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Sperimentazione in fase di reclutamento = Sperimentazione in fase di reclutamento
; Sperimentazione in corso = Sperimentazione in corso
; Finanziato da un ente associato a IRDiRC = ; Membro di una ERN =

Sperimentazioni cliniche nazionali

FRANCIA

ILE-DE-FRANCE
GARCHES

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

FRANCIA

ILE-DE-FRANCE
GARCHES

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

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCIA

ILE-DE-FRANCE
PARIS

FRANCIA

NOUVELLE AQUITAINE
LIMOGES

FRANCIA

OCCITANIE
MONTPELLIER

Sperimentazione in corso
MD1003-ALS : Effect of MD1003 in Amyotrophic Lateral Sclerosis: a Randomized Double Blind Placebo Controlled Pilot Study (Phase II)
CHU de Montpellier - Hôpital Gui de Chauliac
Clinique du Motoneurone et Pathologies neuromusculaires

FRANCIA

OCCITANIE
MONTPELLIER

Sperimentazione in corso
Finanziato da un ente associato a IRDiRCROCK-ALS: Inhibition of Rho Kinase (ROCK) with Fasudil as disease-modifying treatment for ALS - FR
CHU de Montpellier - Hôpital Gui de Chauliac
Clinique du Motoneurone et Pathologies neuromusculaires

GERMANIA

Baden-Württemberg
ULM

Sperimentazione in fase di reclutamento
Sperimentazione in corso
Pharyngeal Electrical Stimulation in Amyotrophic Lateral Sclerosis: A Pilot Study With 20 Patients (Phase I/II) DE
Universitäts- und Rehabilitationskliniken Ulm (RKU)
Klinik für Neurologie

GERMANIA

Baden-Württemberg
ULM

Sperimentazione in fase di reclutamento
Sperimentazione in corso
Finanziato da un ente associato a IRDiRCTUDCA-ALS: Safety and Efficacy of TUDCA as add-on Treatment in Patients Affected by ALS - DE-
Universitäts- und Rehabilitationskliniken Ulm (RKU)
Klinik für Neurologie

GERMANIA

Bayern
MÜNCHEN

Sperimentazione in fase di reclutamento
Sperimentazione in corso
Finanziato da un ente associato a IRDiRCROCK-ALS: Inhibition of Rho Kinase (ROCK) With Fasudil as Disease-modifying Treatment for ALS -DE-
Klinikum rechts der Isar der Technischen Universität München
Neurologische Klinik und Poliklinik

IRLANDA

County Dublin
DUBLIN

Sperimentazione in fase di reclutamento
Sperimentazione in corso
Finanziato da un ente associato a IRDiRCTUDCA-ALS: Safety and Efficacy of TUDCA as add-on Treatment in Patients Affected by ALS - IE
Trinity College Dublin
Research Motor Neurone - Trinity Biomedical Sciences Institute

SPAGNA

Comunidad Valenciana
VALENCIA

STATI UNITI

Kansas
KANSAS CITY

Sperimentazione in fase di reclutamento
Sperimentazione in corso
Finanziato da un ente associato a IRDiRCPhase 2 Study of Rasagiline for Treatment of Amyotrophic Lateral Sclerosis - US
University of Kansas Medical Center Research Institute

SVIZZERA

Suisse Alémanique
ST. GALLEN

BELGIO

ARRONDISSEMENT BRUSSELS-CAPITAL
BRUSSELS

BELGIO

LIEGE
LIEGE

Sperimentazione in fase di reclutamento
Sperimentazione in corso
Finanziato da un ente associato a IRDiRCRAINBOWFISH: An Open-Label Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy - BE
Centre Hospitalier Régional de la Citadelle
Centre de Référence des Maladies Neuromusculaires (CRMN)

FRANCIA

ILE-DE-FRANCE
PARIS

DANIMARCA

Hovedstaden
COPENHAGEN

GERMANIA

Baden-Württemberg
FREIBURG

Sperimentazione in corso
Can treatment with human growth hormone increase strength in spinal muscular atrophy type II and III (phase II study)?
Zentrum für Kinder- und Jugendmedizin Freiburg
Klinik für Neuropädiatrie und Muskelerkrankungen

GERMANIA

Baden-Württemberg
FREIBURG

Sperimentazione in corso
An Open Label Multi-part First-in-human Study of Oral LMI070 in Infants With Type 1 Spinal Muscular Atrophy (Phase I/II) - DE
Zentrum für Kinder- und Jugendmedizin Freiburg
Klinik für Neuropädiatrie und Muskelerkrankungen

GERMANIA

Baden-Württemberg
HEIDELBERG

GERMANIA

Baden-Württemberg
STUTTGART

GERMANIA

Bayern
MÜNCHEN

GERMANIA

Bayern
REGENSBURG

Sperimentazione in fase di reclutamento
Sperimentazione in corso
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

