Open-Label Extension of the HOPE-2 Duchenne Muscular Dystrophy Trial

CAP-1002-DMD-02-OLE

This Phase 2, multi-center, open-label extension trial will provide 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 sixteen intravenous administrations of CAP-1002, each separated by three months. 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 CAP-1002. 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 and 48. Safety and efficacy assessments will be conducted prior to CAP-1002 administration at the Day 1, Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42 and 45 trial visits, unless otherwise indicated. All CAP-1002 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 and 45. 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.

2020-07-20

2022-02-16

2025-03-01

Active (not recruiting)

Early phase I

13

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 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

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'Open Label Extension of the HOPE-2 Trial', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 13, 'type': 'ACTUAL'}}

2023-10-26