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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
Sponsor:
Information provided by (Responsible Party):
Capricor Inc.

Tracking Information
First Submitted Date  ICMJE January 15, 2018
First Posted Date  ICMJE January 23, 2018
Last Update Posted Date June 5, 2020
Actual Study Start Date  ICMJE March 4, 2018
Actual Primary Completion Date March 10, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 19, 2018)
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.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 19, 2018)
  • 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.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
Official Title  ICMJE 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
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.
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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE
  • Muscular Dystrophies
  • Muscular Dystrophy, Duchenne
  • Muscular Disorders, Atrophic
  • Muscular Diseases
  • Neuromuscular Diseases
  • Nervous System Diseases
  • Genetic Diseases, X-Linked
  • Genetic Diseases, Inborn
Intervention  ICMJE
  • 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
  • Drug: Placebo
    Placebo
Study Arms  ICMJE
  • Experimental: CAP-1002
    Patients will receive 150 million cardiosphere-derived cells (CDCs) via intravenous infusion every 3 months for a total of 4 doses.
    Intervention: Biological: CAP-1002
  • Placebo Comparator: Placebo
    Patients will receive a placebo solution via intravenous infusion every 3 months for a total of 4 doses.
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: January 29, 2019)
18
Original Estimated Enrollment  ICMJE
 (submitted: January 19, 2018)
84
Actual Study Completion Date  ICMJE March 10, 2020
Actual Primary Completion Date March 10, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 10 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03406780
Other Study ID Numbers  ICMJE CAP-1002-DMD-02
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party Capricor Inc.
Study Sponsor  ICMJE Capricor Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Craig McDonald, MD University of California, Davis
PRS Account Capricor Inc.
Verification Date June 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP