Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Open-label Extension of the HOPE-2 Trial (HOPE-2-OLE)

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: NCT04428476
Recruitment Status : Enrolling by invitation
First Posted : June 11, 2020
Last Update Posted : December 1, 2020
Sponsor:
Information provided by (Responsible Party):
Capricor Inc.

Brief 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 four 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 and 12. Safety and efficacy assessments will be conducted prior to CAP-1002 administration at the Day 1, Months 3, 6 and 9 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. 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.


Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Biological: CAP-1002 Phase 2

Detailed Description:

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 four 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 and 12 (±14 days, each). Safety and efficacy assessments will be conducted prior to CAP-1002 administration at the Day 1, Months 3, 6 and 9 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. 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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 14 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: Open Label Extension of the HOPE-2 Trial
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Open-Label Extension of the HOPE-2 Duchenne Muscular Dystrophy Trial
Actual Study Start Date : July 20, 2020
Estimated Primary Completion Date : October 2021
Estimated Study Completion Date : October 2021


Arm Intervention/treatment
Open-label arm
Open-label CAP-1002 will be administered to all subjects enrolled in the trial
Biological: CAP-1002
Peripheral infusion of 150 million allogeneic cardiosphere-derived cells administered every three months
Other Name: Allogeneic Cardiosphere-Derived Cells




Primary Outcome Measures :
  1. The primary safety endpoint is the incidence and severity of all treatment-emergent adverse events [ Time Frame: From baseline through Month 12 timepoint ]
    The incidence and severity of all treatment-emergent adverse events will be evaluated over the course of the trial

  2. The primary efficacy endpoint is change from baseline at the Month 12 timepoint in functional capacity as assessed by the full PUL 2.0 [ Time Frame: Change from baseline at the Month 12 time point ]
    The full PUL 2.0 version (i.e., high-level + mid-level + distal level dimensions of the PUL 2.0 tool) will be assessed over the course of the trial


Secondary Outcome Measures :
  1. The secondary efficacy endpoints include the changes from baseline for distal-level PUL 2.0 and mid-level PUL 2.0 [ Time Frame: Change from baseline at the Month 12 time point ]
    Distal-level PUL 2.0 for a subgroup of subjects with entry level scores of 2 and 3 and mid-level PUL 2.0 for a subgroup of subjects with entry level scores of 4 and 5 will be evaluated



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   10 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  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

Exclusion Criteria:

  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

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): NCT04428476


Locations
Layout table for location information
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, 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, Wisconsin
Children's Hospital Wisconsin
Milwaukee, Wisconsin, United States, 53226
Sponsors and Collaborators
Capricor Inc.
Investigators
Layout table for investigator information
Principal Investigator: Craig McDonald, MD UC Davis
Layout table for additonal information
Responsible Party: Capricor Inc.
ClinicalTrials.gov Identifier: NCT04428476    
Other Study ID Numbers: CAP-1002-DMD-02-OLE
First Posted: June 11, 2020    Key Record Dates
Last Update Posted: December 1, 2020
Last Verified: November 2020

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Capricor Inc.:
DMD, Muscular dystrophy, Duchenne
Additional relevant MeSH terms:
Layout table for MeSH terms
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked