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 : Completed
First Posted : January 23, 2018
Last Update Posted : June 5, 2020
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|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)|
|Actual Enrollment :||18 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 :||March 4, 2018|
|Actual Primary Completion Date :||March 10, 2020|
|Actual Study Completion Date :||March 10, 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.
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|Ages Eligible for Study:||10 Years and older (Child, Adult, Older Adult)|
|Sexes Eligible for Study:||Male|
|Accepts Healthy Volunteers:||No|
- Genetically confirmed DMD
- Reduced upper arm strength as measured by the Performance of Upper Limb
- Reduced ability to walk/run (if ambulatory)
- Treatment with systemic glucocorticoids for at least 12 months and at a stable dose at least 6 months prior to study participation, except for weight-based or toxicity-related adjustments
- Current and up-to-date immunizations
- Left ventricular ejection fraction < 35%
- BMI > 45
- Ambulant if ≥ 18 years of age
- Exon 44 skip-amenable mutation(s) in the DMD gene
- Deletion mutation(s) encompassing exons 3-7 of the DMD gene
- Percent-predicted forced vital capacity (FVC) < 35%
- Chronic respiratory disease not related to DMD (for example, asthma, bronchitis, and tuberculosis)
- History of diabetes requiring treatment with metformin or insulin within 3 months prior to randomization
- Treatment with an FDA-approved exon skipping therapy for the treatment of DMD if on a stable dose for less than 24 months prior to randomization
- Treatment with human growth hormone (HGH) within 3 months prior to randomization, unless on a stable dose for at least 24 months prior to randomization
- Treatment with idebenone within 3 months prior to randomization
- Treatment with a cell therapy product within 12 months prior to randomization
- Treatment with an investigational product within 6 months prior to randomization
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
|United States, California|
|University of California, Davis|
|Sacramento, California, United States, 95817|
|United States, Colorado|
|Children's Hospital Colorado|
|Aurora, Colorado, United States, 80045|
|United States, Florida|
|Nemours Children's Hospital|
|Orlando, Florida, United States, 32827|
|United States, Missouri|
|Saint Louis, Missouri, United States, 63110|
|United States, Ohio|
|Cincinnati Children's Hospital Medical Center|
|Cincinnati, Ohio, United States, 45229|
|United States, Utah|
|University of Utah|
|Salt Lake City, Utah, United States, 84112|
|United States, Wisconsin|
|Children's Hospital Wisconsin|
|Milwaukee, Wisconsin, United States, 53226|
|Principal Investigator:||Craig McDonald, MD||University of California, Davis|
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
|Responsible Party:||Capricor Inc.|
|Other Study ID Numbers:||
|First Posted:||January 23, 2018 Key Record Dates|
|Last Update Posted:||June 5, 2020|
|Last Verified:||June 2020|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||Undecided|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Duchenne Muscular Dystrophy
Performance of the Upper Limb
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked