Join a Study to Determine the Efficacy and Safety of CAP-1002 in Treating Duchenne Muscular Dystrophy (DMD)
Patients with Duchenne Muscular Dystrophy can participate in a study for safety and efficacy of investigational therapy. See if you qualify!
| Age: | 10 and older |
| Sex: |
Male
|
| Healthy Volunteers: | No |
| Type: | Phase 3 Interventional Study |
| Target: |
82 Participants
|
or |
| United States | Akron Children's Hospital Akron, OH, USAUnited States | Signup at this site | |
| United States | Arkansas Children's Hospital Little Rock, AR, USAUnited States | Signup at this site | |
| United States | Boston Children's Hospital Boston, MA 02115United States | ||
| United States | Children's Hospital Colorado Aurora, CO, USAUnited States | Signup at this site | |
| United States | Children's Hospital of Los Angeles Los Angeles, CA 90027United States | ||
| United States | Cincinnati Children's Hospital Medical Center Cincinnati, Ohio 45229United States | ||
| United States | Department of Neurology Boston, MA, USAUnited States | Signup at this site | |
| United States | Division: Developmental Pediatrics Charlottesville, VA, USAUnited States | Signup at this site | |
| United States | Lurie Children's Hospital of Chicago Chicago, Illinois 60611United States | ||
| United States | Neuromuscular Disease & ALS Center Setauket- East Setauket, NY, USAUnited States | Signup at this site | |
| United States | Rare Disease Atlanta, GA, USAUnited States | Signup at this site | |
| United States | Saint Louis Children's Hospital St. Louis, MO 63110United States | ||
| United States | Seattle Children's Neurology Seattle, WA, USAUnited States | Signup at this site | |
| United States | The Neurological Institute, Division of Neurology Los Angeles, CA, USAUnited States | Signup at this site | |
| United States | UC Davis Health Physical Medicine & Rehabilitation Sacramento, CA, USAUnited States | Signup at this site | |
| United States | UC San Diego Health Neurological Institute San Diego, CA, USAUnited States | Signup at this site | |
| United States | University of Iowa, Department of Neurology Iowa City, IA, USAUnited States | Signup at this site | |
| United States | University of Missouri Columbia, MO, USAUnited States | Signup at this site | |
| United States | University of Utah Salt Lake City, UT 84132United States | ||
| United States | UT Southwestern Dallas, Texas 75235United States | ||
| United States | Wisconsin Children's Wauwatosa, WI, USAUnited States | Signup at this site |
Description
DMD is a genetic disorder that causes muscles to weaken and waste away over time. This happens because of changes in a protein called dystrophin, which helps keep muscle cells healthy.
Although treatment discoveries over the past decade have improved the quality of life of those with the disease, there is still an urgent need for medication that will work to reduce or reverse the advancement of DMD.
This study hopes to determine how safe and effective CAP-1002 is in the treatment of DMD. CAP-1002 is an investigational drug that has not been approved by the Food and Drug Administration.
This study requires
The study may last for about 25 months and will require up to 11 visits to the study center.
- You will have a screening visit to see if you are eligible.
- If you are eligible to participate you will be randomized to receive CAP-1002 or placebo (dummy drug) every 3 months for a total of 4 intravenous infusions.
- After the first 12 months, all patients will receive 4 additional infusions over a 12-month extended period of CAP-1002 (meaning that even if you receive placebo for the first 4 infusions you will then receive active drug for the last 4 infusions).
- Study visits will include blood tests, completing questionnaires, physical examinations, MRI scans (of your heart), assessment of vital signs (like blood pressure), measurement of height, weight, and arm length, and physical activity tests to assess muscle function.
Who can participate
- Age 10 and above
- Patients with DMD and impaired skeletal muscle function
Benefits and risks of participating
BENEFITSWe cannot promise you any benefits as a result of participation in this study, but your participation may help us learn more about treatment options for the disease and may be of benefit to people with DMD in the future.
RISKS
Participation in any research study carries some risk. Your study doctor will go over the risks associated with participating in this research study.
Compensation
You will be eligible for financial support for travel expenses.
Resources
Links
Study ID
ClinicalTrials.gov: NCT05126758
Schedule
Study duration and period
25 months with about 11 visits in total
Sending
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