A phase 3 clinical trial testing a new experimental treatment for Duchenne muscular dystrophy (DMD) started dosing its first participant, according to Capricor Therapeutics, the developers of the treatment.

The randomized, double-blind, placebo-controlled trial called HOPE-3 is investigating the safety and efficacy of a cell therapy called CAP-1002 in 68 DMD patients, aged 10 or more years who can or cannot walk. Participants are randomly assigned to either receive CAP-1002 or a placebo every 3 months for a total of 4 doses. 

The primary outcome measure of the study is any change in full upper limb function. Secondary outcome measures include any changes in cardiac muscle function and structure and other upper limb functions.


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Data from a previous phase 2 study suggested that the experimental treatment can slow the loss of upper limb function by as much as 70%.

Read more about the etiology of DMD.

“We are delighted to begin dosing patients in HOPE-3,” said Capricor chief executive officer Linda Marbán, PhD. “Beginning this clinical trial is a significant milestone, not only for Capricor, but most importantly for those boys and young men with DMD.”

The trial started in May 2022 and is still recruiting participants in Arkansas, California, and Georgia. It is estimated to be completed in December 2025.

CAP-1002 is made of allogeneic cardiosphere-derived cells, a type of progenitor cell which have a differentiated mechanism of action. They can modulate the immune system to drive cellular regeneration. Importantly, they are suitable for DMD patients with any type of mutation.

DMD is caused by a mutation in the DMD gene, which results in the body not being able to produce any functional dystrophin protein. Dystrophin is essential for muscle health and in its absence muscle cells degenerate, over time causing muscle weakness and wasting.

References

Capricor Therapeutics announces first patient dosed in pivotal phase 3 study of CAP-1002 for the treatment of Duchenne muscular dystrophy. News release. Capricor Therapeutics; July 19, 2022. 

A study of CAP-1002 in ambulatory and non-ambulatory patients with Duchenne muscular dystrophy (HOPE-3). US National Library of Medicine. Last updated May 20, 2022. Accessed July 22, 2022.