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A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy (HOPE-2)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03406780
Recruitment Status : Completed
First Posted : January 23, 2018
Last Update Posted : June 5, 2020
Sponsor:
Information provided by (Responsible Party):
Capricor Inc.

Study Description
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Brief Summary:
HOPE-2 is a double-blind clinical trial evaluating the safety and efficacy of a cell therapy called CAP-1002 in study participants with Duchenne muscular dystrophy (DMD). Non-ambulatory and ambulatory boys and young men who meet eligibility criteria will be randomly assigned to receive either CAP-1002 or placebo every 3 months for a total of 4 doses during a 12-month period.

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

Detailed Description:
  • 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 Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 18 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: CAP-1002
Patients will receive 150 million cardiosphere-derived cells (CDCs) via intravenous infusion every 3 months for a total of 4 doses.
 Biological: CAP-1002
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.
Other Names:
  • Cardiosphere-Derived Cells
  • CDCs
Placebo Comparator: Placebo
Patients will receive a placebo solution via intravenous infusion every 3 months for a total of 4 doses.
 Drug: Placebo
Placebo


Outcome Measures
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Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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.


Eligibility Criteria
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Information from the National Library of Medicine

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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
Criteria

Inclusion Criteria:

  1. Genetically confirmed DMD
  2. Reduced upper arm strength as measured by the Performance of Upper Limb
  3. Reduced ability to walk/run (if ambulatory)
  4. 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
  5. Current and up-to-date immunizations

Exclusion Criteria:

  1. Left ventricular ejection fraction < 35%
  2. BMI > 45
  3. Ambulant if ≥ 18 years of age
  4. Exon 44 skip-amenable mutation(s) in the DMD gene
  5. Deletion mutation(s) encompassing exons 3-7 of the DMD gene
  6. Percent-predicted forced vital capacity (FVC) < 35%
  7. Chronic respiratory disease not related to DMD (for example, asthma, bronchitis, and tuberculosis)
  8. History of diabetes requiring treatment with metformin or insulin within 3 months prior to randomization
  9. 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
  10. 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
  11. Treatment with idebenone within 3 months prior to randomization
  12. Treatment with a cell therapy product within 12 months prior to randomization
  13. Treatment with an investigational product within 6 months prior to randomization
Contacts and Locations
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Information from the National Library of Medicine

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


Locations
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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
Washington University
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
Sponsors and Collaborators
Capricor Inc.
Investigators
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Principal Investigator: Craig McDonald, MD University of California, Davis
More Information
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Additional Information:

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Capricor Inc.
ClinicalTrials.gov Identifier: NCT03406780    
Other Study ID Numbers: CAP-1002-DMD-02
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

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Capricor Inc.:
Duchenne Muscular Dystrophy
Cell Therapy
Performance of the Upper Limb
Pulmonary Function
 Ambulatory
Non-Ambulatory
Glucocorticoids
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Diseases
Muscular Disorders, Atrophic
Nervous System Diseases
 Neuromuscular Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Musculoskeletal Diseases