Researchers at Capricor Therapeutics are looking for ambulatory and non-ambulatory children and adults with Duchenne muscular dystrophy (DMD) to participate in a Phase 2 study. The goal of the study is to evaluate the safety and efficacy of CAP-1002. Researchers hope that this therapy may slow disease progression in heart and skeletal muscle by modulating immunologic activity, reducing inflammation and fibrosis, and stimulating regeneration. Participants will be randomized to receive either CAP-1002 or placebo. At the end of the trial, an open-label extension will offer CAP-1002 to those who received placebo if the safety profile is acceptable.
Total study duration for each patient will be about one year, with nine clinic visits (screening, baseline/day one, weeks four and six, and months three, four-and-a-half, six, nine and 12). At each visit, efficacy will be evaluated by the performance of the upper limb (PUL), pulmonary function testing, the North Star Ambulatory Assessment (ambulatory subjects only), strength testing, cardiac MRI and quality of life. Safety tests will include adverse events, a physical exam, vital signs, a 12-lead ECG and clinical laboratory testing. CAP-1002 or placebo will be delivered through an IV infusion during visits at day one and months three, six and nine. Throughout the study, participants will continue to see their regular doctor for routine care.
In order to be eligible to participate, boys (older than 10 years old) and adult males must, along with meeting additional study criteria, have:
- Reduced upper arm strength
- Reduced ability to walk/run (if ambulatory)
- Treatment with systemic glucocorticoids for at least 12 months and at a stable dose at least six months prior to study participation, except for weight-based or toxicity-related adjustments
Travel support for the participant and one travel companion for each visit is available.
To learn more or to inquire about participation, contact Brian Fedor at 310-358-3201 or HOPEfirstname.lastname@example.org.
To find out more information about the study, including a list of active trial sites, you may also visit https://clinicaltrials.gov/ct2/show/NCT03406780 or the Capricor website at http://capricor.com/ or http://hope2trial.com/.