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Recruiting trial = Recruiting trial
; Ongoing trial = Ongoing trial
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National clinical trial(s)

FRANCE

ILE-DE-FRANCE
GARCHES

Ongoing trial
Long-Term Effect of LIAM (Lung Insufflation Assist Maneuver) on Respiratory Performance in Non-invasive Ventilated Patients Suffering From Neuromuscular Disease - FR
CHU Paris IdF Ouest - Hôpital Raymond Poincaré
Centre d'Investigation Clinique et Technologique 805 - CIC-IT de Garches

FRANCE

ILE-DE-FRANCE
GARCHES

Ongoing trial
Establishment of a Non-invasive and Indirect Measure of Volitional Pimax (PI MAX STIM) - FR
CHU Paris IdF Ouest - Hôpital Raymond Poincaré
Service de Physiologie-Explorations Fonctionnelles

UNITED KINGDOM

Greater Manchester
ADDRESS: NOT PROVIDED - UK

DENMARK

Hovedstaden
COPENHAGEN

GERMANY

Baden-Württemberg
HEIDELBERG

GERMANY

Baden-Württemberg
STUTTGART

GERMANY

Bayern
REGENSBURG

Recruiting trial
Ongoing trial
INFORM2 NivEnt: exploratory multinational phase I/II combination study of Nivolumab and Entinostat in children and adolescents with refractory high-risk malignancies
Universitätsklinikum Regensburg
Abteilung für Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation

GERMANY

Nordrhein-Westfalen
ESSEN

Recruiting trial
Ongoing trial
INFORM2 NivEnt: exploratory multinational phase I/II combination study of Nivolumab and Entinostat in children and adolescents with refractory high-risk malignancies
Universitätsklinikum Essen
Klinik für Kinderheilkunde III - Abteilung für pädiatrische Hämatologie und Onkologie

HUNGARY

Közép-Magyarország
BUDAPEST

IRELAND

County Dublin
DUBLIN

Ongoing trial
A Protocol For Nonmetastatic Rhabdomyosarcoma [RMS-2005] (Phase III) - IE
Our Lady's Children's Hospital
National Oncology/ Bone Marrow Transplant Centre and Haematology Department

ITALY

LIGURIA
GENOVA

Ongoing trial
Five-year actively controlled clinical trial in new onset juvenile dermatomyositis
IRCCS Istituto G. Gaslini - Ospedale Pediatrico
U.O. Pediatria II

SPAIN

Andalucía
SEVILLA

Recruiting trial
Ongoing trial
TRASTS: Phase I-II prospective trial, multicenter, open label, exploring the combination of Trabectedin plus Radiotherapy in Soft Tissue Sarcoma patients - ES
Hospital Universitario Virgen del Rocío
MUsculoSkeletal Tumor Board of Excellence Sevilla (MUSTBE SEVILLA)

SPAIN

Andalucía
SEVILLA

Recruiting trial
Ongoing trial
GEIS-51: Phase II multicenter trial of palbociclib in second line of advanced sarcomas with CDK4 overexpression - ES
Hospital Universitario Virgen del Rocío
MUsculoSkeletal Tumor Board of Excellence Sevilla (MUSTBE SEVILLA)

UNITED KINGDOM

Greater London
LONDON

UNITED KINGDOM

Greater London
LONDON

UNITED KINGDOM

Greater London
LONDON

Recruiting trial
Ongoing trial
Funded by an IRDiRC memberPhase II Study of Arimoclomol for the Treatment of Sporadic Inclusion Body Myositis (IBM) - GB
UCL Institute of Neurology, University College London,
Clinical Neurosciences

UNITED KINGDOM

Greater Manchester
MANCHESTER

UNITED KINGDOM

South Glamorgan
CARDIFF

Recruiting trial
Ongoing trial
EpSSG-RMS-2005: A Protocol For Nonmetastatic Rhabdomyosarcoma (Phase IV) - UK
Children's Hospital for Wales
Department of Paediatric Oncology

