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Open-label Extension of the HOPE-2 Trial (HOPE-2-OLE)

Primary Purpose

Duchenne Muscular Dystrophy

Status
Active
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
CAP-1002
Sponsored by
Capricor Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Duchenne Muscular Dystrophy focused on measuring DMD, Muscular dystrophy, Duchenne

Eligibility Criteria

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

Inclusion Criteria:

  1. Documented enrollment in the HOPE-2 trial and completion of trial follow-up through Month 12
  2. Willing and able to provide informed consent to participate in the trial if ≥ 18 years of age, and assent with parental or guardian informed consent if < 18 years of age
  3. Adequate venous access for intravenous CAP-1002 infusions in the judgement of the Investigator
  4. Assessed by the Investigator as willing and able to comply with the requirements of the trial

Exclusion Criteria:

  1. Planned or likely major surgery in the next 12 months after planned first infusion
  2. 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 mmol/L
  3. History of non DMD-related chronic respiratory disease including, but not limited to, asthma, bronchitis, and tuberculosis
  4. Acute respiratory illness within 60 days prior to first infusion
  5. Known hypersensitivity to dimethyl sulfoxide (DMSO) or bovine products
  6. Treatment with an investigational product ≤ 6 months prior to first infusion
  7. History, or current use, of drugs or alcohol that could impair ability to comply with participation in the trial
  8. Inability to comply with the investigational plan and follow-up visit schedule for any reason, in the judgment of the investigator

Sites / Locations

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

Arms of the Study

Arm 1

Arm Type

Other

Arm Label

Open-label arm

Arm Description

Open-label CAP-1002 will be administered to all subjects enrolled in the trial

Outcomes

Primary Outcome Measures

The primary safety endpoint is the incidence and severity of all treatment-emergent adverse events
Change from baseline in the incidence and severity of all treatment-emergent adverse events
The primary efficacy endpoint is change in upper limb function
Mean change from baseline in upper limb function assessed by full Performance of the Upper Limb test, version 2 (PUL 2.0). Items are scored on a three-point scale: 0=unable to perform the item, 1=impaired or performs with compensation, 2=performs task without compensation.

Secondary Outcome Measures

Incidence and severity of all treatment-emergent adverse events
Change from baseline in the incidence and severity of all treatment-emergent adverse events
Change from baseline in upper limb function
Mean change from baseline in upper limb function assessed by full Performance of the Upper Limb test, version 2 (PUL 2.0). Items are scored on a three-point scale: 0=unable to perform the item, 1=impaired or performs with compensation, 2=performs task without compensation.
Change from from baseline in distal-level (wrist and hand) upper limb function
Mean change from baseline in distal-level (wrist and hand) function assessed by Performance of the Upper Limb test, version 2 (PUL 2.0) for a subgroup of subjects with entry level scores of 2 and 3. Items are scored on a three-point scale: 0=unable to perform the item, 1=impaired or performs with compensation, 2=performs task without compensation.
Change from from baseline in mid-level (elbow) upper limb function
Mean change from baseline in mid-level (elbow) function assessed by Performance of the Upper Limb test, version 2 (PUL 2.0) for a subgroup of subjects with entry level scores of 4 and 5. Items are scored on a three-point scale: 0=unable to perform the item, 1=impaired or performs with compensation, 2=performs task without compensation.
Change in cardiac muscle function and structure by assessment of Left Ventricular Ejection Fraction (LVEF)
Mean change from baseline in LVEF as assessed by Cardiac Magnetic Resonance (cMRI)

Full Information

First Posted
June 9, 2020
Last Updated
April 28, 2023
Sponsor
Capricor Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT04428476
Brief Title
Open-label Extension of the HOPE-2 Trial
Acronym
HOPE-2-OLE
Official Title
Open-Label Extension of the HOPE-2 Duchenne Muscular Dystrophy Trial
Study Type
Interventional

2. Study Status

Record Verification Date
April 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
July 20, 2020 (Actual)
Primary Completion Date
February 16, 2022 (Actual)
Study Completion Date
March 2024 (Anticipated)

