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

Primary Purpose

Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic

Status

Completed

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

CAP-1002

Placebo

About this trial

This is an interventional treatment trial for Muscular Dystrophies focused on measuring Duchenne Muscular Dystrophy, Cell Therapy, Performance of the Upper Limb, Pulmonary Function, Ambulatory, Non-Ambulatory, Glucocorticoids

Eligibility Criteria

10 Years - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

Inclusion Criteria:

Genetically confirmed DMD
Reduced upper arm strength as measured by the Performance of Upper Limb
Reduced ability to walk/run (if ambulatory)
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
Current and up-to-date immunizations

Exclusion Criteria:

Left ventricular ejection fraction < 35%
BMI > 45
Ambulant if ≥ 18 years of age
Exon 44 skip-amenable mutation(s) in the DMD gene
Deletion mutation(s) encompassing exons 3-7 of the DMD gene
Percent-predicted forced vital capacity (FVC) < 35%
Chronic respiratory disease not related to DMD (for example, asthma, bronchitis, and tuberculosis)
History of diabetes requiring treatment with metformin or insulin within 3 months prior to randomization
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
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
Treatment with idebenone within 3 months prior to randomization
Treatment with a cell therapy product within 12 months prior to randomization
Treatment with an investigational product within 6 months prior to randomization

Sites / Locations

University of California, Davis
Children's Hospital Colorado
Nemours Children's Hospital
Washington University
Cincinnati Children's Hospital Medical Center
University of Utah
Children's Hospital Wisconsin

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

CAP-1002

Placebo

Arm Description

Patients will receive 150 million cardiosphere-derived cells (CDCs) via intravenous infusion every 3 months for a total of 4 doses.

Patients will receive a placebo solution via intravenous infusion every 3 months for a total of 4 doses.

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Change in the mid-level (elbow) dimension of the PUL

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.

Full Information

NCT ID

NCT03406780

First Posted

January 15, 2018

Last Updated

June 3, 2020

Sponsor

Capricor Inc.

1. Study Identification

Unique Protocol Identification Number

NCT03406780

Brief Title

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

Acronym

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

Study Type

Interventional

2. Study Status

Record Verification Date

June 2020

Overall Recruitment Status

Completed

Study Start Date

March 4, 2018 (Actual)

Primary Completion Date

March 10, 2020 (Actual)

Study Completion Date

March 10, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Capricor Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. 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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

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

Keywords

Duchenne Muscular Dystrophy, Cell Therapy, Performance of the Upper Limb, Pulmonary Function, Ambulatory, Non-Ambulatory, Glucocorticoids

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

18 (Actual)

8. Arms, Groups, and Interventions

Arm Title

CAP-1002

Arm Type

Experimental

Arm Description

Patients will receive 150 million cardiosphere-derived cells (CDCs) via intravenous infusion every 3 months for a total of 4 doses.

Arm Title

Placebo

Arm Type

Placebo Comparator

Arm Description

Patients will receive a placebo solution via intravenous infusion every 3 months for a total of 4 doses.

Intervention Type

Biological

Intervention Name(s)

CAP-1002

Other Intervention Name(s)

Cardiosphere-Derived Cells, CDCs

Intervention Description

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.

Intervention Type

Drug

Intervention Name(s)

Placebo

Intervention Description

Placebo

Primary Outcome Measure Information:

Title

Change in the mid-level (elbow) dimension of the Performance of the Upper Limb (PUL)

Description

Time Frame

Month 12

Secondary Outcome Measure Information:

Title

Change in the mid-level (elbow) dimension of the PUL

Description

Time Frame

Months 3, 6, and 9

Title

Change in regional systolic left ventricular wall thickening as assessed by cardiac MRI

Description

Systolic thickening is thought to be a principal mechanism of cardiac output generation in people with DMD.

Time Frame

Months 6 and 12

10. Eligibility

Sex

Male

Minimum Age & Unit of Time

10 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Genetically confirmed DMD Reduced upper arm strength as measured by the Performance of Upper Limb Reduced ability to walk/run (if ambulatory) 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 Current and up-to-date immunizations Exclusion Criteria: Left ventricular ejection fraction < 35% BMI > 45 Ambulant if ≥ 18 years of age Exon 44 skip-amenable mutation(s) in the DMD gene Deletion mutation(s) encompassing exons 3-7 of the DMD gene Percent-predicted forced vital capacity (FVC) < 35% Chronic respiratory disease not related to DMD (for example, asthma, bronchitis, and tuberculosis) History of diabetes requiring treatment with metformin or insulin within 3 months prior to randomization 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 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 Treatment with idebenone within 3 months prior to randomization Treatment with a cell therapy product within 12 months prior to randomization Treatment with an investigational product within 6 months prior to randomization

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Craig McDonald, MD

Organizational Affiliation

University of California, Davis

Official's Role

Principal Investigator

Facility Information:

Facility Name

University of California, Davis

City

Sacramento

State/Province

California

ZIP/Postal Code

95817

Country

United States

Facility Name

Children's Hospital Colorado

City

Aurora

State/Province

Colorado

ZIP/Postal Code

80045

Country

United States

Facility Name

Nemours Children's Hospital

City

Orlando

State/Province

Florida

ZIP/Postal Code

32827

Country

United States

Facility Name

Washington University

City

Saint Louis

State/Province

Missouri

ZIP/Postal Code

63110

Country

United States

Facility Name

Cincinnati Children's Hospital Medical Center

City

Cincinnati

State/Province

Ohio

ZIP/Postal Code

45229

Country

United States

Facility Name

University of Utah

City

Salt Lake City

State/Province

Utah

ZIP/Postal Code

84112

Country

United States

Facility Name

Children's Hospital Wisconsin

City

Milwaukee

State/Province

Wisconsin

ZIP/Postal Code

53226

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Undecided

Citations:

PubMed Identifier

35279258

Citation

McDonald CM, Marban E, Hendrix S, Hogan N, Ruckdeschel Smith R, Eagle M, Finkel RS, Tian C, Janas J, Harmelink MM, Varadhachary AS, Taylor MD, Hor KN, Mayer OH, Henricson EK, Furlong P, Ascheim DD, Rogy S, Williams P, Marban L; HOPE-2 Study Group. Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial. Lancet. 2022 Mar 12;399(10329):1049-1058. doi: 10.1016/S0140-6736(22)00012-5.

Results Reference

derived

Links:

URL

http://capricor.com

Description

Company website

Learn more about this trial

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

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