Full results & analysis
Available in the Gosset platform where reported.
Trial intelligence / Profile preview
Givinostat PK and Safety in Young DMD Patients
NCT06769633Phase 2
Key facts
- Status
- Recruiting
- Start
- Jan 2025
- Primary completion
- Dec 2029
- Countries
- 4
- Sponsor
- Italfarmaco
- Modality
- Covalent Small Molecules, Classical Binding Small Molecules
- Drugs
- givinostat
- Conditions
- Duchenne muscular dystrophy (DMD)
01
Overview
Phase 2, open-label trial of givinostat in Duchenne muscular dystrophy (DMD) (2 cohorts), sponsored by Italfarmaco. Primary endpoints: Area under the concentration-time curve from dosing to time t at steady state at Week 48, Maximum plasma concentration at steady state at Week 48 and 2 more. Started Jan 2025; status: recruiting.
02
Design
2 arms- Study design
- Non-randomizedOpen-label
Cohort 1 (4-6 years)
Patients aged 4 to <6 years receiving Givinostat.
Cohort 2 (2-4 years)
Patients aged 2 to <4 years receiving Givinostat.
03
Who it enrols
Male children aged 2 to <6 years with Duchenne Muscular Dystrophy. Must be on stable corticosteroids or no corticosteroids (cannot start during Core Phase). Excludes those with recent exposure to other investigational drugs, dystrophin restoration products, or gene therapy.
04
Endpoints
Primary
Area under the concentration-time curve from dosing to time t at steady state
Maximum plasma concentration at steady state
Elimination half-life
Type, incidence, and severity of treatment-emergent adverse events (Extension Phase)
Secondary
Type, incidence, and severity of treatment-emergent adverse events (Core Phase)
North Star Ambulatory Assessment
Bayley Scales of Infant and Toddler Development, Third Edition (Gross Motor)
Health-Related Quality of Life
Beyond the preview
Go deeper on NCT06769633.
Full results, competitive context and analysis are in the Gosset platform.
Competitive context
Available in the Gosset platform where reported.
Bring the full picture into focus.
See how Gosset can support your research on NCT06769633.