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

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 ]

Central Contacts

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