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
This trial does not accept healthy volunteersQualifiers
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
Design model
Single group
Treatments tested in this trial
Givinostat
Drugsuspension of givinostat (10 mg/mL)
Treatment groups
206 Participants
are divided into 1 treatment groupGroup A: givinostatExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Type, incidence, and severity of treatment related/not related adverse events(AEs) and serious adverse event (SAEs)
Time frame
Through study completion, an average of 1 yearSponsors and contacts
Click on the lead sponsor to view all of their trials.
Italfarmaco
Lead sponsor
Cromsource
Collaborator
Source ClinicalTrials.gov