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Clinical Trial

Offered by: Nemours Children's
Location: Orlando, Fla.

Trial Name

A Phase 2, Randomized, Double-Blind, Placebo Trial: Halt myopathy progression in Duchenne (HOPE-2)

What is the trial about?

This study will evaluate the safety and effectiveness of repeat intravenous (IV) infusions of CAP-1002 in children with Duchenne Muscular Dystrophy (DMD) who have reduced muscle function. This study will evaluate the safety and effectiveness of repeat intravenous (IV) infusions of CAP-1002 in children with Duchenne Muscular Dystrophy (DMD) who have reduced muscle function.

Who can participate?

A child that has been diagnosed with Duchenne Muscular Dystrophy and is at least 10 to 18 years of age currently treated, for at least 12 months, with a glucocorticoid.

What is involved?

CAP-1002 is an investigational, cell-based product called “cardiosphere-derived cells”. “Investigational” means that CAP-1002 has only been approved for research purposes. CAP-1002 (also known as the ‘study drug’) has been shown to decrease inflammation, block or prevent tissue scarring, and stimulate new tissue growth. CAP-1002 is made from the cells of donated healthy human hearts and grown in a laboratory. This study will compare the safety, muscle function, and quality of life of participants who receive repeat IV infusions of CAP-1002 to participants who receive repeat IV infusions of a placebo. A placebo is an inactive substance that looks like the active study drug and will be used for comparison in this research study.

Participation in this study will last for about 13 months (between screening and treatment) and includes 6-7 visits to Nemours Children’s Hospital, Florida. There are also six blood draws between visits that can either be completed at Nemours Children’s Hospital or at a Quest Patient Service Center closer to where you live. There will be four phone calls during the study, one after each infusion.

Contact Nemours Children's Clinical Trials

Trial Name: A Phase 2, Randomized, Double-Blind, Placebo Trial: Halt myopathy progression in Duchenne (HOPE-2)

IRB#: 1186162

(800) 354-5690 Email Us

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