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

Trial statusRecruiting
Trial phasePhase 2, Phase 3
Trial typeInterventional
Biological sexMale
Age7+
SponsorItalfarmaco

About this trial

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 criteria

Qualifiers

all the inclusion criteria and none of the

Disqualifiers

had a baseline vastus lateralis muscle fat fraction (VL MFF) assessed by MRS in the range ≤5% or >30%, i.e. included in"off-target" group,

never been randomized because, the enrollment in the off target group was completed.

Aged ≥6 years old;

Are able to give informed assent and/or consent in writing signed by the subject and/or parent/legal guardian (according to localregulations);

Trial design

Treatments tested in this trial

  • Givinostat

Treatment groups

206 Participants
are divided into 1 treatment group

Locations

39
Belgium
University Hospitals Leuven, Neuromuscular Reference Centre, Child Neurology03000, Leuven Belgium
Hospital de La Citadelle, Centre de Référence des Maladies Neuromuscolaires (CRMN)04000, Liège Belgium
Canada
Kinsmen Research Centre - Alberta Children's HospitalT3B 6A8, CalgaryAlberta, Canada
The University of British Columbia, Children's and Womens Health Centre of BC BranchV6H 3V4, VancouverBritish Columbia, Canada

Sponsors and collaborators

Italfarmaco

Lead sponsor

Cromsource

Collaborator