Duchenne Muscular DystrophyPhase 2HOPE-OLENCT06304064
Halt cardiomyOPathy progrEssion in Duchenne (HOPE-OLE)
A Phase 2 study testing Allogeneic Cardiosphere-Derived Cells (CAP-1002) in people with Duchenne Muscular Dystrophy, run by Capricor Inc. at 2 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 years and older, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Allogeneic Cardiosphere-Derived Cells (CAP-1002)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 2 sitesUnited States
- Study dates
- 21 Jun 2018to 6 Mar 2019
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Show all 9Show fewer
- 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.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Cincinnati Children's Medical Center
Cincinnati, Ohio, 45229
University of Florida
Gainesville, Florida, 32610
About this study
Official title: Open-Label Extension of the Halt Cardiomyopathy Progression in Duchenne (HOPE-Duchenne) Trial (CAP-1002-DMD-03)
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.
Read the detailed description
Participants with documented enrollment in the Usual Care treatment group of the HOPE-Duchenne study and completion of study follow-up through Month 12 were eligible for this study.
Participants 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.
All CAP-1002 infusions will be conducted in an outpatient setting at the investigative site on Day 1 and at Month 3. Participants 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.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Allogeneic Cardiosphere-Derived Cells (CAP-1002)
- Keywords
- Duchenne Muscular Dystrophy, Cardiomyopathy, Duchenne
- Medical subject headings
- Muscular Dystrophy, Duchenne, Cardiomyopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Allogeneic Cardiosphere-Derived Cells (CAP-1002)Biological
Also called CAP-1002
Intravenous infusion delivery of Allogeneic Cardiosphere-Derived Cells (CAP-1002; 75 million CDCs)
| Study group | Role | What they receive |
|---|---|---|
| Allogeneic Cardiosphere-Derived Cells (CAP-1002) | Experimental | All participants who were randomized to the Usual Care Treatment Group and completed 12 months of follow-up in the HOPE-Duchenne trial (NCT02485938), will receive CAP-1002 intravenous infusion on Day 1 and at Month 3 in the current study. Receives: Biological: Allogeneic Cardiosphere-Derived Cells (CAP-1002) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Experiencing Acute Respiratory Decompensation
Acute respiratory decompensation is defined as an unexplained rapid deterioration of the participant's condition with increasing shortness of breath requiring oxygen supplementation. Acute respiratory decompensation within 2 hours following investigational product (IP) administration will be reported.
Measured: 2 hours post-dose on Day 1 and Month 3
Number of Participants With Hypersensitivity Reactions
Hypersensitivity reaction is defined as a clinical syndrome including, but not limited to, fever, leukocytosis, or rash with onset <= 2 hours post-infusion and lasting < 24 hours, in the absence of clinical signs of concomitant infection.
Measured: From Day 1 up to Month 6
All-cause Mortality
Number of deaths due to any cause will be reported.
Measured: From Day 1 up to Month 6
Number of Treatment-emergent Adverse Events (TEAEs) Related to Investigational Product or Administration and Serious Adverse Events (SAEs)
An adverse events (AEs) is any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. TEAEs are defined as AEs occurring after the initiation of the IV catheter placement for the initial dose of IP. TEAEs related to investigational product or administration are reported for this outcome measure. A SAE is defined as an AE that results in any of the following outcomes: Death; life-threatening adverse event; Inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions; congenital anomaly/birth defect.
Measured: From Day 1 up to Month 6
Number of Participants With Immune Sensitization Syndrome
Immune sensitization syndrome shall be defined as: (a) clinical signs and symptoms consistent with systemic inflammation (e.g., fever, leukocytosis, rash, or arthralgia) with onset >= 24 hours post infusion and the absence of clinical signs of concomitant infection, and (b) elevation of anti-human leukocyte antigen (HLA) antibodies against the donor cells (i.e., DSAs), detected <= 30 days following onset of syndrome, of (i) >= 2000 mean fluorescent intensity (MFI) if baseline MFI <= 1000, or (ii) >= 2 times baseline otherwise.
