CAP-1002 for Duchenne Muscular Dystrophy

(HOPE-2-OLE Trial)

Not currently recruiting at 5 trial locations

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: Capricor Inc.

Disqualifiers: Major surgery, Respiratory disease, Hypersensitivity, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 21

Duration: 60 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called deramiocel (CAP-1002) for Duchenne Muscular Dystrophy, a condition that progressively weakens muscles. The goal is to determine if administering this treatment every three months is safe and effective. Participants from the previous HOPE-2 study who completed the 12-month follow-up may qualify for this trial. Participants will receive 20 infusions in an outpatient setting, spaced over 60 months. As a Phase 2 trial, the research focuses on assessing the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that deramiocel, also known as CAP-1002, is safe to use. Studies have found that people with Duchenne muscular dystrophy tolerate the treatment well. No new safety issues have emerged, and it remains safe over the long term.

For instance, in one study where patients used deramiocel for three years, it proved safe and helped improve heart and muscle health. Another study lasting four years confirmed these benefits and found no new safety problems. These findings suggest that deramiocel is safe for people, especially those with Duchenne muscular dystrophy. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Duchenne Muscular Dystrophy?

Unlike the standard treatments for Duchenne Muscular Dystrophy, which often include corticosteroids and gene therapy, CAP-1002 offers a unique approach by utilizing cardiosphere-derived cells (CDCs). These cells have the potential to repair and regenerate damaged heart and skeletal muscle tissues, which is a novel mechanism of action compared to traditional therapies. Researchers are excited about CAP-1002 because it is administered through intravenous infusions, potentially providing systemic benefits and improving muscle function without the side effects commonly associated with other treatments.

What evidence suggests that CAP-1002 might be an effective treatment for Duchenne Muscular Dystrophy?

Research has shown that deramiocel (CAP-1002), the treatment under study in this trial, can help slow Duchenne Muscular Dystrophy (DMD). In earlier studies, patients who received deramiocel experienced a 52% reduction in disease progression over three years. After four years of treatment, their condition changed very little, with an average change of just -0.5 points from their starting point. This suggests that deramiocel might help maintain muscle function over time. The treatment also improved heart muscle function, a common concern in DMD. These findings offer hope that deramiocel could effectively manage DMD symptoms in the long term. ^{1 2 4 5 6}

Who Is on the Research Team?

Craig McDonald, MD

Principal Investigator

UC Davis

Mark Awadalla

Principal Investigator

Capricor Inc.

Are You a Good Fit for This Trial?

This trial is for those who were in the HOPE-2 study, finished a year of follow-up, and can give consent. They must have good veins for infusions and be able to stick to the trial plan. People with planned major surgery, recent investigational drug use, substance abuse issues, or severe respiratory problems can't join.

Inclusion Criteria

Documented enrollment in the HOPE-2 trial and completion of trial follow-up through Month 12

I am willing and able to agree to participate in the trial, and if under 18, I have parental or guardian consent.

I am willing and able to follow the trial's requirements.

Exclusion Criteria

I have a chronic lung condition not related to muscular dystrophy.

History, or current use, of drugs or alcohol that could impair ability to comply with participation in the trial

Treatment with an investigational product ≤ 6 months prior to first infusion

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

4 weeks

1 visit (in-person)

Treatment

Participants receive 20 intravenous infusions of deramiocel, each separated by three months

60 months

20 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 months

What Are the Treatments Tested in This Trial?

Interventions

• CAP-1002

Trial Overview The safety and effectiveness of CAP-1002 are being tested through sixteen intravenous doses given every three months over two years. Participants will have regular check-ups before each dose to monitor their response to the treatment.

How Is the Trial Designed?

Group I: Deramiocel Experimental Treatment 1 Intervention

Participants will receive an intravenous (IV) infusion of deramiocel (150 million Cardiosphere-Derived Cells (CDCs) per infusion) every 3 months

Who Is Running the Clinical Trial?

Capricor Inc.

Lead Sponsor

Trials

12

Recruited

480+

Published Research Related to This Trial

Daily treatment with SMT022357, a second-generation compound, significantly increases utrophin expression in various muscle types, which helps stabilize muscle fibers and prevent damage associated with Duchenne muscular dystrophy (DMD).

The study in mdx mice shows that SMT022357 not only reduces muscle degeneration but also enhances overall muscle function, indicating its potential as a disease-modifying therapy for all DMD patients, regardless of their specific dystrophin mutation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Second-generation compound for the modulation of utrophin in the therapy of DMD. Guiraud, S., Squire, SE., Edwards, B., et al.[2022]

Edasalonexent, a small molecule designed to inhibit the NF-κB pathway, was well tolerated in a 1-week study involving 17 boys aged 4 to 8 with Duchenne muscular dystrophy, with no serious adverse events reported.

The treatment led to a decrease in NF-κB-regulated genes and serum proteins, indicating its potential efficacy in reducing muscle damage associated with the disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Phase 1 Study of Edasalonexent (CAT-1004), an Oral NF-κB Inhibitor, in Pediatric Patients with Duchenne Muscular Dystrophy. Finanger, E., Vandenborne, K., Finkel, RS., et al.[2020]

Citations

1. capricor.com capricor.com/investors/news-events/press-releases/detail/317/capricor-therapeutics-announces-positive-4-year-data-from

Capricor Therapeutics Announces Positive 4-Year Data ... After four years of continuous treatment, Deramiocel-treated patients showed a median change of -0.5 points compared to baseline.

2. clinicaltrials.gov clinicaltrials.gov/study/NCT05126758

A Study of Deramiocel (CAP-1002) in Ambulatory and Non- ... Secondary endpoints evaluated at the 12-month time point include assessment of changes in cardiac muscle function and structure by cardiac magnetic resonance ...

3. capricor.com capricor.com/investors/news-events/press-releases/detail/308/capricor-therapeutics-announces-positive-data-demonstrating

Capricor Therapeutics Announces Positive Data ... --Preservation of Skeletal Muscle Function Shown Over 3 Years Resulting in 52% Slowing of Disease--. --Data Presented at the 2025 Muscular ...

4. neurologylive.com neurologylive.com/view/long-term-4-year-data-highlights-sustained-effects-deramiocel-dmd-cardiomyopathy

Long-Term 4-Year Data Highlights Sustained Effects of ... Capricor Therapeutics Announces Positive 4-Year Data from HOPE-2 Open-Label Extension Study of Deramiocel in Duchenne Muscular Dystrophy.

5. parentprojectmd.org parentprojectmd.org/clinical-trial/a-study-of-deramiocel-cap-1002-in-ambulatory-and-non-ambulatory-patients-with-duchenne-muscular-dystrophy/

A Study of Deramiocel (CAP-1002) in Ambulatory and Non- ... A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (MIS51ON). Active, Not Recruiting. 4 Years ...

6. capricor.com capricor.com/investors/news-events/press-releases/detail/294/capricor-therapeutics-announces-positive-long-term-data

Capricor Therapeutics Announces Positive Long-Term ... The results of this study showed sustained cardiac and skeletal benefits after 3 years of continuous treatment with deramiocel.