Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy

Sponsor
Italfarmaco
Study ID
NCT05933057
Phase
PHASE3
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
MALE
Age
9 Years - 17 Years
Healthy Volunteers
Not accepted

Interventions

  • Givinostat — DRUG
    Givinostat has to be administered twice daily in a fed state according to a flexible dose regimen based on patient weight. Starting dose could be reduced based on predefined safety rules.
  • Placebo — DRUG
    Placebo, manufactured to mimic givinostat, has to be administered twice daily in a fed state according to a flexible dose regimen based on patient weight. Starting dose could be reduced based on predefined safety rules.

Study Details

This is a randomised, double-blind, placebo-controlled, multicentre study to evaluate the efficacy, safety, and tolerability of givinostat in non-ambulant male paediatric (aged 9 to \<18 years) patients with DMD. 138 patients will be randomised 2:1 to givinostat or placebo and will be treated for 18 months. * Planned screening duration: approximately 4 weeks (±14 days) * Planned treatment duration: 18 months (approximately 72 weeks) * Planned follow-up duration: 4 weeks (±7 days) (for patients not participating in the long-term safety study) * Total duration of study participation: up to 83 weeks (ie, 20-21 months)

Key Dates

Start date
Feb 19, 2024
Status verified
May 2025
Primary completion
Feb 29, 2028
Completion
Feb 29, 2028

Study Design

Enrollment
138 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Givinostat
    Patients will receive concomitant corticosteroid treatment as part of the standard of care.
  • Placebo Comparator: Placebo
    Patients will receive concomitant corticosteroid treatment as part of the standard of care.

Primary Outcome Measure

Change of Performance of Upper Limb 2.0 (PUL) total score at 18 months of treatment of givinostat compared to placebo group. [ Time Frame: Baseline and 18 months ]

Central Contacts

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