Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study
ConditionDuchenne Muscular Dystrophy
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 groupSponsors and collaborators
Italfarmaco
Lead sponsor
Cromsource
Collaborator
Source ClinicalTrials.gov