Open-label Extension of the HOPE-2 Trial

NCT04428476 | Active Not Recruiting Phase 2 Results DMC FDA Drug

• 1 other identifier: CAP-1002-DMD-02-OLE
• Study Type: interventional
• Target: 13
• Locations: 1 country
• Sites: 5

Brief Summary

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, over a period of approximately 60 months. Following completion of the initial open-label phase (Month 60), subjects who have completed all Month 60 assessments will be eligible to continue into a long-term open label extension (LT-OLE) period and can continue to receive deramiocel once every 3 months until deramiocel is commercially available or the sponsor terminates the study, or the subject withdraws consent or study participation is terminated by the sponsor. 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, 60, and all LT-OLE visits. 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, 57, and at all LT-OLE 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, 57, with continued dosing in the LT-OLE visits. 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.

Conditions

Duchenne Muscular Dystrophy

Keywords

DMD, Muscular dystrophy, Duchenne

Outcome Measures

Primary Outcomes (3)

• Number of Participants With Treatment-Emergent Adverse Events (TEAEs) From Baseline Through Month 12

  Adverse event (AE) is defined as any unfavourable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of study drug, whether or not considered related to the study drug. Serious AE: an AE that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial or prolonged inpatient hospitalization; congenital anomaly/birth defect or was otherwise considered medically important. TEAE: AE with onset after start of treatment or with onset date before the treatment start date but worsening after the treatment start date. TEAEs included both serious and non-serious TEAEs.

  Baseline up to Month 12

• Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Severity From Baseline Through Month 12

  Severity of adverse events (AE) were assessed by the investigator as Grade 1 = Mild (Transient or mild discomfort; no limitation in activity; no medical intervention/therapy required), Grade 2 = Moderate (Mild to moderate limitation in activity - some assistance may be needed; no or minimal medical intervention/therapy required), Grade 3 = Severe (Marked limitation in activity, some assistance usually required; medical intervention/therapy required and often requiring hospitalization or prolongation of hospitalization), Grade 4 = Life-threatening (Extreme limitation in activity, significant assistance required; significant medical intervention/therapy required; hospitalization, prolongation of hospitalization, or hospice care) and Grade 5 = Death.

  Baseline up to Month 12

• Change From Baseline in Functional Capacity as Assessed by Performance of the Upper Limb Test, Version 2 (PUL 2.0) Total Score.

  PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function.

  Baseline, Month 12

Secondary Outcomes (12)

• Number of Participants With Treatment-Emergent Adverse Events (TEAEs) at Month 24, Month 36, Month 48 and Month 60

  Month 24, Month 36, Month 48 and Month 60

• Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Severity From Baseline Through Month 60

  Baseline up to Month 60

• Change From Baseline in Upper Limb Function as Assessed by Performance of the Upper Limb Test, Version 2 (PUL 2.0) at Month 12, Month 24, Month 36, Month 48, and Month 60

  Baseline, Month 12, Month 24, Month 36, Month 48, and Month 60

• Change From Baseline in Distal-Level (Wrist and Hand) Upper Limb Function as Assessed by Performance of the Upper Limb Test, Version 2 (PUL 2.0) at Month 12, Month 24, Month 36, Month 48, and Month 60

  Baseline, Month 12, Month 24, Month 36, Month 48, and Month 60

• Change From Baseline in Mid-Level (Elbow) as Assessed by Performance of the Upper Limb Test, Version 2 (PUL 2.0) at Month 12, Month 24, Month 36, Month 48, and Month 60

  Baseline, Month 12, Month 24, Month 36, Month 48, and Month 60

Study Arms (1)

Deramiocel

OTHER

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

Biological: Deramiocel (CAP-1002)

Interventions

Deramiocel (CAP-1002) BIOLOGICAL

Peripheral infusion of 150 million allogeneic cardiosphere-derived cells administered every three months

Also known as: Allogeneic Cardiosphere-Derived Cells

Deramiocel

Eligibility Criteria

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

You may qualify if:

• Documented enrollment in the HOPE-2 trial and completion of trial follow-up through Month 12
• Willing and able to provide informed consent to participate in the trial if ≥ 18 years of age, and assent with parental or guardian informed consent if \< 18 years of age
• Adequate venous access for intravenous deramiocel (CAP-1002) infusions in the judgement of the Investigator
• Assessed by the Investigator as willing and able to comply with the requirements of the trial

You may not qualify if:

• Planned or likely major surgery in the next 12 months after planned first infusion
• Risk of near-term respiratory decompensation in the judgment of the investigator, or the need for initiation of non-invasive ventilator support as defined by serum bicarbonate ≥ 29 mmol/L
• History of non DMD-related chronic respiratory disease including, but not limited to, asthma, bronchitis, and tuberculosis
• Acute respiratory illness within 60 days prior to first infusion
• Known hypersensitivity to dimethyl sulfoxide (DMSO) or bovine products
• Treatment with an investigational product ≤ 6 months prior to first infusion
• History, or current use, of drugs or alcohol that could impair ability to comply with participation in the trial
• Inability to comply with the investigational plan and follow-up visit schedule for any reason, in the judgment of the investigator

Sponsors & Collaborators

• Capricor Inc.: lead

Study Sites (5)

University of California, Davis

Sacramento, California, 95817, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Washington University

St Louis, Missouri, 63110, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Children's Hospital Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne; Muscular Dystrophies

Condition Hierarchy (Ancestors)

Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Mark Awadalla
• Organization: Capricor, Inc.

clinicaltrialsgov@capricor.com

858-727-1755

Study Officials

• Craig McDonald, MD

  UC Davis

  PRINCIPAL INVESTIGATOR

• Mark Awadalla

  Capricor Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Open Label Extension of the HOPE-2 Trial

Study Record Dates

• First Submitted: June 9, 2020
• First Posted: June 11, 2020
• Study Start: August 5, 2020
• Primary Completion: February 16, 2022
• Study Completion: May 1, 2026
• Last Updated: November 3, 2025
• Results First Posted: February 24, 2025

Record last verified: 2025-10

Locations

US (5)