseases (Metabole stoornissen kinderen/Metabolic Disorders in Children)

CANADA

Québec
MONTRÉAL

ESPANHA

Galicia
SANTIAGO DE COMPOSTELA

Ensaio em fase de recrutamentoEnsaio a decorrerPhase I/II Gene Therapy Clinical Trial of scAAV9.U1A.SGSH for Mucopolysaccharidosis IIIA - ES
Complejo Hospitalario Universitario de Santiago
Departamento de Pediatría

ESPANHA

Galicia
SANTIAGO DE COMPOSTELA

ESTADOS UNIDOS

Texas
DALLAS

Ensaio a decorrerFinanciado por um membro do IRDiRCPhase 2 Study of Orlistat and SLx-4090 for the treatment of Type 1 Hyperlipoproteinemia - US
University of Texas Southwestern Medical Center
Department of Internal Medicine

ESTADOS UNIDOS

Washington
ADDRESS: NOT PROVIDED - US

FRANCA

GRAND-EST
VANDOEUVRE-LÈS-NANCY

FRANCA

GRAND-EST
VANDOEUVRE-LÈS-NANCY

Ensaio em fase de recrutamentoEnsaio a decorrerNOVASED: Efficiency Clinical Study of NOVATEX MEDICAL Compression Garments in Patients With Ehlers-Danlos Syndrome.
CHU de Nancy - Hôpitaux de Brabois
Service de médecine interne et immunologie clinique

FRANCA

ILE-DE-FRANCE
PARIS

ITALIA

CAMPANIA
NAPOLI

Ensaio a decorrerEfficacy and safety of treatment with miglustat in patients with Niemann-Pick disease type c
Azienda Ospedaliera Universitaria "Federico II"
Laboratorio Dipartimento di Pediatria

PAISES BAIXOS

Limburg
MAASTRICHT

Ensaio a decorrerDoes Arginine Enhance Galactose Oxidative Capacity in Classic Galactosemia: A Pilot Study
AZM - Academisch Ziekenhuis Maastricht
Afdeling Kindergeneeskunde

PAISES BAIXOS

Noord-Holland
AMSTERDAM

REINO UNIDO

Greater London
LONDON

REPUBLICA CHECA

Capital City Prague
ADDRESS: NOT PROVIDED - CZ

AUSTRIA

SALZBURG
SALZBURG

Ensaio a decorrerMitoFibrate CT1: Clinical study for the assessment of safety and efficacy of Bezafibrate as a drug therapy for patients with mitochondrial myopathies (phase II) - AT
Universitätsklinikum der Paracelsus Medizinischen Privatuniversität
Universitätsklinik für Kinder- und Jugendheilkunde

FRANCA

AUVERGNE-RHONE-ALPES
LYON

ITALIA

LAZIO
ROMA

Ensaio em fase de recrutamentoEnsaio a decorrerProspective open label trial to evaluate the maintenance of the efficacy and the long term safety of EPI-743 in children with Leigh Disease (Phase II) - IT
IRCCS Ospedale Pediatrico Bambino Gesù - SEDE SAN PAOLO
Unità di Malattie Neuromuscolari e Neurodegenerative - Laboratorio di Medicina Molecolare

ALEMANHA

Bayern
MÜNCHEN

FRANCA

ILE-DE-FRANCE
PARIS

Ensaio em fase de recrutamentoEnsaio a decorrerAOA1 : Evolution of Albumin on AOA1 Patients Supplemented With Coenzyme Q10 (Phase III)
Institut du Cerveau et de la Moelle épinière (ICM) - Hôpital Pitié-Salpêtrière
Institut du Cerveau et de la Moëlle épinière - INSERM U1127

Ensaio (s) clínico (s) multinacional (ais)

ITALIA

CAMPANIA
NAPOLI

Ensaio a decorrerFinanciado por um membro do IRDiRCMeuSIX: Clinical trial of gene therapy for MPS VI - a severe lysosomal storage disorder
TIGEM - Telethon Institute of Genetics and Medicine
Laboratorio di Ricerca

SUECIA

Stockholms läns landsting
STOCKHOLM

ESTADOS UNIDOS

Washington
ADDRESS: NOT PROVIDED - US

PAISES BAIXOS

Utrecht
WOERDEN

REINO UNIDO

Merseyside
LIVERPOOL

Ensaio a decorrerFinanciado por um membro do IRDiRCDEVELOPAKURE: Clinical Development of Nitisinone for Alkaptonuria
Royal Liverpool University Hospital NHS
The Robert Gregory Alkaptonuria Centre (NAC)

ESTADOS UNIDOS

Washington
ADDRESS: NOT PROVIDED - US