Open-label Extension of the HOPE-2 Trial

Phase 2

Active, not recruiting

• Conditions: Duchenne Muscular Dystrophy
• Interventions: Biological: Deramiocel (CAP-1002)

• Registration Number: NCT04428476
• Lead Sponsor: Capricor Inc.

Brief Summary

This Phase 2, multi-center, open-label extension trial will provide deramiocel (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 twenty intravenous administrations of deramiocel, 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 deramiocel.

Subjects will complete trial assessments at Screening; Day 1; Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48, 51, 54, 57, and 60. Safety and efficacy assessments will be conducted prior to deramiocel administration at the Day 1, Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48, 51, 54, and 57 trial visits, unless otherwise indicated.

All deramiocel 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, 33, 36, 39, 42, 45, 48, 51, 54, and 57. 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 deramiocel (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 twenty intravenous administrations of deramiocel, 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 deramiocel. Administration of deramiocel (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, 36, 39, 42, 45, 48, 51, 54, 57, and 60 (± 21 days, each). Safety and efficacy assessments will be conducted prior to deramiocel administration at the Day 1, Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48, 51, 54, and 57 trial visits, unless otherwise indicated.

All deramiocel 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, 33, 36, 39, 42, 45, 48, 51, 54, and 57. Prior to each deramiocel 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.

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: 2020-06-09
• Study First Submitted QC: 2020-06-09
• Study First Posted: 2020-06-11
• Study Start: 2020-07-20
• Primary Completion: 2022-02-16
• Disposition First Submitted: 2023-02-03
• Disposition First Submitted QC: 2023-02-03
• Disposition First Posted: 2023-02-13
• Status Verified: 2024-11
• Last Update Submitted: 2024-11-05
• Last Update Posted: 2024-11-11
• Study Completion: 2026-03

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 13

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 deramiocel (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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Open-label arm	Deramiocel (CAP-1002)	Open-label deramiocel (CAP-1002) will be administered to all subjects enrolled in the trial

Primary Outcome Measures

Name	Time	Method
The primary efficacy endpoint is change in upper limb function	At Month 12 timepoint	Mean change from baseline in upper limb function assessed by Performance of the Upper Limb test, version 2 (PUL 2.0) Total Score. 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.
The primary safety endpoint is the incidence and severity of all treatment-emergent adverse events	At Month 12 timepoint	Change from baseline in the incidence and severity of all treatment-emergent adverse events

Secondary Outcome Measures

Name	Time	Method
Incidence and severity of all treatment-emergent adverse events	At Month 24, Month 36, Month 48, and Month 60 timepoint	Change from baseline in the incidence and severity of all treatment-emergent adverse events
Change from baseline in upper limb function	At Month 24, Month 36, Month 48, and Month 60 timepoint	Mean change from baseline in upper limb function assessed by Performance of the Upper Limb test, version 2 (PUL 2.0) Total Score. 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	At Month 12, Month 24, Month 36, Month 48, and Month 60 timepoint	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 baseline in mid-level (elbow) upper limb function	At Month 12, Month 24, Month 36, Month 48, and Month 60 timepoint	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)	At Month 24, Month 36, Month 48, and Month 60 timepoint	Mean change from baseline in LVEF as assessed by Cardiac Magnetic Resonance (cMRI)
Change in cardiac muscle function and structure by assessment of Left Ventricular End Systolic Volumes-Indexed (ESVI)	At Month 24, Month 36, Month 48, and Month 60 timepoint	Mean change from baseline in ESVI as assessed by Cardiac Magnetic Resonance (cMRI)
Change in cardiac muscle function and structure by assessment of Left Ventricular End Diastolic Volumes-Indexed (EDVI)	At Month 24, Month 36, Month 48, and Month 60 timepoint	Mean change from baseline in EDVI as assessed by Cardiac Magnetic Resonance (cMRI)
Change in cardiac muscle function and structure by assessment of mass	At Month 24, Month 36, Month 48, and Month 60 timepoint	Mean change from baseline in mass as assessed by Cardiac Magnetic Resonance (cMRI)
Change in cardiac muscle function and structure by assessment of unindexed volumes	At Month 24, Month 36, Month 48, and Month 60 timepoint]	Mean change from baseline in unindexed volumes as assessed by Cardiac Magnetic Resonance (cMRI)
Change in cardiac muscle function and structure by assessment of cardiac output	At Month 24, Month 36, Month 48, and Month 60 timepoint	Mean change from baseline in cardiac output as assessed by Cardiac Magnetic Resonance (cMRI)
Change in cardiac muscle function and structure by assessment of wall thickening percentage	At Month 24, Month 36, Month 48, and Month 60 timepoint	Mean change from baseline in wall thickening percentage as assessed by Cardiac Magnetic Resonance (cMRI)
Change in cardiac muscle function and structure by assessment of end diastolic wall thickness	At Month 24, Month 36, Month 48, and Month 60 timepoint	Mean change from baseline in end diastolic wall thickness as assessed by Cardiac Magnetic Resonance (cMRI)
Change in cardiac muscle function and structure by assessment of end systolic wall thickness	At Month 24, Month 36, Month 48, and Month 60 timepoint	Mean change from baseline in end systolic wall thickness as assessed by Cardiac Magnetic Resonance (cMRI)

Trial Locations

Locations (5)

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

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