A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

NCT05126758 | Active Not Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: CAP-1002-DMD-04
• Study Type: interventional
• Target: 106
• Locations: 1 country
• Sites: 20

Brief Summary

HOPE-3 is a two cohort, Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating the efficacy and safety of a cell therapy called deramiocel (CAP-1002) in study participants with Duchenne muscular dystrophy (DMD) and impaired skeletal muscle function. Non-ambulatory and ambulatory boys and young men who meet eligibility criteria will be randomly assigned to receive either deramiocel or placebo every 3 months for a total of 4 doses during the first 12 months of the study. All participants will be eligible to receive 4 doses of deramiocel for an additional 12 months as part of an open-label extended assessment period. After completion of the first open-label extension (Months 12-24), subjects who have completed Month 24 are eligible to continue onto a Long-Term Open-Label Extension period that will provide treatment with deramiocel until commercial availability, or until sponsor's decision to terminate the trial, or the participant withdraws consent.

Conditions

Muscular Dystrophies; Nervous System Diseases; Muscular Dystrophy, Duchenne; Muscular Disorders, Atrophic; Muscular Diseases; Neuromuscular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn

Keywords

Duchenne Muscular Dystrophy; Cell Therapy; Performance of the Upper Limb; Ambulatory; Non-Ambulatory

Outcome Measures

Primary Outcomes (1)

• Change in the upper limb function

  Mean percent change from baseline in Performance of the Upper Limb test, version 2 (PUL 2.0) Total Score. Items are scored on a three-point scale: 0=unable to perform the item, 1=impaired or performs with compensation, 2=performs task without compensation. Percent change from baseline is calculated as change from baseline divided by baseline score at the subject level.

  At Month 12

Secondary Outcomes (10)

• Change in cardiac muscle function and structure by assessment of left ventricular ejection fraction

  At Month 12

• Change in mid-level (elbow) upper limb function

  At Month 12

• Change in Global Statistical Test (Total GST) combining upper limb function, cardiac muscle function, and patient reported measure of disease severity

  At Month 12

• Change in the number of segments of myocardial scarring (Cohort B only)

  at Month 12

• Change in cardiac muscle function and structure by assessment of left ventricular end-systolic volume

  At Month 12

Study Arms (2)

Deramiocel (CAP-1002)

EXPERIMENTAL

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

Biological: Deramiocel (CAP-1002)

Placebo

PLACEBO COMPARATOR

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

Biological: Placebo

Interventions

Deramiocel (CAP-1002) BIOLOGICAL

Cohort A: CAP-1002A manufactured in Los Angeles, CA; Cohort B: CAP-1002B manufactured in San Diego, CA

Also known as: Cardiosphere-Derived Cells (CDCs)

Deramiocel (CAP-1002)

Placebo BIOLOGICAL

Placebo

Placebo

Eligibility Criteria

• Age: 10 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Male subjects at least 10 years of age at time of consent who are willing and able to provide informed consent to participate in the trial if ≥ 18 years of age or assent with parental or guardian informed consent if \< 18 years of age. If a third-party caregiver is involved, they must provide informed consent.
• Diagnosis of DMD based on clinical and phenotypic manifestations consistent with DMD (e.g., family history of DMD, elevated creatine kinase, dystrophin muscle biopsy, calf pseudohypertrophy, history of Gowers' sign, and gait impairment before 7 years of age) as confirmed by the Investigator.
• Confirmatory genetic testing performed to have reached a diagnosis of DMD at any time in the past or currently performed at a Clinical Laboratory Improvement Amendments (CLIA)-certified laboratory or equivalent.
• Performance of the Upper Limb test (PUL) entry item scores 2-6 and total PUL score less than or equal to 40. For Cohort A only: enrollment of patients with PUL entry score 6, Exon 44 skipping amenable, and/or Exon 3 through 7 deletions will be capped at no more than 10% of the total study population (approximately 6 patients with these characteristics).
• Reduced ability to walk/run (if ambulatory): subjects must take more than 10 seconds for the 10-meter walk/run (i.e., velocity \< 1 meter/second).
• If non-ambulatory, loss of independent ambulation between 10th and 18th year birthday (standing unassisted or ability to take, at most, several steps independently is not considered ambulation). Subjects who are considered non-ambulatory between the ages of 9 and10 may be enrolled with prior approval from the sponsor.
• Receiving standard of care therapy at an experienced, multidisciplinary DMD center as evidenced by regular cardiac and pulmonary monitoring, systemic glucocorticoid treatment, and at-home range of motion exercises.
• Treatment with systemic glucocorticoids for at least 12 months and at a stable dose at least 6 months prior to study participation, except for either weight-based dose adjustment or a decrease in steroid dose of ≤ 10% for toxicity. For patients on chronic deflazacort, treatment with an equivalent dose of prednisone or prednisolone for a period of ≤ 30 days to bridge lack of availability of deflazacort during the 6 months prior to randomization is acceptable.
• Current and up-to-date immunizations according to children and adolescent Centers for Disease Control and Prevention immunization schedule at the discretion of the Investigator.
• Adequate venous access for parenteral IP infusions and routine blood collection.
• Assessed by the Investigator as willing and able to comply with the requirements of the trial.
• Sexually active subjects and their partners who are fertile must agree to use effective method(s) of contraception.

