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

Nervous System Diseases Clinical Trial

— HOPE-2  

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03406780
• Other study ID #: CAP-1002-DMD-02
• Status: Completed
• Phase: Phase 2
• Start date: March 4, 2018
• Est. completion date: March 10, 2020

Study information

• Verified date: June 2020
• Source: Capricor Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 18
• Est. completion date: March 10, 2020
• Est. primary completion date: March 10, 2020
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 10 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Genetic Diseases, Inborn
• Genetic Diseases, X-Linked
• Muscular Diseases
• Muscular Disorders, Atrophic
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne
• Nervous System Diseases
• Neuromuscular Diseases

Intervention

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.

Drug:
• Placebo
Placebo

Locations

Country	Name	City	State
United States	Children's Hospital Colorado	Aurora	Colorado
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	Children's Hospital Wisconsin	Milwaukee	Wisconsin
United States	Nemours Children's Hospital	Orlando	Florida
United States	University of California, Davis	Sacramento	California
United States	Washington University	Saint Louis	Missouri
United States	University of Utah	Salt Lake City	Utah

Sponsors (1)

Lead Sponsor	Collaborator
Capricor Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in the mid-level (elbow) dimension of the Performance of the Upper Limb (PUL)	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.	Month 12
Secondary	Change in the mid-level (elbow) dimension of the PUL	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.	Months 3, 6, and 9
Secondary	Change in regional systolic left ventricular wall thickening as assessed by cardiac MRI	Systolic thickening is thought to be a principal mechanism of cardiac output generation in people with DMD.	Months 6 and 12

External Links

•   Company website