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 range
12 Years
Sex
MALE
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
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
. 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.
. 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.