Cell Therapy for Duchenne Muscular Dystrophy

(HOPE-3 Trial)

Not currently recruiting at 20 trial locations

Overseen By Han Phan, MD

Age: Any Age

Sex: Male

Trial Phase: Phase 3

Sponsor: Capricor Inc.

Must be taking: Glucocorticoids

Must not be taking: Metformin, Insulin, Exon skipping

Disqualifiers: Low LVEF, High BMI, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: 14

Duration: 12 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new cell therapy called deramiocel (CAP-1002) to determine if it can improve muscle function in boys and young men with Duchenne Muscular Dystrophy (DMD). Participants will receive either the treatment or a placebo, with both groups later having the opportunity to try the actual treatment. The study seeks participants with DMD who experience muscle function difficulties, regardless of their ability to walk. It aims to ensure the safety and effectiveness of deramiocel before potential widespread availability. As a Phase 3 trial, this study represents the final step before FDA approval, allowing participants to contribute to potentially groundbreaking treatment advancements.

Do I have to stop taking my current medications for the trial?

The trial protocol does not specify if you must stop taking your current medications. However, you must have been on a stable dose of systemic glucocorticoids for at least 6 months before the study. Some medications, like metformin, insulin, and certain exon skipping therapies, should not have been started recently. It's best to discuss your specific medications with the trial team.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but it does require that you have been on a stable dose of systemic glucocorticoids for at least 6 months before joining. If you are taking certain medications like metformin, insulin, or human growth hormone, you may need to have been on a stable dose for a specific period before participating.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that deramiocel (CAP-1002) is generally safe for people. In past studies, this treatment was well tolerated, and no new safety issues emerged. Recipients of deramiocel did not experience any unexpected harmful effects.

Long-term studies have also shown positive results. After four years, participants continued to benefit from deramiocel without any new safety concerns. This suggests that the treatment may be safe for long-term use in people with Duchenne muscular dystrophy.

Overall, deramiocel has a good safety record based on the available data. Participants in earlier studies handled the treatment well, which is important for those considering joining a trial. ^{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 focus on managing symptoms with steroids or physical therapy, CAP-1002 offers a new approach by using cardiosphere-derived cells (CDCs). These cells are administered via intravenous infusion and are designed to potentially repair and regenerate muscle tissue, targeting the underlying cause of muscle deterioration. Researchers are excited about CAP-1002 because it taps into the regenerative power of CDCs, offering hope for not just managing symptoms but possibly slowing the progression of the disease itself.

What evidence suggests that this trial's treatments could be effective for Duchenne muscular dystrophy?

Research has shown that deramiocel (CAP-1002), which participants in this trial may receive, may help treat Duchenne muscular dystrophy (DMD). One study found that patients who received deramiocel experienced a 52% slower decline in muscle function over three years. After four years, these patients maintained almost stable muscle function, with only a small average change. This evidence suggests that deramiocel could help maintain muscle function in DMD patients and slow the disease's progression. ^{2 3 4 5 6}

Who Is on the Research Team?

Craig McDonald

Principal Investigator

University of California, Davis

Mark Awadalla

Principal Investigator

Capricor Inc.

Are You a Good Fit for This Trial?

This trial is for boys and young men at least 10 years old with genetically confirmed Duchenne muscular dystrophy (DMD). They must have been on stable glucocorticoids for a year, have up-to-date immunizations, and adequate venous access. Ambulatory participants should take more than 10 seconds to walk/run 10 meters; non-ambulatory ones should have lost independent walking between ages 10-18.

Inclusion Criteria

I have good veins for IV treatments and blood tests.

I am using effective birth control methods if I'm sexually active.

I lost the ability to walk on my own between ages 10 and 18.

Exclusion Criteria

Inability to perform consistent PUL 2.0 measurement within specific ranges during paired testing at screening.

Treatment with an investigational product within 6 months prior to randomization.

I cannot fully straighten my arms due to stiffness.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Treatment

Participants receive either deramiocel or placebo every 3 months for a total of 4 doses during the first 12 months

12 months

Visits at Baseline/Day 1, Month 1, and Months 3, 6, 9, and 12

Open-label extension

All participants receive 4 doses of deramiocel for an additional 12 months

12 months

Visits at Month 12, 15, 18, and 21

Long-term open-label extension

Participants continue to receive deramiocel every 3 months until commercial availability or trial termination

Indefinite

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• CAP-1002
• Placebo

Trial Overview The HOPE-3 trial tests CAP-1002 cell therapy's effectiveness in improving muscle function in DMD patients. Participants are randomly assigned to receive either CAP-1002 or placebo every three months over a year, followed by an open-label extension where all get CAP-1002.

How Is the Trial Designed?

