NCT06769633

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

A Phase 2 Open-label (Core Phase Plus Extension Phase) With 2 Cohorts Study to Assess the Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Than 6 Years Old

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 18 (estimated)
• Sponsor: Italfarmaco · Industry
• Sex: Male
• Age: 2 Years – 6 Years
• Healthy volunteers: Not accepted

Summary

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)

Detailed description

This is an open-label, multicentre, multicountry, 2 cohorts study to evaluate the PK profile and safety of givinostat in subjects with DMD aged ≥4 to \<6 years for Cohort 1 and aged ≥2 to \<4 years for Cohort 2. The starting dose for Cohort 2 will be confirmed/adjusted with results of the interim analysis of Cohort 1. The study will consist of 2 phases, a Core Phase and an Extension Phase. Two final analyses will be conducted, the first at the end of the Core Phase and the second at the end of the Extension Phase (core and extension data will be combined). The study will enrol approximately 18 subjects (approximately 9 subjects \[aged ≥4 to \<6 years\] in Cohort 1 and approximately 9 subjects \[aged ≥2 to \<4 years\] in Cohort 2).

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	Givinostat Hydrochloride	Cohort 1 - from 4 to 6 years old
DRUG	Givinostat Hydrochloride - Cohort 2	Cohort 2 - from 2 to 4 years old

Timeline

• Start date: 2025-01-02
• Primary completion: 2029-12-01
• Completion: 2029-12-01
• First posted: 2025-01-10
• Last updated: 2025-07-10

Locations

9 sites across 4 countries: Belgium, Italy, Netherlands, United Kingdom

Regulatory

• FDA-regulated drug study