Measured: From Day 1 up to Month 6
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Mar 2024
- Study start21 Jun 2018
- Primary completion6 Mar 2019
- Study completion6 Mar 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Capricor Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Mark Awadalla, Study director, Capricor Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06304064
- Sponsor's protocol ID
- CAP-1002-DMD-03
- Acronym
- HOPE-OLE
- Status last verified
- Mar 2024
- First submitted
- 5 Mar 2024
- Last updated
- 24 Apr 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open-Label Extension of the Halt Cardiomyopathy Progression in Duchenne (HOPE-Duchenne) Trial (CAP-1002-DMD-03)
- ClinicalTrials.gov ID
- NCT06304064
- Acronym / sponsor ID
- HOPE-OLE / CAP-1002-DMD-03
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne Muscular Dystrophy, Cardiomyopathy, Duchenne
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Capricor Inc.Industry (pharma, biotech, devices)
- Study start
- 21 Jun 2018 (actual)
- Primary completion
- 6 Mar 2019 (actual)
- Study completion
- 6 Mar 2019 (actual)
- First posted
- 12 Mar 2024 (actual)
- Results
- Posted 24 Apr 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Participants with documented enrollment in the Usual Care treatment group of the HOPE-Duchenne study and completion of study follow-up through Month 12 were eligible for this study.
Participants 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.
All CAP-1002 infusions will be conducted in an outpatient setting at the investigative site on Day 1 and at Month 3. Participants 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Allogeneic Cardiosphere-Derived Cells (CAP-1002) | Experimental | Biological: Allogeneic Cardiosphere-Derived Cells (CAP-1002)All participants who were randomized to the Usual Care Treatment Group and completed 12 months of follow-up in the HOPE-Duchenne trial (NCT02485938), will receive CAP-1002 intravenous infusion on Day 1 and at Month 3 in the current study. |
Who may qualify
- Ages
- 12 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (9 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Allogeneic Cardiosphere-Derived Cells (CAP-1002)Also: CAP-1002Intravenous infusion delivery of Allogeneic Cardiosphere-Derived Cells (CAP-1002; 75 million CDCs) | Biological | Allogeneic Cardiosphere-Derived Cells (CAP-1002) |
Primary outcome
Number of Participants Experiencing Acute Respiratory Decompensation
Time frame: 2 hours post-dose on Day 1 and Month 3
Acute respiratory decompensation is defined as an unexplained rapid deterioration of the participant's condition with increasing shortness of breath requiring oxygen supplementation. Acute respiratory decompensation within 2 hours following investigational product (IP) administration will be reported.
Number of Participants With Hypersensitivity Reactions
Time frame: From Day 1 up to Month 6
Hypersensitivity reaction is defined as a clinical syndrome including, but not limited to, fever, leukocytosis, or rash with onset <= 2 hours post-infusion and lasting < 24 hours, in the absence of clinical signs of concomitant infection.
All-cause Mortality
Time frame: From Day 1 up to Month 6
Number of deaths due to any cause will be reported.
Number of Treatment-emergent Adverse Events (TEAEs) Related to Investigational Product or Administration and Serious Adverse Events (SAEs)
Time frame: From Day 1 up to Month 6
An adverse events (AEs) is any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. TEAEs are defined as AEs occurring after the initiation of the IV catheter placement for the initial dose of IP. TEAEs related to investigational product or administration are reported for this outcome measure. A SAE is defined as an AE that results in any of the following outcomes: Death; life-threatening adverse event; Inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions; congenital anomaly/birth defect.
Number of Participants With Immune Sensitization Syndrome
Time frame: From Day 1 up to Month 6
Immune sensitization syndrome shall be defined as: (a) clinical signs and symptoms consistent with systemic inflammation (e.g., fever, leukocytosis, rash, or arthralgia) with onset >= 24 hours post infusion and the absence of clinical signs of concomitant infection, and (b) elevation of anti-human leukocyte antigen (HLA) antibodies against the donor cells (i.e., DSAs), detected <= 30 days following onset of syndrome, of (i) >= 2000 mean fluorescent intensity (MFI) if baseline MFI <= 1000, or (ii) >= 2 times baseline otherwise.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Apr 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mark Awadalla study director
Capricor Inc.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Allogeneic Cardiosphere-Derived Cells (CAP-1002) in people with Duchenne Muscular Dystrophy, run by Capricor Inc. at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 24 Apr 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants Experiencing Acute Respiratory Decompensation (time frame: 2 hours post-dose on Day 1 and Month 3). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Apr 2024.