You may not qualify if:

• Left ventricular ejection fraction (LVEF) less than or equal to 35% prior to randomization.
• Elbow-flexion contractures \> 30° in both extremities.
• Body mass index (BMI) \> 45.
• Percent predicted forced vital capacity (FVC%) \< 35% within 6 months prior to randomization.
• Inability to perform consistent PUL 2.0 measurement within ± 2 points without shoulder domain or within ± 3 points with shoulder domain during paired testing at screening.
• Risk of near-term respiratory decompensation in the judgment of the Investigator, or the need for initiation of day and night non-invasive ventilator support as defined by serum bicarbonate ≥ 29 mmol/L at screening.
• History of non DMD-related chronic respiratory disease requiring ongoing or intermittent treatment, including, but not limited to, asthma, bronchitis, and tuberculosis.
• Acute respiratory illness within 30 days prior to screening and during screening.
• Initiation of nocturnal non-invasive ventilation within 30 days prior to screening.
• Planned or anticipated thoracic or spinal surgery within the 6 months following randomization.
• Planned or anticipated lower extremity surgery within the 6 months following randomization, if ambulatory.
• Known hypersensitivity to dimethyl sulfoxide (DMSO) or bovine products.
• Initiation of treatment with metformin or insulin within 3 months prior to randomization.
• Initiation of treatment with an FDA-approved exon skipping therapy for the treatment of DMD and/or non-weight based adjustments within 12 months prior to randomization.
• Treatment with human growth hormone within 3 months prior to randomization, unless on a stable dose allowing for weight-based dose adjustments (as determined by the site Investigator) for at least 24 months prior to randomization.

Sponsors & Collaborators

• Capricor Inc.: lead

Study Sites (20)

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

Location

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

Location

UCSD Altman Clinical and Translational Research Institute

La Jolla, California, 92037, United States

Location

Children's Hospital of Los Angeles, Division of Neurology

Los Angeles, California, 90027, United States

Location

University of California, Davis

Sacramento, California, 95817, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Rare Disease Research, LLC

Atlanta, Georgia, 30329, United States

Location

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

University of Iowa Hospitals and Clinics

Iowa City, Iowa, 52242, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

University of Missouri Health Care

Columbia, Missouri, 65212, United States

Location

Saint Louis Children's Hospital

St Louis, Missouri, 63110, United States

Location

Rare Disease Research NC LLC

Hillsborough, North Carolina, 27278, United States

Location

Akron Children's Hospital

Akron, Ohio, 44308, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Children's Health Specialty Care Pavilion

Dallas, Texas, 75207, United States

Location

University of Utah Hospital

Salt Lake City, Utah, 84112, United States

Location

University of Virginia Children's Hospital

Charlottesville, Virginia, 22903, United States

Location

Seattle Children's

Seattle, Washington, 98105, United States

Location

Children's Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

MeSH Terms

Conditions

Muscular Dystrophies; Muscular Dystrophy, Duchenne; Muscular Disorders, Atrophic; Muscular Diseases; Neuromuscular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Nervous System Diseases

Condition Hierarchy (Ancestors)

Musculoskeletal Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Craig McDonald, MD

  University of California, Davis

  PRINCIPAL INVESTIGATOR

• Mark Awadalla

  Capricor Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Cohort A: placebo-controlled, double-blind, randomized 1:1 (active:placebo); Cohort B: placebo-controlled, double-blind, randomized 1:1 (active:placebo)

Study Record Dates

• First Submitted: November 4, 2021
• First Posted: November 19, 2021
• Study Start: June 22, 2022
• Primary Completion: June 18, 2025
• Study Completion (Estimated): December 1, 2026
• Last Updated: December 17, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (20)