NCT04428476Active, not recruitingPhase 2
Open-label Extension of the HOPE-2 Trial
Open-Label Extension of the HOPE-2 Duchenne Muscular Dystrophy Trial
Source: ClinicalTrials.gov · Last updated 22 Sept 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 13 (actual)
- First submitted
- 9 Jun 2020
- Start date
- 5 Aug 2020
- Primary completion date
- 16 Feb 2022
- Lead sponsor
- Capricor Inc.
- Diseases
- Duchenne muscular dystrophy
- NCT number
- NCT04428476
What you commit to
- Study type: Interventional · Phase 2
- Participants enrolled: 13
- From 5 Aug 2020 to the primary completion date, 16 Feb 2022
- 5 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Planned or likely major surgery in the next 12 months after planned first infusion
- Acute respiratory illness within 60 days prior to first infusion
- 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
Other illness
- History of non DMD-related chronic respiratory disease including, but not limited to, asthma, bronchitis, and tuberculosis
- Known hypersensitivity to dimethyl sulfoxide (DMSO) or bovine products
Lab values
- 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
Other
- Inability to comply with the investigational plan and follow-up visit schedule for any reason, in the judgment of the investigator
Who the study is looking for
- 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
Age in the registry: 10 years to - · Sex: All · Does not accept healthy volunteers
Sites (5)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Children's Hospital Colorado Coordinator of this site? Confirm its status | Aurora | United States | Unknown status |
| Cincinnati Children's Hospital Medical Center Coordinator of this site? Confirm its status | Cincinnati | United States | Unknown status |
| Children's Hospital Wisconsin Coordinator of this site? Confirm its status | Milwaukee | United States | Unknown status |
| University of California - Davis Coordinator of this site? Confirm its status | Sacramento | United States | Unknown status |
| Washington University Coordinator of this site? Confirm its status | St Louis | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.