Halt cardiomyOPathy progrEssion in Duchenne (HOPE-OLE) (NCT06304064) | Clinical Trial Compass
CompletedPhase 2
Halt cardiomyOPathy progrEssion in Duchenne (HOPE-OLE)
United States8 participantsStarted 2018-06-21
Plain-language 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.
Who can participate
Age range12 Years
SexMALE
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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 (\<) 18 years of age.
✓. Adequate venous access for intravenous CAP-1002 infusions and routine blood collections in the judgement of the Investigator.
✓. Assessed by the Investigator as willing and able to comply with the requirements of the trial.
Exclusion criteria
✕. Left ventricular ejection fraction (LVEF) \< 35 percent (%) within 6 months of screening.
✕. Planned or likely major surgery in the next 6 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 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.
✕
What they're measuring
1
Number of Participants Experiencing Acute Respiratory Decompensation
Timeframe: 2 hours post-dose on Day 1 and Month 3
2
Number of Participants With Hypersensitivity Reactions
Timeframe: From Day 1 up to Month 6
3
All-cause Mortality
Timeframe: From Day 1 up to Month 6
4
Number of Treatment-emergent Adverse Events (TEAEs) Related to Investigational Product or Administration and Serious Adverse Events (SAEs)
Timeframe: From Day 1 up to Month 6
5
Number of Participants With Immune Sensitization Syndrome