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Recruiting trial = Recruiting trial
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National clinical trial(s)

AUSTRIA

SALZBURG
SALZBURG

Ongoing trial
MitoFibrate 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

AUSTRIA

TIROL
INNSBRUCK

AUSTRIA

WIEN
ADDRESS: NOT PROVIDED - AT

BELGIUM

VLAAMS BRABANT
LEUVEN

Ongoing trial
Immunization against oxLDL in patients with lysosomal lipid diseases and associated metabolic disorders - BE
University Hospitals Leuven - Gasthuisberg
Centrum metabole ziekten - UZ Leuven

CANADA

Québec
MONTRÉAL

Recruiting trial
Ongoing trial
Longitudinal Natural History Study of Patients With Peroxisome Biogenesis Disorders (PBD)
Montreal Children's Hospital Research Institute - McGill University
Montreal Children's Hospital Research Institute

GERMANY

Baden-Württemberg
HEIDELBERG

Ongoing trial
A 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

GERMANY

Baden-Württemberg
HEIDELBERG

GERMANY

Bayern
MÜNCHEN

Recruiting trial
Ongoing trial
CHELATE STUDY: Trientine Tetrahydrochloride (TETA 4HCL) for the Treatment of Wilson's Disease (Phase 3) - DE
Ludwig-Maximilians-Universität München
Klinikum der Universität München

GERMANY

Hamburg
HAMBURG

Recruiting trial
Ongoing trial
Funded by an IRDiRC memberCOMPASS: 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

GERMANY

Hessen
GIEßEN

Ongoing trial
Open-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

ITALY

CAMPANIA
NAPOLI

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

ITALY

LAZIO
ROMA

Recruiting trial
Ongoing trial
Prospective 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

ITALY

LOMBARDIA
MILANO

Recruiting trial
Ongoing trial
Funded by an IRDiRC memberA 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

UNITED KINGDOM

Greater London
LONDON

Ongoing trial
Pharmacodynamic Studies of a Histone Deacetylase Inhibitor in FRDA (Phase II)
Hammersmith Hospital
Gene Control Mechanisms and Disease Research Group

UNITED STATES

Colorado
AURORA

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

BELGIUM

ANTWERPEN
ANTWERPEN-EDEGEM

Recruiting trial
Ongoing trial
Funded by an IRDiRC memberEmbolden: 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)

FRANCE

GRAND-EST
VANDOEUVRE-LÈS-NANCY

Recruiting trial
Ongoing trial
NOVASED: 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

FRANCE

ILE-DE-FRANCE
PARIS

Recruiting trial
Ongoing trial
AOA1 : 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

GERMANY

Hamburg
HAMBURG

SPAIN

Galicia
SANTIAGO DE COMPOSTELA

Recruiting trial
Ongoing trial
Phase I/II Gene Therapy Clinical Trial of scAAV9.U1A.SGSH for Mucopolysaccharidosis IIIA - ES
Complejo Hospitalario Universitario de Santiago
Departamento de Pediatría

SPAIN

Galicia
SANTIAGO DE COMPOSTELA

UNITED KINGDOM

Tyne & Wear
NEWCASTLE UPON TYNE

GERMANY

Hamburg
HAMBURG

GERMANY

Hamburg
HAMBURG

Multinational clinical trial(s)

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US