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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)

REINO UNIDO

Tyne & Wear
NEWCASTLE-UPON-TYNE

ALEMANHA

Bayern
MÜNCHEN

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

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

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

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

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

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

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

FRANCA

GRAND-EST
VANDOEUVRE-LÈS-NANCY

FRANCA

ILE-DE-FRANCE
PARIS

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

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

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

Noord-Holland
AMSTERDAM

POLONIA

Warszawa
WARSAW

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

REPUBLICA CHECA

Capital City Prague
ADDRESS: NOT PROVIDED - CZ

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. lekárska fakulta UK
General Teaching Hospital and 1st Faculty of Medicine

ALEMANHA

Hamburg
HAMBURG

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)

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
Hospital Clínico Universitario de Santiago
Servicio de Pediatría

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

ITALIA

CAMPANIA
NAPOLI

Financiado 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

PAISES BAIXOS

Utrecht
WOERDEN