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Recruiting trial = Recruiting 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

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
Children's Health Ireland @ Crumlin
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)

SPAIN

Cataluña
BADALONA

Recruiting trial
Ongoing trial
GEIS-51: Phase II multicenter trial of palbociclib in second line of advanced sarcomas with CDK4 overexpression - ES
ICO Badalona - Hospital Germans Trias i Pujol
Servicio de Oncología Médica

SPAIN

Madrid
ADDRESS: NOT PROVIDED - ES

UNITED KINGDOM

Greater London
LONDON

UNITED KINGDOM

Greater London
LONDON

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

DENMARK

Hovedstaden
COPENHAGEN

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

GERMANY

Nordrhein-Westfalen
ESSEN

ITALY

LAZIO
ROMA

Ongoing trial
Efficacy of metformin on motility and strength in myotonic dystrophy type 1. A randomized, double blind, placebo-controlled, multicenter clinical trial
A.O.U. Policlinico di Tor Vergata - Facoltà di Medicina e Chirurgia
Dipartimento di Medicina dei Sistemi

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

Recruiting trial
Ongoing trial
Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (PolarisDMD)-IE
Children's Health Ireland @ Temple Street
Department of Neurology

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

SPAIN

Madrid
MADRID

Recruiting trial
Ongoing trial
A Phase 2 Open-Label Study of Continuation Treatment With Combination Pyrimidine Nucleosides in Patients With Thymidine Kinase 2 Deficiency (TK2)
Hospital Universitario 12 de Octubre
Unidad multidisciplinar de enfermedades neuromusculares y ELA

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 STATES

Florida
GAINESVILLE

UNITED STATES

New York
ROCHESTER

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