UNITED KINGDOM

South Yorkshire
SHEFFIELD

DENMARK

Hovedstaden
COPENHAGEN

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

GERMANY

Nordrhein-Westfalen
ESSEN

UNITED KINGDOM

Tyne & Wear
NEWCASTLE UPON TYNE

Ongoing trial
PTC124-GD-007-DMD: A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy - UK
Newcastle upon Tyne Hospitals NHS Trust
John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine

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

BELGIUM

ARRONDISSEMENT BRUSSELS-CAPITAL
BRUSSELS

CANADA

Ontario
LONDON

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCE

ILE-DE-FRANCE
PARIS

GERMANY

Baden-Württemberg
FREIBURG

Ongoing trial
FOR-DMD: Double-blind randomized phase III trial to find optimum steroid regimen for Duchenne Muscular Dystrophy - DE
Zentrum für Kinder- und Jugendmedizin Freiburg
Klinik für Neuropädiatrie und Muskelerkrankungen

GERMANY

Bayern
MÜNCHEN

GERMANY

Bayern
MÜNCHEN

GERMANY

Nordrhein-Westfalen
ESSEN

IRELAND

County Dublin
DUBLIN

ITALY

CAMPANIA
NAPOLI

Ongoing trial
(FOR-DMD): Duchenne Muscular Dystrophy: Double-blind Randomized Trial to Find Optimum Steroid Regimen
AOU Università degli Studi della Campania "Luigi Vanvitelli" - Centro storico
Servizio di Cardiomiologia e Genetica Medica

ITALY

LAZIO
ROMA

Recruiting trial
Ongoing trial
Open Label, Long-term Safety, Tolerability, and Efficacy Study of GIVINOSTAT in All DMD Patients Who Have Been Previously Treated in One of the GIVINOSTAT Studies - IT
IRCCS Ospedale Pediatrico Bambino Gesù - SEDE SAN PAOLO
Unità di Malattie Neuromuscolari e Neurodegenerative - Laboratorio di Medicina Molecolare

ITALY

VENETO
PADOVA

Ongoing trial
(FOR-DMD): Duchenne Muscular Dystrophy: Double-blind Randomized Trial to Find Optimum Steroid Regimen
Azienda Ospedaliera Universitaria di Padova - Campus Biomedico Pietro d'Abano
Neuromuscular Laboratory

SWITZERLAND

Suisse Alémanique
BASEL

UNITED KINGDOM

Cambridgeshire
CAMBRIDGE

UNITED KINGDOM

Greater London
LONDON

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

UNITED KINGDOM

Greater Manchester
ADDRESS: NOT PROVIDED - UK

UNITED KINGDOM

Tyne & Wear
NEWCASTLE UPON TYNE

Recruiting trial
Ongoing trial
Funded by an IRDiRC memberA Phase III Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - UK
Newcastle upon Tyne Hospitals NHS Trust
John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine

UNITED KINGDOM

Tyne & Wear
NEWCASTLE UPON TYNE

Ongoing trial
An open-label extension study of the long-term safety, tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy (phase III) - UK
Newcastle upon Tyne Hospitals NHS Trust
John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine

UNITED KINGDOM

Tyne & Wear
NEWCASTLE UPON TYNE

Ongoing trial
An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy (phase III) - UK
Newcastle upon Tyne Hospitals NHS Trust
John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine

UNITED STATES

Florida
GAINESVILLE

UNITED STATES

New York
ROCHESTER

UNITED KINGDOM

Greater Manchester
SALFORD

Multinational clinical trial(s)

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED KINGDOM

Tyne & Wear
NEWCASTLE UPON TYNE

Funded by an IRDiRC memberSCOPE-DMD: Consortium for Products across Europe in Duchenne Muscular Dystrophy
Newcastle upon Tyne Hospitals NHS Trust
John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US