GERMANIA

Nordrhein-Westfalen
ESSEN

Sperimentazione in corso
An Open Label Multi-part First-in-human Study of Oral LMI070 in Infants With Type 1 Spinal Muscular Atrophy (Phase I/II) - DE
Universitätsklinikum Essen
Klinik für Kinderheilkunde I - Bereich Neuropädiatrie und SPZ

GERMANIA

Nordrhein-Westfalen
ESSEN

Sperimentazione in fase di reclutamento
Sperimentazione in corso
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

IRLANDA

County Dublin
DUBLIN

Sperimentazione in corso
A Protocol For Nonmetastatic Rhabdomyosarcoma [RMS-2005] (Phase III) - IE
Children's Health Ireland @ Crumlin
National Oncology/ Bone Marrow Transplant Centre and Haematology Department

ITALIA

LOMBARDIA
MILANO

Sperimentazione in fase di reclutamento
Sperimentazione in corso
Studio a lungo termine sulla sicurezza dell'amifampridina fosfato in pazienti ambulatoriali con atrofia muscolare spinale (SMA) tipo 3
Fondazione IRCCS Istituto Neurologico "Carlo Besta"
U.O. Neurologia IV - Malattie Neuromuscolari e Neuroimmunologia

POLONIA

Warszawa
WARSAW

REGNO UNITO; GRAN BRETAGNA

Greater London
LONDON

REGNO UNITO; GRAN BRETAGNA

Greater London
LONDON

Sperimentazione in corso
Finanziato da un ente associato a IRDiRCPhase II Study of Arimoclomol for the Treatment of Sporadic Inclusion Body Myositis (IBM) - GB
UCL Institute of Neurology, University College London,
Clinical Neurosciences

SPAGNA

Andalucía
SEVILLA

Sperimentazione in fase di reclutamento
Sperimentazione in corso
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)

SPAGNA

Andalucía
SEVILLA

Sperimentazione in fase di reclutamento
Sperimentazione in corso
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)

SPAGNA

Cataluña
BADALONA

Sperimentazione in fase di reclutamento
Sperimentazione in corso
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

UNGHERIA

Közép-Magyarország
BUDAPEST

DANIMARCA

Hovedstaden
COPENHAGEN

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

REGNO UNITO; GRAN BRETAGNA

Tyne & Wear
NEWCASTLE-UPON-TYNE

AUSTRIA

SALZBURG
SALZBURG

Sperimentazione in corso
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

BELGIO

ARRONDISSEMENT BRUSSELS-CAPITAL
BRUSSELS

CANADA

Ontario
LONDON

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCIA

ILE-DE-FRANCE
PARIS

GERMANIA

Baden-Württemberg
FREIBURG

Sperimentazione in corso
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

GERMANIA

Bayern
MÜNCHEN

GERMANIA

Bayern
MÜNCHEN

GERMANIA

Nordrhein-Westfalen
ESSEN

IRLANDA

County Dublin
DUBLIN

Sperimentazione in fase di reclutamento
Sperimentazione in corso
Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (PolarisDMD)-IE
Children's Health Ireland @ Temple Street
Department of Neurology

ITALIA

CAMPANIA
NAPOLI

Sperimentazione in corso
(DMD-FOR): Studio mirato alla ricerca del regime steroideo ottimale per la Distrofia Muscolare di Duchenne
AOU Università degli Studi della Campania "Luigi Vanvitelli" - Centro storico
Servizio di Cardiomiologia e Genetica Medica

ITALIA

LAZIO
ROMA

ITALIA

VENETO
PADOVA

Sperimentazione in corso
(DMD-FOR): Studio mirato alla ricerca del regime steroideo ottimale per la Distrofia Muscolare di Duchenne
Azienda Ospedaliera Universitaria di Padova - Campus Biomedico Pietro d'Abano
Neuromuscular Laboratory

REGNO UNITO; GRAN BRETAGNA

Cambridgeshire
CAMBRIDGE

REGNO UNITO; GRAN BRETAGNA

Greater London
LONDON

REGNO UNITO; GRAN BRETAGNA

Greater London
LONDON

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

REGNO UNITO; GRAN BRETAGNA

Greater Manchester
ADDRESS: NOT PROVIDED - UK

SPAGNA

Madrid
MADRID

Sperimentazione in fase di reclutamento
Sperimentazione in corso
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

STATI UNITI

Florida
GAINESVILLE

STATI UNITI

New York
ROCHESTER

SVIZZERA

Suisse Alémanique
BASEL

Sperimentazioni cliniche internazionali

GERMANIA

Bayern
MÜNCHEN

Finanziato da un ente associato a IRDiRCROCK-ALS: Inhibition of Rho Kinase (ROCK) with Fasudil as disease-modifying treatment for ALS
Klinikum rechts der Isar der Technischen Universität München
Neurologische Klinik und Poliklinik

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

REGNO UNITO; GRAN BRETAGNA

Tyne & Wear
NEWCASTLE UPON TYNE

Finanziato da un ente associato a IRDiRCSCOPE-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

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US