A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy (HOPE-2)
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|ClinicalTrials.gov Identifier: NCT03406780|
Recruitment Status : Recruiting
First Posted : January 23, 2018
Last Update Posted : April 19, 2018
|Condition or disease||Intervention/treatment||Phase|
|Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Disorders, Atrophic Muscular Diseases Neuromuscular Diseases Nervous System Diseases Genetic Diseases, X-Linked Genetic Diseases, Inborn||Biological: CAP-1002 Drug: Placebo||Phase 2|
- Approximately 84 eligible study participants will be randomized to either CAP-1002 or placebo in a 1:1 ratio.
- The trial will include visits at Screening, Baseline/Day 1, Week 4, and Months 3, 6, 9, and 12 with IV infusions of CAP-1002 or placebo on Day 1 and Months 3, 6, and 9.
- Safety evaluations will include adverse events, concomitant medications, physical exam, vital signs, 12-lead ECG, and clinical laboratory testing.
- Efficacy will be evaluated in the Performance of the Upper Limb, pulmonary function testing, North Star Ambulatory Assessment (ambulatory subjects only), strength testing, cardiac MRI, and quality of life.
- If trial data suggests an appropriate risk/benefit profile of CAP-1002, Capricor, upon the recommendation of the Data Safety Monitoring Board (DSMB), will introduce an open-label extension study to offer CAP-1002 to study participants who were randomized to placebo and completed all trial visits during the 12-month period.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||84 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||A Phase 2, Randomized, Double-Blind, Placebo-Controlled Trial Evaluating the Safety and Efficacy of Intravenous Delivery of Allogeneic Cardiosphere-Derived Cells in Subjects With Duchenne Muscular Dystrophy|
|Actual Study Start Date :||April 4, 2018|
|Estimated Primary Completion Date :||December 2019|
|Estimated Study Completion Date :||April 2020|
Patients will receive 150 million cardiosphere-derived cells (CDCs) via intravenous infusion every 3 months for a total of 4 doses.
The active pharmaceutical ingredient in CAP-1002 is Cardiosphere-Derived Cells (CDCs). CDCs are known to secrete numerous bioactive elements (growth factors, exosomes) which impact the therapeutic benefits of the cell-based therapy. The mechanism of action is the composite ability to be immunomodulatory, anti-fibrotic and regenerative.
Placebo Comparator: Placebo
Patients will receive a placebo solution via intravenous infusion every 3 months for a total of 4 doses.
- Change in the mid-level (elbow) dimension of the Performance of the Upper Limb (PUL) [ Time Frame: Month 12 ]The PUL includes functional tasks that relate to activities of daily living that are very important for quality of life. The PUL has been validated for the assessment of upper limb motor function in individuals with DMD.
- Change in the mid-level (elbow) dimension of the PUL [ Time Frame: Months 3, 6, and 9 ]The PUL includes functional tasks that relate to activities of daily living that are very important for quality of life. The PUL has been validated for the assessment of upper limb motor function in individuals with DMD.
- Change in regional systolic left ventricular wall thickening as assessed by cardiac MRI [ Time Frame: Months 6 and 12 ]Systolic thickening is thought to be a principal mechanism of cardiac output generation in people with DMD.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03406780
|Contact: Brian Fedor||310-358-3201||HOPEfirstname.lastname@example.org|
|United States, California|
|University of California, Davis||Recruiting|
|Sacramento, California, United States, 95817|
|Contact: Colleen Anthonisen 916-734-4307 email@example.com|
|Principal Investigator: Craig McDonald, MD|
|Study Director:||Deborah D Ascheim, MD||Capricor Inc.|
|Principal Investigator:||Craig McDonald, MD||University of California, Davis|