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Also known as Allogeneic Cardiosphere-Derived Cells, CDCs, Cardiosphere-Derived Cells, deramiocel, Allogeneic Cardiosphere-Derived Cells (CAP-1002)
CAP-1002 · 6 trials · 11 indications
Mean percent change from baseline in Performance of the Upper Limb test, version 2 (PUL 2.0) Total Score. Items are scored on a three-point scale: 0=unable to perform the item, 1=impaired or performs with compensation, 2=performs task without compensation. Percent change from baseline is calculated as change from baseline divided by baseline score at the subject level.
Adverse event (AE) is defined as any unfavourable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of study drug, whether or not considered related to the study drug. Serious AE: an AE that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial or prolonged inpatient hospitalization; congenital anomaly/birth defect or was otherwise considered medically important. TEAE: AE with onset after start of treatment or with onset date before the treatment start date but worsening after the treatment start date. TEAEs included both serious and non-serious TEAEs.
Severity of adverse events (AE) were assessed by the investigator as Grade 1 = Mild (Transient or mild discomfort; no limitation in activity; no medical intervention/therapy required), Grade 2 = Moderate (Mild to moderate limitation in activity - some assistance may be needed; no or minimal medical intervention/therapy required), Grade 3 = Severe (Marked limitation in activity, some assistance usually required; medical intervention/therapy required and often requiring hospitalization or prolongation of hospitalization), Grade 4 = Life-threatening (Extreme limitation in activity, significant assistance required; significant medical intervention/therapy required; hospitalization, prolongation of hospitalization, or hospice care) and Grade 5 = Death.
PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function.
| Arm | Type | Description |
|---|---|---|
| Deramiocel (CAP-1002) | EXPERIMENTAL | Cohort A: Approximatetly 29 subjects will receive deramiocel (CAP-1002A) active treatment consisting of 150 million cardiosphere-derived cells (CDCs) via intravenous infusion every 3 months Cohort B: Approximately 22 participants will receive deramiocel (CAP-1002B) active treatment consisting of 150 million cardiosphere-derived cells (CDCs) via intravenous infusion every 3 months Open-Label Extension: After completion of the double-blind, placebo-controlled phase, subjects will receive deramiocel (CAP-1002) active treatment consisting of 150 million cardiosphere-derived cells (CDCs) via intravenous infusion every 3 months for 12 months followed by the long-term open label extension phase which continues deramiocel administration every 3 months until sponsor decision. |
| Placebo | PLACEBO_COMPARATOR | Cohort A: Approximately 29 subjects will receive a Placebo solution via intravenous infusion every 3 months Cohort B: Approximately 22 participants will receive a Placebo solution via intravenous infusion every 3 months |
| Deramiocel | OTHER | Participants will receive an intravenous (IV) infusion of deramiocel (150 million Cardiosphere-Derived Cells (CDCs) per infusion) every 3 months |
| Name | Type | Description |
|---|---|---|
| Deramiocel (CAP-1002) | BIOLOGICAL | Cohort A: CAP-1002A manufactured in Los Angeles, CA; Cohort B: CAP-1002B manufactured in San Diego, CA; Open-Label Extension: CAP-1002A or CAP-1002B |
| Placebo | BIOLOGICAL | Placebo |
| CAP-1002 | BIOLOGICAL | Peripheral infusion of 150 million cardiosphere-derived cells (CDCs) |
| Allogeneic Cardiosphere-Derived Cells (CAP-1002) | BIOLOGICAL | Intravenous infusion delivery of Allogeneic Cardiosphere-Derived Cells (CAP-1002; 75 million CDCs) |
Inclusion Criteria: 1. Male subjects at least 10 years of age at time of consent who are willing and able to provide informed consent to participate in the trial if ≥ 18 years of age or assent with parental or guardian informed consent if \< 18 years of age. If a third-party caregiver is involved, ...
CAP-1002 is an investigational cell therapy developed by Capricor Therapeutics, Inc. (ticker CAPR). It is being studied in Phase 2 clinical trials for Covid19 and muscular dystrophies, including Duchenne muscular dystrophy. CAP-1002 has not been approved by the FDA and remains in clinical development.
CAP-1002 was studied as an intravenous infusion treatment for patients with Covid19. A Phase 2 trial, NCT04623671, evaluated intravenous infusion of CAP-1002 in 55 patients with Covid19. That trial is now completed. CAP-1002 is investigational and has not been approved for treating Covid19.
CAP-1002 has been studied in Phase 2 for muscular dystrophies, including Duchenne muscular dystrophy. The trial NCT03406780 evaluated CAP-1002 in ambulatory and non-ambulatory patients with Duchenne muscular dystrophy and enrolled 20 participants. That study is completed. CAP-1002 remains investigational for these indications.
CAP-1002 is developed by Capricor Therapeutics, Inc., which trades under the ticker CAPR. Capricor is the sponsor of the clinical trials evaluating CAP-1002 in Covid19 and muscular dystrophies, including Duchenne muscular dystrophy.
CAP-1002 is in Phase 2 clinical development. Both of its recorded trials, NCT04623671 in Covid19 and NCT03406780 in Duchenne muscular dystrophy, were Phase 2 studies and are now completed. CAP-1002 is investigational and has not been approved by the FDA.
CAP-1002 has been evaluated in two completed Phase 2 trials. NCT04623671 studied intravenous infusion of CAP-1002 in 55 patients with Covid19. NCT03406780 studied CAP-1002 in 20 ambulatory and non-ambulatory patients with Duchenne muscular dystrophy. Both trials are completed, and no CAP-1002 trials are currently active.