for males ages 10 years and up (full criteria)
at UC Davis
study started
estimated completion
Craig McDonald, MD(ucdavis)



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.

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


  • 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.


Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Disorders, Atrophic Muscular Diseases Neuromuscular Diseases Nervous System Diseases Genetic Diseases, X-Linked Genetic Diseases, Inborn Duchenne Muscular Dystrophy Cell Therapy Performance of the Upper Limb Pulmonary Function Ambulatory Non-Ambulatory Glucocorticoids CAP-1002


You can join if…

Open to males ages 10 years and up

  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

You CAN'T join if...

  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


  • University of California, Davis
    Sacramento California 95817 United States
  • University of Utah
    Salt Lake City Utah 84112 United States

Lead Scientist

  • Craig McDonald, MD (ucdavis)
    Professor, Physical Medicine and Rehabilitation. Authored (or co-authored) 173 research publications


in progress, not accepting new patients
Start Date
Completion Date
Capricor Inc.
Company website
Phase 2
Study Type
Last Updated