Real-world experience of oral givinostat for Duchenne muscular dystrophy in the U.S.

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a prospective, observational U.S. study that follows patients with genetically confirmed Duchenne muscular dystrophy who are newly starting oral givinostat or who began treatment within the prior six months. No treatments are provided by the study; data are collected from routine clinical visits and medical records over a planned minimum follow-up of two years and up to five years for early enrollees. The primary focus is on safety, including monitoring for thrombocytopenia and serious bleeding events, along with other adverse events, gastrointestinal effects, and functional outcomes. The FDA requires this post‑marketing study to better characterize the incidence, frequency, and severity of these safety events in real-world practice.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Patients of any gender at least 6 years of age, diagnosed with DMD (according to genetic test) and have received a prescription for oral givinostat in accordance with the USPI.
* Patient has provided informed consent (and assent when applicable) for participation in the study.
* Patient's index date (first date of givinostat treatment) is no more than 6 months prior to signing of informed consent.
* Patient has the required data available (DMD diagnosis, givinostat administration \[dose and schedule\], laboratory results \[hematology and triglycerides\]) for addressing the study objectives for the period between index date and study entry (for patients enrolled after the index date).

Exclusion Criteria:

* Patient previously received givinostat (commercial or investigational product) and permanently discontinued treatment or patient started commercial givinostat for \> 6 months before signing of informed consent.

Where it's running

• Arkansas Children's Research Institute
  Little Rock, Arkansas, United States Recruiting
• Children's National Hospital
  Washington D.C., District of Columbia, United States Recruiting
• Ann and Robert H. Lurie Children's Hospital of Chicago
  Chicago, Illinois, United States Recruiting
• Boston Children's Hospital
  Boston, Massachusetts, United States Recruiting
• University of Massachusetts Chan Medical School
  Worcester, Massachusetts, United States Recruiting
• Columbia University Medical Center
  New York, New York, United States Recruiting

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• GNT0004 gene therapy for Duchenne muscular dystrophy in ambulant boys

  This study tests a one-time IV gene therapy (GNT0004) to see if it slows DMD progression in ambulant boys aged 6–10.

  Recruiting at 10 sites Phase 2/3 Genethon · Brussels and 13 other locations · NCT07856329

• AOC 1044 in Duchenne Muscular Dystrophy with Exon 44 Skipping

  This study tests whether intravenous AOC 1044 helps boys with Duchenne muscular dystrophy caused by mutations amenable to exon 44 skipping.

  Recruiting at 20 sites Phase 3 Avidity Biosciences, Inc. · Westmead, New South Wales and 19 other locations · NCT07587242

• Ultrafast ultrasound biomarkers to track Duchenne and Becker muscular dystrophy

  This study tests whether multiparametric ultrafast ultrasound can non-invasively monitor disease progression in people with Duchenne or Becker muscular dystrophy.

  Recruiting Nantes University Hospital · Nantes · NCT07378553

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.