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

NCT05933057 · Status: RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 138

Last updated 2025-05-11

No results posted yet for this study

Summary

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)

Conditions

Interventions

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.

DRUG

Placebo

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.

Sponsors & Collaborators

  • Fortrea

    collaborator INDUSTRY

  • Italfarmaco

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
9 Years
Max Age
17 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-02-19
Primary Completion
2028-02-29
Completion
2028-02-29

Countries

  • Belgium
  • Canada
  • France
  • Germany
  • Italy
  • Netherlands
  • United Kingdom

Study Locations

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Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05933057 on ClinicalTrials.gov