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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Allogeneic Cardiosphere-Derived Cells (CAP-1002) | Biological | Intravenous infusion delivery of Allogeneic Cardiosphere-Derived Cells (CAP-1002; 75 million CDCs) |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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. | From Day 1 up to Month 6 |
| All-cause Mortality | Number of deaths due to any cause will be reported. | 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. | From Day 1 up to Month 6 |
| Number of Participants With Immune Sensitization Syndrome |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Mark Awadalla | Capricor Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Florida | Gainesville | Florida | 32610 | United States | ||
| Cincinnati Children's Medical Center |
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| ID | Title | Description |
|---|---|---|
| FG000 | 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) and received CAP-1002 intravenous infusion on Day 1 and at Month 3 in the current study. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | 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), and received CAP-1002 intravenous infusion on Day 1 and at Month 3 in the current study. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Analysis was performed on safety population that included all enrolled participants. | Posted | Count of Participants | Participants | 2 hours post-dose on Day 1 and Month 3 |
|
From Day 1 up to Month 6
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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), and received CAP-1002 intravenous infusion on Day 1 and at Month 3 in the current study. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaphylactic Reaction | Immune system disorders | Non-systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nasopharyngitis | Infections and infestations | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Capricor Inc. | 858-727-1755 | mawadalla@capricor.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Feb 12, 2018 | Mar 29, 2024 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 26, 2019 | Mar 29, 2024 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| D009202 | Cardiomyopathies |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
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|
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.
| From Day 1 up to Month 6 |
| Cincinnati |
| Ohio |
| 45229 |
| United States |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Primary | 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. | Analysis was performed on safety population. | Posted | Count of Participants | Participants | From Day 1 up to Month 6 |
|
|
|
| Primary | All-cause Mortality | Number of deaths due to any cause will be reported. | Analysis was performed on safety population. | Posted | Count of Participants | Participants | From Day 1 up to Month 6 |
|
|
|
| Primary | 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. | Analysis was performed on safety population. | Posted | Number | number of events | From Day 1 up to Month 6 |
|
|
|
| Primary | 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. | Analysis was performed on safety population. | Posted | Count of Participants | Participants | From Day 1 up to Month 6 |
|
|
|
| 0 |
| 8 |
| 1 |
| 8 |
| 7 |
| 8 |
| Oesophageal Ulcer | Gastrointestinal disorders | Non-systematic Assessment |
|
| Bronchitis | Infections and infestations | Non-systematic Assessment |
|
| Drug Eruption | Skin and subcutaneous tissue disorders | Non-systematic Assessment |
|
| Tachycardia | Cardiac disorders | Non-systematic Assessment |
|
| Dizziness | Nervous system disorders | Non-systematic Assessment |
|
| Headache | Nervous system disorders | Non-systematic Assessment |
|
| Eye Pain | Eye disorders | Non-systematic Assessment |
|
| Nausea | Gastrointestinal disorders | Non-systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | Non-systematic Assessment |
|
| Infusion Related Reaction | Injury, poisoning and procedural complications | Non-systematic Assessment |
|
| Rash Erythematous | Skin and subcutaneous tissue disorders | Non-systematic Assessment |
|
| Nasal Congestion | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
|
| Haemorrhoids | Gastrointestinal disorders | Non-systematic Assessment |
|
| Paraesthesia | Nervous system disorders | Non-systematic Assessment |
|
| Onychomycosis | Infections and infestations | Non-systematic Assessment |
|
| Tibia Fracture | Injury, poisoning and procedural complications | Non-systematic Assessment |
|
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | Non-systematic Assessment |
|
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| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D006331 | Heart Diseases |
| D002318 | Cardiovascular Diseases |