Group I: Deramiocel (CAP-1002) Experimental Treatment 1 Intervention

Cohort A: Approximatetly 29 subjects will receive deramiocel (CAP-1002A) active treatment consisting of 150 million cardiosphere-derived cells (CDCs) via intravenous infusion every 3 months Cohort B: Approximately 22 participants will receive deramiocel (CAP-1002B) active treatment consisting of 150 million cardiosphere-derived cells (CDCs) via intravenous infusion every 3 months

Group II: Placebo Placebo Group 1 Intervention

Cohort A: Approximately 29 subjects will receive a Placebo solution via intravenous infusion every 3 months Cohort B: Approximately 22 participants will receive a Placebo solution via intravenous infusion every 3 months

Who Is Running the Clinical Trial?

Capricor Inc.

Lead Sponsor

Trials

12

Recruited

480+

Published Research Related to This Trial

Transplantation of dystrophin-expressing chimeric (DEC) cells derived from myoblasts significantly increased dystrophin expression in the skeletal, cardiac, and respiratory muscles of a mouse model for Duchenne muscular dystrophy (DMD), leading to improved muscle function and reduced pathology.

The study demonstrated that DEC transplantation not only preserved heart function and respiratory capacity but also improved muscle strength and reduced inflammation and fibrosis, suggesting a promising new therapeutic approach for enhancing the overall health of DMD patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Human dystrophin expressing chimeric (DEC) cell therapy ameliorates cardiac, respiratory, and skeletal muscle's function in Duchenne muscular dystrophy. Siemionow, M., Langa, P., Harasymczuk, M., et al.[2022]

A new immunodeficient rat model of Duchenne muscular dystrophy (DMD) was developed, showing similar disease characteristics to human patients, which is crucial for studying potential treatments.

Successful engraftment of human myoblasts in this rat model indicates its potential for preclinical studies on cell transplantation therapies for DMD, paving the way for future research in this area.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A new immunodeficient Duchenne muscular dystrophy rat model to evaluate engraftment after human cell transplantation. Sato, M., Goto, M., Yamanouchi, K., et al.[2023]

The DT-DEC01 cell therapy demonstrated a strong safety profile with no adverse events reported up to 21 months after administration, indicating it is a safe treatment option for patients with Duchenne Muscular Dystrophy (DMD).

Functional improvements were observed in patients, including better performance in the 6-Minute Walk Test and other assessments, suggesting that DT-DEC01 may effectively enhance muscle function and overall health in DMD patients over a 12-month period.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and Efficacy of DT-DEC01 Therapy in Duchenne Muscular Dystrophy Patients: A 12 - Month Follow-Up Study After Systemic Intraosseous Administration. Siemionow, M., Biegański, G., Niezgoda, A., et al.[2023]

In a 7-month phase I clinical trial involving eight boys with Duchenne muscular dystrophy, the autologous transplantation of muscle-derived CD133+ stem cells was found to be safe, with no local or systemic side effects reported.

The treatment led to positive changes in muscle structure, including an increased ratio of capillaries to muscle fibers and a shift towards faster myosin-positive muscle fibers, suggesting potential benefits for muscle repair.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Autologous transplantation of muscle-derived CD133+ stem cells in Duchenne muscle patients. Torrente, Y., Belicchi, M., Marchesi, C., et al.[2019]

The first-in-human study of DEC cell therapy for Duchenne Muscular Dystrophy (DMD) showed no adverse events in the first 3 patients over 14 months, indicating a strong safety profile for this novel treatment.

Patients receiving DEC therapy demonstrated significant improvements in muscle function and strength, as measured by various functional tests, suggesting that this therapy could effectively enhance quality of life for DMD patients without the need for immunosuppression.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Dystrophin Expressing Chimeric (DEC) Cell Therapy for Duchenne Muscular Dystrophy: A First-in-Human Study with Minimum 6 Months Follow-up. Heydemann, A., Bieganski, G., Wachowiak, J., et al.[2023]

The novel DT-DEC01 therapy, which uses Dystrophin Expressing Chimeric (DEC) cells, was found to be safe in a small study of 3 boys aged 6-15 years, with no therapy-related adverse events reported up to 22 months after treatment.

Preliminary efficacy was observed through electromyography (EMG) assessments, showing increased motor unit potentials in treated muscles at 12 months, indicating potential benefits for muscle function in boys with Duchenne muscular dystrophy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Assessment of Motor Unit Potentials Duration as the Biomarker of DT-DEC01 Cell Therapy Efficacy in Duchenne Muscular Dystrophy Patients up to 12 Months After Systemic-Intraosseous Administration. Niezgoda, A., Biegański, G., Wachowiak, J., et al.[2023]

The study demonstrated that human dystrophin expressing chimeric (DEC) cell therapy is safe for long-term use, showing no complications or DNA damage in a mouse model of Duchenne muscular dystrophy (DMD) after 180 days post-administration.

Biodistribution analysis confirmed that DEC cells effectively targeted the heart, diaphragm, and gastrocnemius muscle while showing minimal presence in non-target organs, indicating a promising therapeutic approach for DMD patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-Term Biodistribution and Safety of Human Dystrophin Expressing Chimeric Cell Therapy After Systemic-Intraosseous Administration to Duchenne Muscular Dystrophy Model. Siemionow, M., Brodowska, S., Langa, P., et al.[2022]

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.