A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

Phase 2

Completed

• Conditions: Muscular Dystrophies; Genetic Diseases, Inborn; Muscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Muscular Dystrophy, Duchenne; Neuromuscular Diseases; Muscular Disorders, Atrophic
• Interventions: Drug: Placebo; Biological: CAP-1002

• Registration Number: NCT03406780
• Lead Sponsor: Capricor Inc.

Brief Summary

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.

Detailed Description

* 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.

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations
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Related News

Study Timeline

• Study First Submitted: 2018-01-15
• Study First Submitted QC: 2018-01-19
• Study First Posted: 2018-01-23
• Study Start: 2018-03-04
• Primary Completion: 2020-03-10
• Study Completion: 2020-03-10
• Status Verified: 2020-06
• Last Update Submitted: 2020-06-03
• Last Update Posted: 2020-06-05

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 18

Inclusion Criteria

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

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Exclusion Criteria

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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	Patients will receive a placebo solution via intravenous infusion every 3 months for a total of 4 doses.
CAP-1002	CAP-1002	Patients will receive 150 million cardiosphere-derived cells (CDCs) via intravenous infusion every 3 months for a total of 4 doses.

Primary Outcome Measures

Name	Time	Method
Change in the mid-level (elbow) dimension of the Performance of the Upper Limb (PUL)	Month 12	The PUL includes functional tasks that relate to activities of daily living that are very important for quality of life. The PUL has been validated for the assessment of upper limb motor function in individuals with DMD.

Secondary Outcome Measures

Name	Time	Method
Change in the mid-level (elbow) dimension of the PUL	Months 3, 6, and 9	The PUL includes functional tasks that relate to activities of daily living that are very important for quality of life. The PUL has been validated for the assessment of upper limb motor function in individuals with DMD.
Change in regional systolic left ventricular wall thickening as assessed by cardiac MRI	Months 6 and 12	Systolic thickening is thought to be a principal mechanism of cardiac output generation in people with DMD.

Trial Locations

Locations (7)

Nemours Children's Hospital; 🇺🇸 Orlando, Florida, United States

University of Utah; 🇺🇸 Salt Lake City, Utah, United States

Children's Hospital Wisconsin; 🇺🇸 Milwaukee, Wisconsin, United States

University of California, Davis; 🇺🇸 Sacramento, California, United States

Children's Hospital Colorado; 🇺🇸 Aurora, Colorado, United States

Washington University; 🇺🇸 Saint Louis, Missouri, United States

Cincinnati Children's Hospital Medical Center; 🇺🇸 Cincinnati, Ohio, United States