Capricor Therapeutics put a voluntary hold on a Phase 2 clinical trial of its Duchenne muscular dystrophy (DMD) investigative cell therapy CAP-1002 after a patient experienced a severe allergic reaction during blind testing.
According to a recent U.S. Securities and Exchange Commission report, Capricor halted its HOPE-2 (NCT03406780) study, pending results of an ongoing safety review. Following the adverse reaction, the patient responded well to treatment and has no symptoms, the filing said.
The clinical-stage biotechnology company also said it is working with the U.S. Food and Drug Administration (FDA) on a mitigation plan.
HOPE-2 is a randomized, placebo-controlled clinical trial, testing repeat intravenous doses of CAP-1002. The study was enrolling up to 84 males — ages 10 or older — at 14 sites nationwide. Participants randomly received intravenous doses of CAP-1002 or a placebo every three months over the course of a year.
Study recruitment began early last year. Participants are in advanced stages of Duchenne, and are largely ineligible for many of the other clinical trials being conducted for Duchenne, Capricor said in a November press release.
Pre-clinical and clinical investigations had demonstrated that a single administration of CAP-1002 into the heart improved cardiac and skeletal muscle function in Duchenne patients and mice models, and also reduced tissue scarring. Enhancements in upper limb and finger movements are vital for day-to-day tasks.
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The CAP-1002 therapy is comprised of cardiosphere-derived cells (CDCs), which contain cardiac progenitor cells capable of generating mature heart cells. CDCs are a type of heart progenitor cells that are known to have regenerative, immune system-regulating, anti-inflammatory, and anti-scarring elements. The therapy foments cellular regeneration by altering immune system function.
Capricor announced it would meet with the FDA to discuss aspects of the clinical trial, including the design, goals and manufacturing processes, in the November release. That meeting, set for December, resulted from the FDA’s earlier designations of CAP-1002 as a regenerative medicine advanced therapy (RMAT) and rare pediatric disease therapy.
The RMAT designation is granted to potential regenerative therapies designed to treat a serious condition, and which have shown the potential to address the condition’s unmet needs. In addition to orphan drug status, the designations put the therapy on a faster track for approval and delivery.
Pending resolution of safety concerns and the trial’s resumption, patients may be enrolled in an extension study in which all will be given CAP-1002.
Primarily affecting males, DMD is a genetic disorder characterized by progressive muscle degeneration and weakness. It’s caused by mutations of the gene that encodes the protein dystrophin, the loss of which leads to significant wasting in both skeletal and heart muscles. Skeletal muscle directs voluntary body movements. By late childhood, patients can no longer walk. By their teens, they develop enlarged hearts, a condition that often leads to heart failure.