Orphanet: Simple search
x

Search for a clinical trial

* (*) mandatory field

165 Result(s)

Sort by

Recruiting trial = Recruiting trial
; Ongoing trial = Ongoing trial
; Funded by an IRDiRC member = ; Member of a ERN =

National clinical trial(s)

UNITED KINGDOM

Greater Manchester
MANCHESTER

Ongoing trial
Funded by an IRDiRC memberComparing the Side-lying Sleep Positioning to Back-lying in Infants With Cleft Palate (SLUMBRS2)
Central Manchester University Hospitals NHS Foundation Trust
Manchester University Hospitals NHS Foundation Trust

UNITED KINGDOM

Merseyside
LIVERPOOL

Ongoing trial
Effects of exenatide on appetite and ghrelin levels in patients with Prader-Willi Syndrome
Fazakerley Hospital
University Hospital Aintree - Diabetes and Endocrinology

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

UNITED KINGDOM

Tyne & Wear
NEWCASTLE-UPON-TYNE

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

GERMANY

Bayern
MÜNCHEN

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

UNITED KINGDOM

Greater London
LONDON

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

BELGIUM

VLAAMS BRABANT
LEUVEN

CZECH REPUBLIC

Capital City Prague
PRAHA

Ongoing trial
FIELD : 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

FRANCE

AUVERGNE-RHONE-ALPES
BRON

Ongoing trial
IMPROVES: 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

FRANCE

AUVERGNE-RHONE-ALPES
CLERMONT-FERRAND

FRANCE

GRAND-EST
VANDOEUVRE-LÈS-NANCY

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

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

GERMANY

Rheinland-Pfalz
MAINZ

IRELAND

County Dublin
DUBLIN

Recruiting trial
Ongoing trial
MPSVI CSP: MPS VI Clinical Surveillance Program (CSP) (Phase IV) - IE
Children's Health Ireland @ Temple Street
National Centre for Inherited Metabolic Disorders

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

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

NETHERLANDS

Noord-Holland
AMSTERDAM

POLAND

Warszawa
WARSAW

UNITED KINGDOM

Greater Manchester
ADDRESS: NOT PROVIDED - UK

UNITED KINGDOM

Greater Manchester
MANCHESTER

Recruiting trial
Ongoing trial
MPSVI CSP: MPS VI Clinical Surveillance Program (CSP) (Phase IV) - UK
St Mary's Hospital
Manchester Cente for Genomic Medicine

UNITED KINGDOM

South Glamorgan
CARDIFF

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)

ITALY

FRIULI VENEZIA GIULIA
UDINE

SPAIN

Galicia
SANTIAGO DE COMPOSTELA

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

Multinational clinical trial(s)

ITALY

CAMPANIA
NAPOLI

Funded by an IRDiRC memberMeuSIX: Sperimentazione clinica per la terapia genica nella MPSIV una grave malattia da accumulo lisosomiale
TIGEM - Telethon Institute of Genetics and Medicine
Laboratorio di Ricerca