Open-label Extension of the HOPE-2 Trial

NCT ID: NCT04428476

Last Updated: 2025-11-03

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 13 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-08-05
• Study Completion Date: 2026-05-31

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.

Detailed Description

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 approximately a period of 60 months. Additional analyses of efficacy and safety will be conducted in the subsequent Long Term Open-Label Extension (LT-OLE) phase of the study.

Subjects will undergo a targeted screening during a 30-day screening period to determine eligibility based on protocol inclusion and exclusion criteria.

Eligible subjects will undergo baseline safety and efficacy assessments on Day 1 prior to their first infusion of deramiocel. Administration of deramiocel (Day 1) should occur within a maximum of 30 days following confirmation of eligibility.

Subjects will complete trial assessments at Screening; Day 1; Months 3, 6, 9, 12 (± 14 days, each), 15, 18, 21, 24, 27, 30, 30, 33, 36, 39, 42, 45, 48, 51, 54, 57, 60, and all LT-OLE visits (± 21 days, each). 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, and at all LT-OLE visits.

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 trial visits. Continued dosing in the LT-OLE phase will begin at LT-OLE Visit 1, which may overlap with Month 60 of the initial OLE phase/LT-OLE Visit 1, in some cases. Prior to each deramiocel administration, medications will be administered to the subject as determined by the Investigator based on the pre-treatment guidelines as outlined in the protocol and/or institutional protocols to minimize the risk of potential severe allergic reactions such as anaphylaxis. 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. If clinically indicated, an unscheduled in-person visit will be performed at the investigative site with targeted assessments based on presentation of signs and symptoms following any infusion.

Conditions

Duchenne Muscular Dystrophy

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Deramiocel

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

Group Type: OTHER

Deramiocel (CAP-1002)

Intervention Type: BIOLOGICAL

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

Interventions

Deramiocel (CAP-1002)

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

Intervention Type: BIOLOGICAL

Other Intervention Names

Allogeneic Cardiosphere-Derived Cells

Eligibility Criteria

Inclusion Criteria

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

2. 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

3. Adequate venous access for intravenous deramiocel (CAP-1002) infusions in the judgement of the Investigator

4. Assessed by the Investigator as willing and able to comply with the requirements of the trial

Exclusion Criteria

1. Planned or likely major surgery in the next 12 months after planned first infusion

2. 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

3. History of non DMD-related chronic respiratory disease including, but not limited to, asthma, bronchitis, and tuberculosis

4. Acute respiratory illness within 60 days prior to first infusion

5. Known hypersensitivity to dimethyl sulfoxide (DMSO) or bovine products

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

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

8. Inability to comply with the investigational plan and follow-up visit schedule for any reason, in the judgment of the investigator

• Minimum Eligible Age: 10 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Capricor Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Craig McDonald, MD

Role: PRINCIPAL_INVESTIGATOR

UC Davis

Mark Awadalla

Role: STUDY_DIRECTOR

Capricor Inc.

Locations

University of California, Davis

Sacramento, California, United States

Children's Hospital Colorado

Aurora, Colorado, United States

Washington University

St Louis, Missouri, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Children's Hospital Wisconsin

Milwaukee, Wisconsin, United States

Countries

United States

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

CAP-1002-DMD-02-OLE

Identifier Type: -

Identifier Source: org_study_id