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

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 (7)

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