Halt cardiomyOPathy progrEssion in Duchenne (HOPE-OLE) (HOPE-OLE)

Open-Label Extension of the Halt Cardiomyopathy Progression in Duchenne (HOPE-Duchenne) Trial (CAP-1002-DMD-03)

This Phase 2, multi-center, open-label extension trial will provide CAP-1002 to participants who were randomized to the Usual Care treatment group of the HOPE-Duchenne study (NCT02485938) and completed 12 months of follow-up.

The trial will assess the safety and efficacy of two intravenous administrations of CAP-1002, each separated by three months.

Study Overview

• Status: Completed
• Conditions: Duchenne Muscular Dystrophy
• Intervention / Treatment: Biological: Allogeneic Cardiosphere-Derived Cells (CAP-1002)

Detailed Description

Participants with documented enrollment in the Usual Care treatment group of the HOPE-Duchenne study and completion of study follow-up through Month 12 were eligible for this study.

Participants 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.

All CAP-1002 infusions will be conducted in an outpatient setting at the investigative site on Day 1 and at Month 3. Participants 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.

• Study Type: Interventional
• Enrollment (Actual): 8
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Florida
    • Gainesville, Florida, United States, 32610
      • University of Florida
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Cincinnati Children's Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Documented enrollment in the Usual Care Treatment Group of the HOPE-Duchenne trial and completion of trial follow-up through Month 12.
2. Willing and able to provide informed consent to participate in the trial if greater than or equal to (>=) 18 years of age, and assent with parental or guardian informed consent if less than (<) 18 years of age.
3. Adequate venous access for intravenous CAP-1002 infusions and routine blood collections 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. Left ventricular ejection fraction (LVEF) < 35 percent (%) within 6 months of screening.
2. Planned or likely major surgery in the next 6 months after planned first infusion.
3. 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 millimoles per liter (mmol/L) at screening.
4. History of non DMD-related chronic respiratory disease including, but not limited to, asthma, bronchitis, and tuberculosis.
5. Acute respiratory illness within 30 days prior to screening.
6. Known hypersensitivity to dimethyl sulfoxide (DMSO) or bovine products.
7. Treatment with investigational product <= 6 months prior to first infusion.
8. History, or current use, of drugs or alcohol that could impair ability to comply with participation in the trial.
9. Inability to comply with the investigational plan and follow-up visit schedule for any reason, in the judgment of the investigator.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Allogeneic Cardiosphere-Derived Cells (CAP-1002); All participants who were randomized to the Usual Care Treatment Group and completed 12 months of follow-up in the HOPE-Duchenne trial (NCT02485938), will receive CAP-1002 intravenous infusion on Day 1 and at Month 3 in the current study.	Biological: Allogeneic Cardiosphere-Derived Cells (CAP-1002); Intravenous infusion delivery of Allogeneic Cardiosphere-Derived Cells (CAP-1002; 75 million CDCs); Other Names: CAP-1002

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Hypersensitivity Reactions	Hypersensitivity reaction is defined as a clinical syndrome including, but not limited to, fever, leukocytosis, or rash with onset <= 2 hours post-infusion and lasting < 24 hours, in the absence of clinical signs of concomitant infection.	From Day 1 up to Month 6
Number of Participants Experiencing Acute Respiratory Decompensation	Acute respiratory decompensation is defined as an unexplained rapid deterioration of the participant's condition with increasing shortness of breath requiring oxygen supplementation. Acute respiratory decompensation within 2 hours following investigational product (IP) administration will be reported.	2 hours post-dose on Day 1 and Month 3
All-cause Mortality	Number of deaths due to any cause will be reported.	From Day 1 up to Month 6
Number of Treatment-emergent Adverse Events (TEAEs) Related to Investigational Product or Administration and Serious Adverse Events (SAEs)	An adverse events (AEs) is any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. TEAEs are defined as AEs occurring after the initiation of the IV catheter placement for the initial dose of IP. TEAEs related to investigational product or administration are reported for this outcome measure. A SAE is defined as an AE that results in any of the following outcomes: Death; life-threatening adverse event; Inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions; congenital anomaly/birth defect.	From Day 1 up to Month 6
Number of Participants With Immune Sensitization Syndrome	Immune sensitization syndrome shall be defined as: (a) clinical signs and symptoms consistent with systemic inflammation (e.g., fever, leukocytosis, rash, or arthralgia) with onset >= 24 hours post infusion and the absence of clinical signs of concomitant infection, and (b) elevation of anti-human leukocyte antigen (HLA) antibodies against the donor cells (i.e., DSAs), detected <= 30 days following onset of syndrome, of (i) >= 2000 mean fluorescent intensity (MFI) if baseline MFI <= 1000, or (ii) >= 2 times baseline otherwise.	From Day 1 up to Month 6

Collaborators and Investigators

• Sponsor: Capricor Inc.
• Investigators: Study Director: Mark Awadalla, Capricor Inc.

Study record dates

Study Major Dates

• Study Start (Actual): June 21, 2018
• Primary Completion (Actual): March 6, 2019
• Study Completion (Actual): March 6, 2019

Study Registration Dates

• First Submitted: March 5, 2024
• First Submitted That Met QC Criteria: March 5, 2024
• First Posted (Actual): March 12, 2024

Study Record Updates

• Last Update Posted (Actual): April 24, 2024
• Last Update Submitted That Met QC Criteria: March 29, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Keywords: Duchenne Muscular Dystrophy; Cardiomyopathy; Duchenne
• Additional Relevant MeSH Terms: Heart Diseases; Cardiovascular Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Muscular Disorders, Atrophic; Muscular Dystrophies; Cardiomyopathies; Muscular Dystrophy, Duchenne
• Other Study ID Numbers: CAP-1002-DMD-03

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No