Assessing cognitive and developmental needs in boys with Duchenne Muscular Dystrophy
Assessment of Neurodevelopmental Needs in Duchenne Muscular Dystrophy
This study is trying to understand the thinking and development needs of boys with Duchenne Muscular Dystrophy by having them do tasks on an iPad and share their experiences over 18 months.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 90 (estimated) |
| Ages | 3 Years and up |
| Sex | All |
| Sponsor | Virginia Commonwealth University Academic / other |
| Locations | 1 site (Richmond, Virginia) |
| Trial ID | NCT05280730 on ClinicalTrials.gov |
What this trial studies
This observational study aims to evaluate the neurodevelopmental needs of boys with Duchenne Muscular Dystrophy (DMD) using the National Institutes of Health Toolbox Cognition Battery (NIHTB-CB). Participants will complete cognitive tasks on an iPad at multiple time points over 18 months, while also undergoing brief physical assessments. Additionally, families will have the opportunity to participate in focus groups to discuss their experiences with cognitive and developmental challenges. Optional brain MRI scans will be conducted to investigate changes in brain connectivity over time.
Who should consider this trial
Good fit: Ideal candidates include boys aged 3 and older with a confirmed genetic mutation in the dystrophin gene or clinical features of DMD, as well as symptomatic carrier girls.
Not a fit: Patients who are unable to use an iPad due to physical limitations or have metal implants that preclude MRI participation may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the cognitive and developmental challenges faced by boys with DMD, potentially informing future treatment approaches.
How similar studies have performed: While there have been studies focusing on cognitive assessments in DMD, this specific approach utilizing the NIHTB-CB and MRI is relatively novel.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Boys with confirmed genetic mutation in the dystrophin gene * Boys with clinical features of DMD and in whom muscle biopsy showed absence of dystrophin * Boys with clinical features of DMD and in whom there is a family history of DMD * Symptomatic carrier girls with DMD * Ages 3 and above at time of study screening Exclusion Criteria: * Care-giver unable to give consent * Any handicap that does not allow the ability to use an IPAD * For MRI, braces or any metal implants.
Where this trial is running
Richmond, Virginia
- Children's Hospital of Richmond — Richmond, Virginia, United States (Recruiting)
Study contacts
- Principal investigator: Mathula Thangarajh, MD, PhD — Virginia Commonwealth University
- Study coordinator: Matthew Ridder, BS
- Email: Matthew.Ridder@vcuhealth.org
- Phone: 804-828-5269
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.