HOPE-2: A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

• Sponsor: Capricor Inc. (Industry)
• Overall Status: Completed
• CT.gov ID: NCT03406780
• Collaborator: (none)
• Enrollment: 18
• Locations: 7
• Arms: 2
• Actual Duration (Months): 24.2
• Patients Per Site: 2.6
• Patients Per Site Per Month: 0.1

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Muscular Dystrophies; Muscular Dystrophy, Duchenne; Muscular Disorders, Atrophic; Muscular Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn	Biological: CAP-1002; Drug: Placebo	Phase 2

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.

Study Design

• Study Type: Interventional
• Actual Enrollment: 18 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled Trial Evaluating the Safety and Efficacy of Intravenous Delivery of Allogeneic Cardiosphere-Derived Cells in Subjects With Duchenne Muscular Dystrophy
• Actual Study Start Date: Mar 4, 2018
• Actual Primary Completion Date: Mar 10, 2020
• Actual Study Completion Date: Mar 10, 2020

Arms and Interventions

Arm	Intervention/Treatment
Experimental: CAP-1002 Patients will receive 150 million cardiosphere-derived cells (CDCs) via intravenous infusion every 3 months for a total of 4 doses.	Biological: CAP-1002; The active pharmaceutical ingredient in CAP-1002 is Cardiosphere-Derived Cells (CDCs). CDCs are known to secrete numerous bioactive elements (growth factors, exosomes) which impact the therapeutic benefits of the cell-based therapy. The mechanism of action is the composite ability to be immunomodulatory, anti-fibrotic and regenerative.; Other Names: Cardiosphere-Derived Cells CDCs
Placebo Comparator: Placebo Patients will receive a placebo solution via intravenous infusion every 3 months for a total of 4 doses.	Drug: Placebo; Placebo

Outcome Measures

Primary Outcome Measures

1. 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

1. 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.

2. 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.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 10 Years and Older
• Sexes Eligible for Study: Male
• Accepts Healthy Volunteers: No

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

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	University of California, Davis	Sacramento	California	United States	95817
2	Children's Hospital Colorado	Aurora	Colorado	United States	80045
3	Nemours Children's Hospital	Orlando	Florida	United States	32827
4	Washington University	Saint Louis	Missouri	United States	63110
5	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio	United States	45229
6	University of Utah	Salt Lake City	Utah	United States	84112
7	Children's Hospital Wisconsin	Milwaukee	Wisconsin	United States	53226

Sponsors and Collaborators

• Capricor Inc.

Investigators

• Principal Investigator: Craig McDonald, MD, University of California, Davis

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

• Company website

Publications

None provided.

• Responsible Party: Capricor Inc.
• ClinicalTrials.gov Identifier: NCT03406780
• Other Study ID Numbers: CAP-1002-DMD-02
• First Posted: Jan 23, 2018
• Last Update Posted: Jun 5, 2020
• Last Verified: Jun 1, 2020

• Individual Participant Data (IPD) Sharing Statement: Undecided
• Plan to Share IPD: Undecided
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Capricor Inc.: Duchenne Muscular Dystrophy; Cell Therapy; Performance of the Upper Limb; Pulmonary Function; Ambulatory; Non-Ambulatory; Glucocorticoids
• Additional relevant MeSH terms: Muscular Dystrophies; Muscular Dystrophy, Duchenne; Muscular Diseases; Muscular Disorders, Atrophic; Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked

Study Results

No Results Posted as of Jun 5, 2020