Open-label Extension of the HOPE-2 Trial

Running, not enrolling · Phase 2

Conditions studied: Duchenne Muscular Dystrophy

In brief

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.

Key facts

Study ID: NCT04428476
Run by: Capricor Inc.
People needed: 13
Starts: 2020-08-05
Expected to finish: 2026-05-01
Last updated by the study team: 2025-11-03

Who can join

Age: 10 and older. Sex: any. Healthy volunteers: not accepted.

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

Where it is running

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

Full record on ClinicalTrials.gov

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