Phase 2
N=8
Halt cardiomyOPathy progrEssion in Duchenne (HOPE-OLE)
Duchenne Muscular Dystrophy
Bottom Line
View on ClinicalTrials.gov: NCT06304064 ↗Enrolled (actual)
8
Serious AEs
12.5%
Results posted
Apr 2024
Primary outcome: Primary: Number of Participants Experiencing Acute Respiratory Decompensation — 0; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Allogeneic Cardiosphere-Derived Cells (CAP-1002) (Biological)
- Age
- Pediatric, Adult, Older Adult · 12+ yrs
- Sex
- Male
- Sponsor
- Capricor Inc.
- Primary completion
- Mar 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants Experiencing Acute Respiratory Decompensation |
0; 0 | — |
| PRIMARY Number of Participants With Hypersensitivity Reactions |
1 | — |
| PRIMARY All-cause Mortality |
— | — |
| PRIMARY Number of Treatment-emergent Adverse Events (TEAEs) Related to Investigational Product or Administration and Serious Adverse Events (SAEs) |
11; 1 | — |
| PRIMARY Number of Participants With Immune Sensitization Syndrome |
— | — |
Summary
This Phase 2, multi-center, open-label extension trial will provide CAP-1002 to participants who were randomized to the Usual Care treatment group of the HOPE-Duchenne study (NCT02485938) and completed 12 months of follow-up.
The trial will assess the safety and efficacy of two intravenous administrations of CAP-1002, each separated by three months.
Eligibility Criteria
Inclusion Criteria
- Documented enrollment in the Usual Care Treatment Group of the HOPE-Duchenne trial and completion of trial follow-up through Month 12.
- Willing and able to provide informed consent to participate in the trial if greater than or equal to (>=) 18 years of age, and assent with parental or guardian informed consent if less than ( = 29 millimoles per liter (mmol/L) at screening.
- History of non DMD-related chronic respiratory disease including, but not limited to, asthma, bronchitis, and tuberculosis.
- Acute respiratory illness within 30 days prior to screening.
- Known hypersensitivity to dimethyl sulfoxide (DMSO) or bovine products.
- Treatment with 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.
Data sourced from ClinicalTrials.gov (NCT06304064). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.