179
Participants
Start Date
June 6, 2017
Primary Completion Date
February 22, 2022
Study Completion Date
February 22, 2022
givinostat
"The oral suspension of givinostat (10 mg/mL) was to be dosed in fed condition as described below:~Givinostat or placebo starting dose~* \> or =10 and \< 12.5 kg of weight: 13.3 mg bid = 1.3 ml oral suspension bid~* \> or =12.5 and \< 20 kg: 16.7 mg bid =1.7 ml oral suspension bid~* \> or = 20 and \< 25 kg: 20 mg bid = 2.0 ml oral suspension bid~* \> or = 25 and \< 30 kg: 23.3 mg bid = 2.3 ml oral suspension bid~* \> or = 30 and \< 40 kg: 26.7 mg bid = 2.7 ml oral suspension bid~* \> or = 40 and \< 50 kg: 33.3 mg bid = 3.3 ml oral suspension bid~* \> or = 50 and \< 60 kg: 36.7 mg bid = 3.7 ml oral suspension bid~* \> or = 60 and \< 70 kg: 40 mg bid = 4 ml oral suspension bid~* \> or = 70 kg: 46.7 mg bid = 4.7 ml oral suspension bid"
placebo
The oral suspension of placebo, manufactured to mimic givinostat, was to be dosed in fed condition as described for givinostat.
Radboud University Medical Centre, Nijmegen
Clinic of Neurology and Psychiatry for Children and Youth - Neurology Department Dr. Subotic 6a,, Belgrade
IRCCS Istituto G.Gaslini, U.O.S.D. Centro Traslazionale di Miologia e Patologie Neurodegenerative, Genova
The Children's Hospital of Philadelphia Colket Translational Research Building, Philadelphia
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano, UOS di Neurologia Pediatrica, Milan
Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan
Centro Clinico NeMO Fondazione Serena ONLUS Area SUD, Milan
Klinik un Policlinik fur Kinder und Jugendmedizin - Universitatsklinikum Hamburg Eppendorf, Hamburg
Virginia Commonwealth University Childrens Hospital of Richmond at Virginia Commonwealth University, Richmond
MD Rare Disease Research, LLC, Atlanta
Child Health Research Institute - Department of Pediatrics, Gainesville
Nemours Children's Hospital, Orlando
Hospital Universitario Virgen del Rocio, Seville
CHU de Nantes - Hotel-Dieu - Hopital Nord Laennec, rez-de-chausse haut ail Ouest, Nantes
Universitatsklinikum Essen - Kinder und Jugendmedizin Neuropadiatrie, Essen
Hospital Universitari i Politécnic de la Fe - Servicio Neurologia, Valencia
University of Iowa Children's Hospital, Iowa City
University of Minnesota - Department of Neurology, Minneapolis
Washington University School of Medicine in St Louis - Department of Neurology, St Louis
Hopital Armand Trousseau I-Motion, Plateforme d'essais cliniques pédiatriques, Paris
Children's Hospital Colorado, Aurora
Klinikum der Uniersitat Munchen - Campus Innenstadt, München
Rady Children's Hospital center - UCSD Department of Neuroscience, San Diego
Neuromuscular Research Center UC Davis Department of Physical Medicine and Rehabilitation, Davis
Shriners Hospitals for Children, Portland
A.O.U. Policlinico G. Martino, U.O.C. Neurologia e Malattie Neuromuscolari, Messina
Institute of Neurology - Schneider Children's Medical Center of Israel Kaplan, 14, Petah Tikva
Connecticut Children's Medical Center - Division Neurology, Hartford
University Hospitals Leuven, Neuromuscular Reference Centre, Child Neurology, Leuven
Hospital de La Citadelle, Centre de Référence des Maladies Neuromuscolaires (CRMN), Liège
Kinsmen Research Centre - Alberta Children's Hospital - Alberta Health Services, Calgary
The University of British Columbia, Children's and Womens Health Centre of BC Branch, Vancouver
Holland Bloorview Kids Rehabilitation Hospital, Toronto
Ospedale Pediatrico Bambin Gesù, Malattie Neuromuscolari e Neurodegenerative, Roma
"Fondazione Policlinico Universitario A.Gemelli, UOC Neuropsichiatria Infantile", Roma
Leiden University Medical Center LUMC, Leiden
Hospital Sant Joan de Déu - Neuromuscular Pathology Unit, Esplugues de Llobregat
Hospital Materno-Infantil - Passeig de la Vall d'Hebron, Barcelona
Alder Hey Children's Hospital NHS Trust, Liverpool
UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre and MRC Centre for NMD, London
The John Walton Muscular Dystrophy Research Centre - Freeman Hospital - Newcastle University - Institute of Genetic Medicine, Newcastle upon Tyne
The Robert Jones and Agnes Hunt Orthopaedic Hospital - NHS Foundation Trust, Oswestry
Lead Sponsor
Collaborators (1)
Syneos Health
OTHER
Italfarmaco
INDUSTRY