Male participants with cardiomyopathy secondary to Duchenne muscular dystrophy (DMD) meeting all inclusion and no exclusion criteria will be randomized. All participants will be at least 12 years of age. They will be randomized in a 1:1 manner to either intracoronary infusion of CAP-1002 in three coronary arteries supplying the three major cardiac territories of the left ventricle of the heart (anterior, lateral, inferior/posterior) or usual care. In the active treatment arm, all three major cardiac territories will be treated (infused) during a single procedure in an open-label fashion.
Approximately 24, and not more than 30, participants will be randomized into the study, in two sequential enrollment groups. Safety data from Group 1 will undergo a Data Safety Monitoring Board (DSMB) review prior to initiation of enrollment for Group 2. The first 6-8 randomized participants will comprise Group 1, and will include a minimum of 3 participants completing intracoronary infusion with CAP-1002. The DSMB will conduct a review of interim safety data through 72 hours post-Day 0 for at least 3 infused participants and for at least 6 participants overall. Enrollment of Group 2 will begin per DSMB recommendations following their review of the 72 hour safety data from Group 1. Group 2 will include approximately 18 participants. Screening and randomization will continue until at total of 12 participants are infused with CAP 1002 or 30 participants are randomized into the study, whichever comes first. All participants assigned to the active treatment arm will receive an intended total dose of up to 75 million (M) CAP-1002 cells infused as 25M cells into each of the three left ventricle cardiac territories (anterior, lateral, inferior/posterior). Participants randomized to receive usual care will continue to be cared for and treated in whatever manner the investigator deems most appropriate for the participant on an ongoing basis, and will receive no infusion. Randomization will take place within 30 days of the first screening procedure. After completion of the screening procedures, eligible participants randomized to active treatment arm will receive CAP-1002 administered via intracoronary infusion on Day 0. Day 0 for eligible participants randomized to the usual care arm will occur 7 days after the date of randomization. All randomized participants will have a follow-up telephone call on Study Day 3, and study visits at Weeks 2 and 6, and at Months 3, 6 and 12 post Day 0.
Study Type
INTERVENTIONAL
Allocation
Intracoronary delivery of Allogeneic Cardiosphere-Derived Cells (CAP-1002)
Cedars-Sinai Medical Center
Los Angeles, California, United States
University of Florida
Gainesville, Florida, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Number of Participants Experiencing Any of the Adjudicated Events
Adjudicated Events reported included: New thrombolysis in myocardial infarction (TIMI) flow 0-2 or TIMI myocardial perfusion grade (TMPG) 0-2noted immediately following infusion and persisting greater than (\>) 3 minutes, despite intracoronary vasodilator administration; sudden unexpected death within 72 hours of intracoronary infusion; and Major adverse cardiac event (MACE) within 72 hours of intracoronary infusion, including death, non-fatal myocardial infarction and hospitalization for cardiovascular event (including heart failure hospitalizations).
Time frame: Within 72 hours post-infusion
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
An Adverse Event (AE) was any untoward medical occurrence in a participant administered a pharmaceutical product and which did not necessarily had to have causal relationship with treatment. TEAEs were defined as AEs that occurred or worsened in severity between the first dose of the investigational medicinal product (IMP) until the end of study. An SAE was any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalisation or prolongation of existing hospitalisation, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a medically important event.
Time frame: Up to Month 12 post-infusion
Change From Baseline in Clinical Laboratory Parameters (Chloride, Potassium and Sodium) at Month 6 and Month 12
Clinical chemistry parameters assessed were chloride, potassium and sodium.
Time frame: Baseline, Month 6 and Month 12
Change From Baseline in Clinical Laboratory Parameter - Albumin at Month 6 and Month 12
Clinical chemistry parameter assessed was albumin.
Time frame: Baseline, Month 6 and Month 12
Change From Baseline in Clinical Laboratory Parameter - Glucose at Month 6 and Month 12
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RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
25
Clinical chemistry parameter assessed was glucose.
Time frame: Baseline, Month 6 and Month 12
Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12
Hematological parameters assessed were: platelets, white blood cells, basophils, eosinophils, lymphocytes, monocytes and neutrophils.
Time frame: Baseline, Month 6 and Month 12
Change From Baseline in Hematological Parameter - Hemoglobin at Month 6 and Month 12
Hematological parameter assessed was hemoglobin.
Time frame: Baseline, Month 6 and Month 12
Change From Baseline in Hematological Parameter - Red Blood Cells at Month 6 and Month 12
Hematological parameter assessed was red blood cells.
Time frame: Baseline, Month 6 and Month 12
Change From Baseline in Vital Signs - Blood Pressure at Month 6 and Month 12
Vital signs assessed were systolic and diastolic blood pressure.
Time frame: Baseline, Month 6 and Month 12
Change From Baseline in Vital Signs - Heart Rate at Month 6 and Month 12
Vital signs assessed was heart rate.
Time frame: Baseline, Month 6 and Month 12
Change From Baseline in Vital Signs - Respiratory Rate at Month 6 and Month 12
Vital signs assessed was respiratory rate.
Time frame: Baseline, Month 6 and Month 12
Change From Baseline in Vital Signs - Temperature at Month 6 and Month 12
Vital signs assessed was temperature.
Time frame: Baseline, Month 6 and Month 12
Number of Participants With Clinically Significant Change From Baseline in Cardiac Physical Examinations at Month 6 and Month 12
Cardiac physical examination parameters assessed were: jugular vein distension, edema, heart sounds, murmur, breath sounds.
Time frame: Baseline, Month 6 and Month 12
Change From Baseline in Electrocardiogram (ECG) Parameters (QRS Duration, PR, QT, QTc and QT Interval) at Month 6 and Month 12
ECG parameters assessed were: PR Interval, QRS Duration, QT Interval, QTc interval and QT interval.
Time frame: Baseline, Month 6 and Month 12
Change From Baseline in Electrocardiogram Parameter - Ventricular Rate at Month 6 and Month 12
ECG parameter assessed was ventricular rate; which depends on the degree of atrioventricular conduction.
Time frame: Baseline, Month 6 and Month 12