CAP-1002 for Duchenne Muscular Dystrophy

(HOPE-2-OLE Trial)

Recruiting at 4 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

13 Participants Needed

Trial Summary

What is the purpose of this trial?

This Phase 2, multi-center, open-label extension trial will provide deramiocel (CAP-1002) to subjects that were enrolled in the HOPE-2 trial and completed 12 months of follow-up. The trial will explore the safety and efficacy of twenty intravenous administrations of deramiocel, each separated by three months. Subjects will undergo a targeted screening during a 30-day screening period, eligible subjects will then undergo baseline safety and efficacy assessments on Day 1 prior to their first infusion of deramiocel. Subjects will complete trial assessments at Screening; Day 1; Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48, 51, 54, 57, and 60. Safety and efficacy assessments will be conducted prior to deramiocel administration at the Day 1, Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48, 51, 54, and 57 trial visits, unless otherwise indicated. All deramiocel infusions will be conducted in an outpatient setting at the investigative site on Day 1 and Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48, 51, 54, and 57. Subjects will be observed in the outpatient setting for at least two hours post infusion and then discharged the same day, if medically cleared by the site Investigator.

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.

How is the drug CAP-1002 different from other treatments for Duchenne Muscular Dystrophy?

CAP-1002 (Deramiocel) is unique because it involves the use of cardiac-derived cells, which may help repair and regenerate damaged muscle tissue, unlike other treatments that focus on modulating proteins like utrophin or inhibiting inflammation pathways. ^{1 2 3 4 5}

Research Team

Craig McDonald, MD

Principal Investigator

UC Davis

Mark Awadalla

Principal Investigator

Capricor Inc.

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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 for a total of 20 IV infusions

Who Is Running the Clinical Trial?

Capricor Inc.

Lead Sponsor

Trials

12

Recruited

480+

Findings from Research

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]

References

1. England pubmed.ncbi.nlm.nih.gov

Second-generation compound for the modulation of utrophin in the therapy of DMD. [2022]

2. England pubmed.ncbi.nlm.nih.gov

Safety, tolerability, and pharmacokinetics of SMT C1100, a 2-arylbenzoxazole utrophin modulator, following single- and multiple-dose administration to healthy male adult volunteers. [2022]

3. United States pubmed.ncbi.nlm.nih.gov

Identifying FDA-Approved Drugs that Upregulate Utrophin A as a Therapeutic Strategy for Duchenne Muscular Dystrophy. [2022]

4. Netherlands pubmed.ncbi.nlm.nih.gov

Phase 1 Study of Edasalonexent (CAT-1004), an Oral NF-κB Inhibitor, in Pediatric Patients with Duchenne Muscular Dystrophy. [2020]

5. United States pubmed.ncbi.nlm.nih.gov

Safety, Tolerability, and Pharmacokinetics of SMT C1100, a 2-Arylbenzoxazole Utrophin Modulator, following Single- and Multiple-Dose Administration to Pediatric Patients with Duchenne Muscular Dystrophy. [2021]