Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Deramiocel (CAP-1002), CAP-1002, Cardiosphere-Derived Cells, CDCs
Deramiocel · 4 trials · 10 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) |
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, ...
Deramiocel is being developed for the treatment of Duchenne muscular dystrophy and other muscular dystrophies. It is a cell therapy under development by Capricor Therapeutics, Inc. (ticker: CAPR) for patients with these neuromuscular conditions.
Deramiocel is a cell therapy, and its target class is described as '-cel (cell tx)'. This indicates it is a cellular therapeutic approach rather than a small molecule or antibody directed at a specific molecular target.
Deramiocel is being developed by Capricor Therapeutics, Inc., which trades under the ticker CAPR. The company is responsible for the clinical development program of this cell therapy for Duchenne muscular dystrophy and related muscular dystrophies.
Deramiocel is in the NDA/BLA stage of development, meaning the developer has submitted or is preparing a New Drug Application or Biologics License Application. It has received FDA designations including Orphan Drug, RMAT, Rare Pediatric Disease, and Priority Review.
Deramiocel is being studied in a Phase 3 trial, NCT05126758, titled 'A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy,' which is active but not recruiting and has enrolled 106 patients. An open-label extension of the HOPE-2 trial, NCT04428476, is also active.
Yes, Deramiocel is also known as CAP-1002. The two names refer to the same cell therapy developed by Capricor Therapeutics for Duchenne muscular dystrophy and other muscular dystrophies.