RecruitingPhase 2Phase 3NCT03373968

Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study

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

Sponsor

Italfarmaco

Enrollment

206 participants

Start Date

Oct 24, 2017

Study Type

INTERVENTIONAL

Conditions

Duchenne Muscular Dystrophy

Summary

This is an open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD (Duchenne's muscular dystrophy) patients who have been previously treated in one of the GIVINOSTAT studies.

Eligibility

Sex: MALEMin Age: 7 Years

Plain Language Summary

Simplified for easier understanding

This study is evaluating the long-term safety and tolerability of givinostat, a drug being developed to slow the progression of Duchenne Muscular Dystrophy (DMD) — a severe inherited muscle disease that primarily affects boys. This study is for boys who previously participated in an earlier givinostat trial. You may be eligible if: - You are a male, 7 years old or older - You participated in a previous givinostat trial in DMD, OR you were screened for a prior study but were not enrolled because your muscle fat fraction was outside the target range - You are willing to use appropriate contraception if applicable You may NOT be eligible if: - You have used other medications that could affect muscle strength (other than corticosteroids) within 3 months - You have other significant uncontrolled illnesses unrelated to DMD - You have low blood cell counts or very high triglycerides at screening - You have kidney, liver, or heart failure - You have an abnormal heart rhythm (prolonged QT interval) - You have a known hypersensitivity to any study medication ingredients - You have sorbitol intolerance or hereditary fructose intolerance Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGGivinostat

suspension of givinostat (10 mg/mL)

Locations(39)

University of California - Davis Medical Center - Devis Physical Medicine & Rehabilitation

Sacramento, California, United States

Rady Children's Hospital center - UCSD Department of Neuroscience

San Diego, California, United States

Connecticut Children's Medical Center, Neurology Division

Hartford, Connecticut, United States

Child Health Research Institute

Gainesville, Florida, United States

MD Rare Disease Research, LLC

Atlanta, Georgia, United States

University of Iowa Children's Hospital

Iowa City, Iowa, United States

Washington University School of Medicine in St Louis Department of Neurology 660 S.Euclid Avenue, Campus Box 8111

St Louis, Missouri, United States

Shriners Hospitals for Children

Portland, Oregon, United States

The Children's Hospital of Philadelphia Colket Translational Research Building

Philadelphia, Pennsylvania, United States

Virginia Commonwealth University Childrens Hospital of Richmond at

Richmond, Virginia, United States

University Hospitals Leuven, Neuromuscular Reference Centre, Child Neurology

Leuven, Belgium

Hospital de La Citadelle, Centre de Référence des Maladies Neuromuscolaires (CRMN)

Liège, Belgium

Kinsmen Research Centre - Alberta Children's Hospital

Calgary, Alberta, Canada

The University of British Columbia, Children's and Womens Health Centre of BC Branch

Vancouver, British Columbia, Canada

Holland Bloorview Kids Rehabilitation Hospital

Toronto, Ontario, Canada

CHU de Nantes - Hotel-Dieu - Hopital Nord Laennec, rez-de-chausse haut ail Ouest

Nantes, France

Hôpital Armand Trousseau I-Motion - Plateforme d'essais cliniques pédiatriques Bâtiment Lemariey - Porte 20 * 2ème étage 26 Avenue du Dr Arnold Nette

Paris, France

Universitätsklinikum Essen - Kinder-und Jugendmedizin Neuropadiatrie

Essen, Germany

Klinik- und Poliklinik fur Kinder- und Jugendmedizin, Universitatsklinikum HamburgEppendorf, Martinistr. 52

Hamburg, Germany

Klinikum der Universitat Munchen, Campus Innenstadt, Lindwurmstr. 4

München, Germany

Institute of Neurology - Schneider Children's Medical Center of Israel Kaplan, 14

Petah Tikva, Israel

U.O.S.D. Centro Traslazionale di Miologia e Patologie Neurodegenerative, Building 16 - ground floor IRCCS Istituto Giannina Gaslini,

Genova, Italy

A.O.U. Policlinico G. Martino, U.O.C. Neurologia e Malattie Neuromuscolari

Messina, Italy

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano, UOS di Neurologia Pediatrica

Milan, Italy

IRCCS Istituto Neurologico Carlo Besta

Milan, Italy

Centro Clinico NeMO Fondazione Serena ONLUS Area SUD

Milan, Italy

Ospedale Pediatrico Bambino Gesù, Malattie Neuromuscolari e Neurodegenerative

Roma, Italy

Fondazione Policlinico Universitario "A.Gemelli", UOC Neuropsichiatria Infantile

Roma, Italy

Leiden University Medical Center LUMC, Albinusdreef 2

Leiden, Netherlands

Radboud University Medical Centre

Nijmegen, Netherlands

Clinic of Neurology and Psychiatry for Children and Youth - Neurology Department Dr. Subotic 6a,

Belgrade, Serbia

Neuromuscular Pathology Unit - Hospital Sant Joan de Déu

Esplugues de Llobregat, Barcellona, Spain

Hospital Materno-Infantil

Barcelona, Spain

Hospital Universitario Virgen del Rocio

Seville, Spain

Hospital Universitari i Politècnic La Fe - Servicio Neurologia

Valencia, Spain

Alder Hey Children's Hospital NHS Trust

Liverpool, UK, United Kingdom

The Robert Jones and Agnes Hunt Orthopaedic Hospital NHS Foundation Trust

Gobowen, United Kingdom

UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre and MRC Centre for NMD

London, United Kingdom

The John Walton Muscular Dystrophy Research Centre

Newcastle upon Tyne, United Kingdom

View Full Details on ClinicalTrials.gov

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NCT03373968

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