A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy

Conditions

• Duchene Muscular Dystrophy

Eligibility Criteria

Sex

ALL

Age

6 Years - N/A

Healthy Volunteers

Not accepted

Study Details

This is a prospective observational study conducted to evaluate safety, tolerability, and functional outcomes of patients with DMD newly initiating oral givinostat or having started therapy within 6 months as part of routine clinical care in the US. The study has a planned maximum duration of 5 years for the first enrolled patients, including a 24-month enrollment period and a minimum of 2 years of follow-up.

Key Dates

First listed

Aug 17, 2025

Start date

Oct 23, 2025

Status verified

Apr 2026

Primary completion

Jun 30, 2030

Completion

Jul 30, 2030

Study Design

Enrollment

300 participants (estimated)

Arms

• Arm: Ambulatory
  Ambulatory is defined as being able to complete the 10-meter Walk-Run (10mWR) test within 30 seconds without an assistive device. This cohort will include up to 120 patients who are ambulatory prior to starting givinostat treatment. Within this cohort , 95 patients must have had prior use or a referral for concomitant use of dystrophin-enhancing therapy (DET), such as exon skipping oligonucleotides or gene therapy. Enrollment is capped at 25 patients without prior or referred DET use.
• Arm: Non-Ambulatory
  Non-ambulatory status is determined by inability to perform the 10mWR test as defined above. This cohort will include approximately 180 patients who are non-ambulatory prior to starting givinostat.

Primary Outcome Measure

Characterize the incidence of thrombocytopenia / decreased platelet counts in DMD patients treated with oral givinostat [ Time Frame: From follow up (after first date of givinostat treatment) through end of study (up to 5 years) ]

Locations (9)

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