Summary

Eligibility
for people ages 10 years and up (full criteria)
Location
at UC Davis
Dates
study started
estimated completion
Principal Investigator
by Craig McDonald, MD (ucdavis)

Description

Summary

This Phase 2, multi-center, open-label extension trial will provide CAP-1002 to subjects that were enrolled in the HOPE-2 trial and completed 12 months of follow-up. The trial will explore the safety and efficacy of eight intravenous administrations of CAP-1002, each separated by three months. Subjects will undergo a targeted screening during a 30-day screening period, eligible subjects will then undergo baseline safety and efficacy assessments on Day 1 prior to their first infusion of CAP-1002. Subjects will complete trial assessments at Screening; Day 1; Months 3, 6, 9, 12, 15, 18, 21 and 24. Safety and efficacy assessments will be conducted prior to CAP-1002 administration at the Day 1, Months 3, 6, 9, 12, 15, 18 and 21 trial visits, unless otherwise indicated. All CAP-1002 infusions will be conducted in an outpatient setting at the investigative site on Day 1 and Months 3, 6, 9, 12, 15, 18 and 21. Subjects will be observed in the outpatient setting for at least two hours post infusion and then discharged the same day if medically cleared by the site Investigator.

Official Title

Open-Label Extension of the HOPE-2 Duchenne Muscular Dystrophy Trial

Details

This Phase 2, multi-center, open-label extension trial will provide CAP-1002 to subjects that were enrolled in the HOPE-2 trial and completed 12 months of follow-up. The trial will explore the safety and efficacy of eight intravenous administrations of CAP-1002, each separated by three months. Subjects will undergo a targeted screening during a 30-day screening period to determine eligibility based on protocol inclusion and exclusion criteria. Eligible subjects will undergo baseline safety and efficacy assessments on Day 1 prior to their first infusion of CAP-1002. Administration of CAP-1002 (Day 1) should occur within a maximum of 30 days following confirmation of eligibility. Subjects will complete trial assessments at Screening; Day 1; Months 3, 6, 9 12, 15, 18, 21 and 24 (±14 days, each). Safety and efficacy assessments will be conducted prior to CAP-1002 administration at the Day 1, Months 3, 6, 9, 12, 15, 18 and 21 trial visits, unless otherwise indicated. All CAP-1002 infusions will be conducted in an outpatient setting at the investigative site on Day 1 and Months 3, 6 and 9 , 12, 15, 18 and 21. Prior to each CAP-1002 administration, medications will be administered to the subject as determined by the Investigator based on the pre-treatment guidelines as outlined in the protocol and/or institutional protocols to minimize the risk of potential severe allergic reactions such as anaphylaxis. Subjects will be observed in the outpatient setting for at least two hours post infusion and then discharged the same day if medically cleared by the site Investigator. If clinically indicated, an unscheduled in-person visit will be performed at the investigative site with targeted assessments based on presentation of signs and symptoms following any infusion.

Keywords

Duchenne Muscular Dystrophy DMD, Muscular dystrophy, Duchenne Muscular Dystrophies Muscular Dystrophy, Duchenne CAP-1002

Eligibility

You can join if…

Open to people ages 10 years and up

  1. Documented enrollment in the HOPE-2 trial and completion of trial follow-up through Month 12
  2. Willing and able to provide informed consent to participate in the trial if ≥ 18 years of age, and assent with parental or guardian informed consent if < 18 years of age
  3. Adequate venous access for intravenous CAP-1002 infusions in the judgement of the Investigator
  4. Assessed by the Investigator as willing and able to comply with the requirements of the trial

You CAN'T join if...

  1. Planned or likely major surgery in the next 12 months after planned first infusion
  2. Risk of near-term respiratory decompensation in the judgment of the investigator, or the need for initiation of non-invasive ventilator support as defined by serum bicarbonate ≥ 29 mmol/L
  3. History of non DMD-related chronic respiratory disease including, but not limited to, asthma, bronchitis, and tuberculosis
  4. Acute respiratory illness within 60 days prior to first infusion
  5. Known hypersensitivity to dimethyl sulfoxide (DMSO) or bovine products
  6. Treatment with an investigational product ≤ 6 months prior to first infusion
  7. History, or current use, of drugs or alcohol that could impair ability to comply with participation in the trial
  8. Inability to comply with the investigational plan and follow-up visit schedule for any reason, in the judgment of the investigator

Locations

  • University of California, Davis
    Sacramento California 95817 United States
  • Children's Hospital Colorado
    Aurora Colorado 80045 United States

Lead Scientist at University of California Health

  • Craig McDonald, MD (ucdavis)
    Professor, Physical Medicine and Rehabilitation. Authored (or co-authored) 214 research publications

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Capricor Inc.
ID
NCT04428476
Phase
Phase 2 Duchenne Muscular Dystrophy Research Study
Study Type
Interventional
Participants
At least 13 people participating
Last Updated