Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Deramiocel (CAP-1002)
Deramiocel · 2 trials · 9 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 |
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, also known as CAP-1002, is an investigational cell therapy being developed for Duchenne Muscular Dystrophy and other muscular dystrophies. It is currently in clinical development and has not been approved by the FDA. The drug has received FDA designations including orphan drug, regenerative medicine advanced therapy, rare pediatric disease, and priority review.
Deramiocel is a cell therapy, classified under the -cel (cell tx) target class. It is being studied for its potential effects in Duchenne Muscular Dystrophy, a genetic neuromuscular disease. The specific molecular target of Deramiocel has not been disclosed in available information.
Deramiocel is being developed by Capricor Therapeutics, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol CAPR. The company is conducting clinical trials to evaluate the safety and efficacy of Deramiocel in patients with Duchenne Muscular Dystrophy.
Deramiocel is currently in Phase 2 clinical development. It is being studied in an open-label extension of the HOPE-2 trial and a Phase 3 study in ambulatory and non-ambulatory patients with Duchenne Muscular Dystrophy. The drug is investigational and has not received FDA approval.
Deramiocel is being evaluated in two clinical trials. NCT04428476 is an open-label extension of the HOPE-2 trial in Duchenne Muscular Dystrophy, with 13 participants. NCT05126758 is a Phase 3 study in ambulatory and non-ambulatory patients with Duchenne Muscular Dystrophy, enrolling 106 male participants aged 10 years and older.
Yes, Deramiocel is also known as CAP-1002. Both names refer to the same investigational cell therapy being developed by Capricor Therapeutics for Duchenne Muscular Dystrophy. The drug is currently in Phase 2 clinical trials and has received several FDA designations, including orphan drug and regenerative medicine advanced therapy.