Interviews with patients and caregivers about Duchenne Muscular Dystrophy

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 observational study aims to gather qualitative insights from individuals with Duchenne Muscular Dystrophy (DMD) and their caregivers regarding functional abilities, independence, and quality of life. Participants will engage in semi-structured interviews lasting approximately 60 minutes, focusing on their experiences and perspectives. The study does not involve any treatment or intervention but includes participants currently receiving a drug called givinostat, which is approved in some regions and under investigation elsewhere.

Why it matters

Who can join: the full rules

Inclusion Criteria:

Subsample A - Patients

Eligible participants for Subsample A must:

* Have been treated with Givinostat for at least 2 years
* Be at least 10 years of age with signed consent of a parent or legal guardian
* Be currently taking givinostat
* Willing and able to participate in a video and/or audio recorded interview

Subsample A - Caregivers

Eligible caregivers for Subsample A must:

* Be a parent or legal guardian of an individual with DMD that has been taking givinostat for at least two years
* Willing and able to participate in a video and/or audio recorded interview

Subsample B - Caregivers

Eligible caregivers for Subsample B must:

* Provide care to an individual with DMD who is unable to raise their hands above their head as confirmed by the caregiver
* Reside in the United States or Canada
* Able to read, speak, and understand English
* Willing and able to participate in a video and/or audio recorded interview Have access to a stable internet connection

Exclusion Criteria:

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Where it's running

• Red Nucleus
  Yardley, Pennsylvania, United States Recruiting

Study contacts

• Principal investigator: Mindy Leffler, M.Ed. — Red Nucleus Solutions
• Study coordinator: Mary Hubbard
• Email: mhubbard@rednucleus.com
• Phone: 904-541-9326

Similar studies

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

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• Bone health in adults with Duchenne muscular dystrophy: osteoporosis, fractures, and treatment

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• Respiratory progression in adults with Duchenne muscular dystrophy: a long-term observational study

  This study tests how respiratory function changes over three years in adults with genetically confirmed Duchenne muscular dystrophy.

  Recruiting at 13 sites Istituto Giannina Gaslini · Bologna and 12 other locations · NCT07879989

• Modeling heart failure in Duchenne and Becker muscular dystrophy to find surrogate heart measures

  This study will look at heart disease in people with Duchenne/Becker muscular dystrophy and carriers to see if MRI-based heart measures can predict death or major heart problems.

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• Duchenne/Becker Muscular Dystrophy EHR Data Linkage

  This study tests whether consented patients’ electronic health records can be linked with the Duchenne Registry data to create a richer view of Duchenne and Becker muscular dystrophy care.

  Recruiting at 11 sites The Duchenne Registry · Little Rock, Arkansas and 10 other locations · NCT07609394

• Zeleciment Rostudirsen (DYNE-251) for Duchenne Muscular Dystrophy in Ambulatory Boys

  This trial tests whether intravenous Zeleciment Rostudirsen (DYNE-251) given every 4 weeks is safe and helps improve function in ambulatory boys with Duchenne muscular dystrophy who have a specific dystrophin gene mutation.

  Recruiting Phase 3 Dyne Therapeutics · Hillsborough, North Carolina · NCT07608432

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.