Halt cardiomyOPathy progrEssion in Duchenne (HOPE-OLE)

NCT06304064 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: CAP-1002-DMD-03
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 2

Brief Summary

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.

Conditions

Duchenne Muscular Dystrophy

Keywords

Duchenne Muscular Dystrophy; Cardiomyopathy; Duchenne

Outcome Measures

Primary Outcomes (5)

• 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

• 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

• 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

Study Arms (1)

Allogeneic Cardiosphere-Derived Cells (CAP-1002)

EXPERIMENTAL

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)

Interventions

Allogeneic Cardiosphere-Derived Cells (CAP-1002) BIOLOGICAL

Intravenous infusion delivery of Allogeneic Cardiosphere-Derived Cells (CAP-1002; 75 million CDCs)

Also known as: CAP-1002

Allogeneic Cardiosphere-Derived Cells (CAP-1002)

Eligibility Criteria

• Age: 12 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Documented enrollment in the Usual Care Treatment Group of the HOPE-Duchenne trial and completion of trial follow-up through Month 12.
• 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.
• Adequate venous access for intravenous CAP-1002 infusions and routine blood collections in the judgement of the Investigator.
• Assessed by the Investigator as willing and able to comply with the requirements of the trial.

You may not qualify if:

• Left ventricular ejection fraction (LVEF) \< 35 percent (%) within 6 months of screening.
• Planned or likely major surgery in the next 6 months after planned first infusion.
• 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.
• History of non DMD-related chronic respiratory disease including, but not limited to, asthma, bronchitis, and tuberculosis.
• Acute respiratory illness within 30 days prior to screening.
• Known hypersensitivity to dimethyl sulfoxide (DMSO) or bovine products.
• Treatment with investigational product \<= 6 months prior to first infusion.
• History, or current use, of drugs or alcohol that could impair ability to comply with participation in the trial.
• Inability to comply with the investigational plan and follow-up visit schedule for any reason, in the judgment of the investigator.

Sponsors & Collaborators

• Capricor Inc.: lead

Study Sites (2)

University of Florida

Gainesville, Florida, 32610, United States

Location

Cincinnati Children's Medical Center

Cincinnati, Ohio, 45229, United States

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne; Cardiomyopathies

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heart Diseases; Cardiovascular Diseases

Results Point of Contact

• Title: Study Director
• Organization: Capricor Inc.

mawadalla@capricor.com

858-727-1755

Study Officials

• Mark Awadalla

  Capricor Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 5, 2024
• First Posted: March 12, 2024
• Study Start: June 21, 2018
• Primary Completion: March 6, 2019
• Study Completion: March 6, 2019
• Last Updated: April 24, 2024
• Results First Posted: April 24, 2024

Record last verified: 2024-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)