Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Then 6 Years Old

Sponsor
Italfarmaco
Study ID
NCT06769633
Phase
PHASE2
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
MALE
Age
2 Years - 6 Years
Healthy Volunteers
Not accepted

Interventions

Study Details

This is a Phase 2 Open-label (Core Phase Plus Extension Phase) With 2 Cohorts Study to Assess the Pharmacokinetics and Safety of Givinostat in younger DMD Patients. * Planned screening duration: approximately 4 weeks * Planned Core Treatment duration: approximately 48 weeks * Planned Extension Treatment duration: approximately 96 weeks * Planned Follow Up duration: approximately 4 weeks (± 7 days) * Total duration of study participation: up to 151 weeks (ie, 37-38 months)

Key Dates

First listed
Jan 10, 2025
Start date
Jan 2, 2025
Status verified
Jul 2025
Primary completion
Dec 31, 2029
Completion
Dec 31, 2029

Study Design

Enrollment
18 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Cohort 1 - from 4 to 6 years old
    Drug: Givinostat 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. Other Names: \- ITF2357
  • Experimental: Cohort 2 - from 2 to 4 years old
    Drug: Givinostat Givinostat has to be administered twice daily in a fed state according to a flexible dose regimen based on patient weight. Starting dose will be confirmed/adjusted with results of the interim analysis of cohort 1. Other Names: \- ITF2357

Primary Outcome Measure

Core Phase: Cohort 1 - Change from baseline of the pharmacokinetic (PK) parameter Area under the concentration-time curve from dosing (time 0) to time t at steady state (AUC0-T,ss). [ Time Frame: Baseline up to week 48 ]

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