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
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
This Phase 2, multi-center, open-label extension trial will provide CAP-1002 to subjects that were enrolled in the HOPE-2 trial and completed 12 months of follow-up. The trial will explore the safety and efficacy of twelve intravenous administrations of CAP-1002, each separated by three months. Subjects will undergo a targeted screening during a 30-day screening period, eligible subjects will then undergo baseline safety and efficacy assessments on Day 1 prior to their first infusion of CAP-1002. Subjects will complete trial assessments at Screening; Day 1; Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, and 36. Safety and efficacy assessments will be conducted prior to CAP-1002 administration at the Day 1, Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33 trial visits, unless otherwise indicated. All CAP-1002 infusions will be conducted in an outpatient setting at the investigative site on Day 1 and Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30 and 33. Subjects will be observed in the outpatient setting for at least two hours post infusion and then discharged the same day, if medically cleared by the site Investigator.
Detailed Description
This Phase 2, multi-center, open-label extension trial will provide CAP-1002 to subjects that were enrolled in the HOPE-2 trial and completed 12 months of follow-up. The trial will explore the safety and efficacy of twelve intravenous administrations of CAP-1002, each separated by three months. Subjects will undergo a targeted screening during a 30-day screening period to determine eligibility based on protocol inclusion and exclusion criteria. Eligible subjects will undergo baseline safety and efficacy assessments on Day 1 prior to their first infusion of CAP-1002. Administration of CAP-1002 (Day 1) should occur within a maximum of 30 days following confirmation of eligibility. Subjects will complete trial assessments at Screening; Day 1; Months 3, 6, 9, 12 (± 14 days, each), 15, 18, 21, 24, 27, 30, 30, 33, and 36 (± 21 days, each). Safety and efficacy assessments will be conducted prior to CAP-1002 administration at the Day 1, Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, and 33 trial visits, unless otherwise indicated. All CAP-1002 infusions will be conducted in an outpatient setting at the investigative site on Day 1 and Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, and 33. Prior to each CAP-1002 administration, medications will be administered to the subject as determined by the Investigator based on the pre-treatment guidelines as outlined in the protocol and/or institutional protocols to minimize the risk of potential severe allergic reactions such as anaphylaxis. Subjects will be observed in the outpatient setting for at least two hours post infusion and then discharged the same day if medically cleared by the site Investigator. If clinically indicated, an unscheduled in-person visit will be performed at the investigative site with targeted assessments based on presentation of signs and symptoms following any infusion.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy
Keywords
DMD, Muscular dystrophy, Duchenne

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Model Description
Open Label Extension of the HOPE-2 Trial
Masking
None (Open Label)
Allocation
N/A
Enrollment
13 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Open-label arm
Arm Type
Other
Arm Description
Open-label CAP-1002 will be administered to all subjects enrolled in the trial
Intervention Type
Biological
Intervention Name(s)
CAP-1002
Other Intervention Name(s)
Allogeneic Cardiosphere-Derived Cells
Intervention Description
Peripheral infusion of 150 million allogeneic cardiosphere-derived cells administered every three months
Primary Outcome Measure Information:
Title
The primary safety endpoint is the incidence and severity of all treatment-emergent adverse events
Description
Change from baseline in the incidence and severity of all treatment-emergent adverse events
Time Frame
At Month 12 timepoint
Title
The primary efficacy endpoint is change in upper limb function
Description
Mean change from baseline in upper limb function assessed by full Performance of the Upper Limb test, version 2 (PUL 2.0). Items are scored on a three-point scale: 0=unable to perform the item, 1=impaired or performs with compensation, 2=performs task without compensation.
Time Frame
At Month 12 timepoint
Secondary Outcome Measure Information:
Title
Incidence and severity of all treatment-emergent adverse events
Description
Change from baseline in the incidence and severity of all treatment-emergent adverse events
Time Frame
At Month 24, Month 36 timepoint
Title
Change from baseline in upper limb function
Description
Mean change from baseline in upper limb function assessed by full Performance of the Upper Limb test, version 2 (PUL 2.0). Items are scored on a three-point scale: 0=unable to perform the item, 1=impaired or performs with compensation, 2=performs task without compensation.
Time Frame
At Month 24 and Month 36 timepoint
Title
Change from from baseline in distal-level (wrist and hand) upper limb function
Description
Mean change from baseline in distal-level (wrist and hand) function assessed by Performance of the Upper Limb test, version 2 (PUL 2.0) for a subgroup of subjects with entry level scores of 2 and 3. Items are scored on a three-point scale: 0=unable to perform the item, 1=impaired or performs with compensation, 2=performs task without compensation.
Time Frame
At Month 12, Month 24, Month 36 timepoint
Title
Change from from baseline in mid-level (elbow) upper limb function
Description
Mean change from baseline in mid-level (elbow) function assessed by Performance of the Upper Limb test, version 2 (PUL 2.0) for a subgroup of subjects with entry level scores of 4 and 5. Items are scored on a three-point scale: 0=unable to perform the item, 1=impaired or performs with compensation, 2=performs task without compensation.
Time Frame
At Month 12, Month 24, Month 36 timepoint
Title
Change in cardiac muscle function and structure by assessment of Left Ventricular Ejection Fraction (LVEF)
Description
Mean change from baseline in LVEF as assessed by Cardiac Magnetic Resonance (cMRI)
Time Frame
At Month 24, Month 36 timepoint

10. Eligibility

Sex
All
Minimum Age & Unit of Time
10 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Documented enrollment in the HOPE-2 trial and completion of trial follow-up through Month 12 Willing and able to provide informed consent to participate in the trial if ≥ 18 years of age, and assent with parental or guardian informed consent if < 18 years of age Adequate venous access for intravenous CAP-1002 infusions in the judgement of the Investigator Assessed by the Investigator as willing and able to comply with the requirements of the trial Exclusion Criteria: Planned or likely major surgery in the next 12 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 mmol/L History of non DMD-related chronic respiratory disease including, but not limited to, asthma, bronchitis, and tuberculosis Acute respiratory illness within 60 days prior to first infusion Known hypersensitivity to dimethyl sulfoxide (DMSO) or bovine products Treatment with an 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Craig McDonald, MD
Organizational Affiliation
UC Davis
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Mark Awadalla
Organizational Affiliation
Capricor Inc.
Official's Role
Study Director
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
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
Children's Hospital Wisconsin
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States

12. IPD Sharing Statement

Learn more about this trial

Open-label Extension of the HOPE-2 Trial

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