Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study
Start Date
10/24/2017
Completion Date
12/1/2029
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.
Detailed Description
GIVINOSTAT oral suspension (10 mg/mL) has to be administered orally as 2 oral doses daily while the subject is in a fed state. The starting dose of GIVINOSTAT in the present long term study will be the same that the subject was receiving at the end of the previous DMD GIVINOSTAT study. As weight affects GIVINOSTAT exposures, the dosage will be modified based on subject weight according the rules detailed in the study protocol section 11.2.2.1. In addition, in case a subject will have a consistent (e.g., at least 2 consecutive evaluations) platelets count ≤150 x 10\^9/L and not meet the stopping criteria for platelets, the Investigator will have to reduce the dose of 1/3 or 20% less of the current dose as described in the study protocol section 10.5.1.3. During the first month of treatment, platelets count assessment will be performed weekly, while during the second month it will be performed every 2 weeks, in order to strictly monitor this parameter for safety reasons, with the exclusion of subjects coming from study DSC/11/2357/43 for which the first visit will be 4 months after the Visit 1/baseline visit. Study drug should be permanently interrupted if any of the following occurs: * severe drug-related diarrhoea (i.e., increase of ≥7 stools per day); * any drug-related SAE; * QTcF \>500 msec; * platelets count ≤50 x 10\^9/L; * white blood cells ≤2.0 x 10\^9/L; * hemoglobin ≤8.0 g/dL; To avoid laboratory errors and anomalous values, test must be confirmed with a repeated test performed on the next working day. The treatment should be stopped until the retest result becomes available. If the repeated test is still under the stopping limit value, study drug must be permanently discontinued. If the repeated test is acceptable, the subject can resume treatment. The Investigator will follow up the patient until resolution or acceptable stabilization of the event occurs and document all the relevant information, as applicable. After the resolution/stabilization of the event, the subject will be withdrawn from the study and the EOS Visit (see Section 12.1.10) will be performed. Any decision relevant to the dose adjustment and/or modification of schedule of assessments can be discussed with the Medical Monitor, but the final decision remains with the Investigator only or its authorized designee.
Eligibility Criteria
Age Range: 7 years to No maximum
Interventions
Givinostat
Conditions
Locations
University of California - Davis Medical Center - Devis Physical Medicine & Rehabilitation
Sacramento, California 95817
United States
Rady Children's Hospital center - UCSD Department of Neuroscience
San Diego, California 92123
United States
Connecticut Children's Medical Center, Neurology Division
Hartford, Connecticut 06106
United States
Child Health Research Institute
Gainesville, Florida 32610
United States
MD Rare Disease Research, LLC
Atlanta, Georgia 30318
United States
University of Iowa Children's Hospital
Iowa City, Iowa 52242
United States
Washington University School of Medicine in St Louis Department of Neurology 660 S.Euclid Avenue, Campus Box 8111
St Louis, Missouri 63110
United States
Shriners Hospitals for Children
Portland, Oregon 97239
United States
The Children's Hospital of Philadelphia Colket Translational Research Building
Philadelphia, Pennsylvania 19104
United States
Virginia Commonwealth University Childrens Hospital of Richmond at
Richmond, Virginia 23298
United States
University Hospitals Leuven, Neuromuscular Reference Centre, Child Neurology
Leuven, 03000
Belgium
Hospital de La Citadelle, Centre de Référence des Maladies Neuromuscolaires (CRMN)
Liège, 04000
Belgium
Kinsmen Research Centre - Alberta Children's Hospital
Calgary, Alberta T3B 6A8
Canada
The University of British Columbia, Children's and Womens Health Centre of BC Branch
Vancouver, British Columbia V6H 3V4
Canada
Holland Bloorview Kids Rehabilitation Hospital
Toronto, Ontario M4G1R8
Canada
CHU de Nantes - Hotel-Dieu - Hopital Nord Laennec, rez-de-chausse haut ail Ouest
Nantes, 44093
France
Hôpital Armand Trousseau I-Motion - Plateforme d'essais cliniques pédiatriques Bâtiment Lemariey - Porte 20 * 2ème étage 26 Avenue du Dr Arnold Nette
Paris, 760
France
Universitätsklinikum Essen - Kinder-und Jugendmedizin Neuropadiatrie
Essen, D-45147
Germany
Klinik- und Poliklinik fur Kinder- und Jugendmedizin, Universitatsklinikum HamburgEppendorf, Martinistr. 52
Hamburg, 20246
Germany
Klinikum der Universitat Munchen, Campus Innenstadt, Lindwurmstr. 4
München, 80337
Germany
Institute of Neurology - Schneider Children's Medical Center of Israel Kaplan, 14
Petah Tikva, 4920235
Israel
U.O.S.D. Centro Traslazionale di Miologia e Patologie Neurodegenerative, Building 16 - ground floor IRCCS Istituto Giannina Gaslini,
Genova, 16147
Italy
A.O.U. Policlinico G. Martino, U.O.C. Neurologia e Malattie Neuromuscolari
Messina, 98125
Italy
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano, UOS di Neurologia Pediatrica
Milan, 20122
Italy
IRCCS Istituto Neurologico Carlo Besta
Milan, 20133
Italy
Centro Clinico NeMO Fondazione Serena ONLUS Area SUD
Milan, 20162
Italy
Ospedale Pediatrico Bambino Gesù, Malattie Neuromuscolari e Neurodegenerative
Roma, 00146
Italy
Fondazione Policlinico Universitario "A.Gemelli", UOC Neuropsichiatria Infantile
Roma, 00168
Italy
Leiden University Medical Center LUMC, Albinusdreef 2
Leiden, 2300
Netherlands
Radboud University Medical Centre
Nijmegen, 6500
Netherlands
Clinic of Neurology and Psychiatry for Children and Youth - Neurology Department Dr. Subotic 6a,
Belgrade, 11000
Serbia
Neuromuscular Pathology Unit - Hospital Sant Joan de Déu
Esplugues de Llobregat, Barcellona 08950
Spain
Hospital Materno-Infantil
Barcelona, 08035
Spain
Hospital Universitario Virgen del Rocio
Seville, 41013
Spain
Hospital Universitari i Politècnic La Fe - Servicio Neurologia
Valencia,
Spain
Alder Hey Children's Hospital NHS Trust
Liverpool, UK L12 2AP
United Kingdom
The Robert Jones and Agnes Hunt Orthopaedic Hospital NHS Foundation Trust
Gobowen, SY10 7AG
United Kingdom
UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre and MRC Centre for NMD
London, , WC1N 1EH
United Kingdom
The John Walton Muscular Dystrophy Research Centre
Newcastle upon Tyne, NE1 3BZ
United Kingdom