| ID | Type | Description | Link |
|---|---|---|---|
| R44HL095203 | U.S. NIH Grant/Contract | View source |
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| Name | Class |
|---|---|
| Cedars-Sinai Medical Center | OTHER |
| National Institutes of Health (NIH) | NIH |
| National Heart, Lung, and Blood Institute (NHLBI) | NIH |
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To determine the safety profile of CAP-1002 administered by multi-vessel intracoronary infusion in subjects with DCM. The study will further explore safety and exploratory efficacy endpoints of CAP-1002.
Eligible subjects will undergo sequential intracoronary infusion of CAP-1002 or placebo in up to three coronary arteries supplying three major cardiac territories to the heart (anterior, lateral, inferior/posterior). After completion of the screening procedures, Phase Ia subjects will receive CAP-1002 administered via intracoronary infusion in a dose escalation, stepwise manner. Phase Ia subjects will be followed at Week 2 and at Months 1, 2, 3, 6 and 12 after CAP-1002 infusion. The first fourteen (14) subjects will receive intracoronary infusion of CAP-1002 in an open-label fashion (Phase Ia). Once all 14 subjects in the Phase Ia have reached the primary safety endpoint (1 month visit), the DSMB will conduct a review of the Phase Ia data and recommend whether to proceed with enrollment of the next 28 subjects in the Phase Ib.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Allogeneic Cardiosphere-Derived Cells | Experimental | The Phase I study consists of a Phase Ia portion and a Phase Ib portion. The Phase Ia portion (N=14 subjects) consists of an open-label, single-arm, study design. The potentially conducted Phase Ib portion of the study (N=28 subjects) consists of a double-blind, randomized, placebo-controlled study design. The Phase Ia portion is an open-label, dose escalation of Allogeneic Cardiosphere-Derived Cells (CDCs). |
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| Placebo | Placebo Comparator | The placebo study arm only applies to the Phase Ib portion of the study design. The Phase Ia portion (N=14 subjects) consists of an open-label, single-arm, study design. The potentially conducted Phase Ib portion of the study (N=28 subjects) consists of a double-blind, randomized, placebo-controlled study design. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Allogeneic Cardiosphere-Derived Cells (CDCs) | Biological | Intracoronary delivery of CAP-1002 or placebo |
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| Measure | Description | Time Frame |
|---|---|---|
| Proportion of subjects that experience new TIMI flow 0-2 or TIMI myocardial perfusion grade (TMPG) 0-2. | Intraprocedural | |
| Proportion of subjects that experience acute myocarditis, possibly attributable to CAP-1002. In order to be considered related to CAP-1002, humoral or cellular or immune reaction specific to CAP-1002 must also be documented. | Within one month of intracoronary infusion | |
| Proportion of subjects that experience ventricular tachycardia or ventricular fibrillation resulting in death, appropriate discharge of an ICD or requiring medical intervention. | During or within 72 hours of intracoronary infusion | |
| Proportion of subjects that experience sudden unexpected death occurring within one hour of symptom onset, or un-witnessed death in a person previously observed to be well within the preceding 24 hours without an identified cause. | During or within 72 hours of intracoronary infusion | |
| Proportion of subjects that experience major adverse cardiac events (MACE), including death, non-fatal myocardial infarction and re-hospitalization for cardiovascular event (including heart failure hospitalizations). | During or within 72 hours of intracoronary infusion |
| Measure | Description | Time Frame |
|---|---|---|
| Acute myocarditis possibly attributable to CAP-1002. In order to be considered related to CAP-1002, humoral or cellular immune reaction specific to CAP-1002 must also be documented. | During the six & twelve month follow-up period | |
| Ventricular tachycardia or ventricular fibrillation resulting in death or requiring medical intervention or appropriate discharge of an ICD. |
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Major Inclusion Criteria:
Major Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Rajendra (Raj) Makkar, MD | Cedars-Sinai Medical Center, Heart Institute | Principal Investigator |
| Deborah Ascheim, MD | Capricor Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Cedars-Sinai Medical Center | Los Angeles | California | 90048 | United States |
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| Label | URL |
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| Capricor's website | View source |
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| ID | Term |
|---|---|
| D002311 | Cardiomyopathy, Dilated |
| D006333 | Heart Failure |
| D009202 | Cardiomyopathies |
| D006331 | Heart Diseases |
| D018754 | Ventricular Dysfunction |
| D010335 | Pathologic Processes |
| D002318 | Cardiovascular Diseases |
| ID | Term |
|---|---|
| D006332 | Cardiomegaly |
| D000083083 | Laminopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| During the six & twelve month follow-up period |
| Sudden unexpected death defined as occurring within one hour of symptom onset, or unwitnessed death in a person previously observed to be well within the preceding 24 hours without an identified cause. | During the six & twelve month follow-up period |
| Major adverse cardiac events (MACE), including death, non-fatal myocardial infarction, hospitalization for cardiovascular event, emergency room treatment for heart failure, left ventricular assist device or heart transplant. | During the six & twelve month follow-up period |
| Any hospitalization due to a cardiovascular cause or related to CAP-1002 (or placebo in Phase Ib). | During the six & twelve month follow-up period |
| Any inter-current cardiovascular illness or one related to CAP-1002 (or placebo in Phase Ib) which prolongs hospitalization. | During the six & twelve month follow-up period |
| Development of, or an increase in the frequency of, VT with a duration of 30 seconds or longer ascertained by protocol-mandated ECG ambulatory monitoring. | During the six & twelve month follow-up period |
| Development of increased anti-Human Leukocyte Antigen (HLA) antibody levels with development of sensitization to HLA antigens specific to the CAP-1002 CDC donor at immunologically significant titers. | During the six & twelve month follow-up period |
| Total number of appropriate ICD firings. | During the six & twelve month follow-up period |
| Peak elevation in troponin and CKMB levels following CAP-1002 or placebo infusion. | Through Month 1 |
| D013568 | Pathological Conditions, Signs and Symptoms |