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Muscular DystrophiesPhase 3HOPE-3NCT05126758

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

A Phase 3 study testing Deramiocel (CAP-1002) in people with Muscular Dystrophies and Muscular Dystrophy, Duchenne, run by Capricor Inc. at 20 sites in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
Search all trials

Trial summary

HOPE-3 is a two cohort, Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating the efficacy and safety of a cell therapy called deramiocel (CAP-1002) in study participants with Duchenne muscular dystrophy (DMD) and impaired skeletal muscle function. Read more about this study

Who is it for?
Ages 10 years and older, men only
What is being tested?
Deramiocel (CAP-1002)
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
106 participants
Where
20 sitesUnited States
Study dates
22 Jun 2022to Mar 2027
Phase
Phase 3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 10 years and older
  • Men only
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Male subjects at least 10 years of age at time of consent who are willing and able to provide informed consent to participate in the trial if ≥ 18 years of age or assent with parental or guardian informed consent if < 18 years of age. If a third-party caregiver is involved, they must provide informed consent.
  • Diagnosis of DMD based on clinical and phenotypic manifestations consistent with DMD (e.g., family history of DMD, elevated creatine kinase, dystrophin muscle biopsy, calf pseudohypertrophy, history of Gowers' sign, and gait impairment before 7 years of age) as confirmed by the Investigator.
  • Confirmatory genetic testing performed to have reached a diagnosis of DMD at any time in the past or currently performed at a Clinical Laboratory Improvement Amendments (CLIA)-certified laboratory or equivalent.
  • Performance of the Upper Limb test (PUL) entry item scores 2-6 and total PUL score less than or equal to 40. For Cohort A only: enrollment of patients with PUL entry score 6, Exon 44 skipping amenable, and/or Exon 3 through 7 deletions will be capped at no more than 10% of the total study population (approximately 6 patients with these characteristics).
  • Reduced ability to walk/run (if ambulatory): subjects must take more than 10 seconds for the 10-meter walk/run (i.e., velocity < 1 meter/second).
  • If non-ambulatory, loss of independent ambulation between 10th and 18th year birthday (standing unassisted or ability to take, at most, several steps independently is not considered ambulation). Subjects who are considered non-ambulatory between the ages of 9 and10 may be enrolled with prior approval from the sponsor.
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  • Receiving standard of care therapy at an experienced, multidisciplinary DMD center as evidenced by regular cardiac and pulmonary monitoring, systemic glucocorticoid treatment, and at-home range of motion exercises.
  • Treatment with systemic glucocorticoids for at least 12 months and at a stable dose at least 6 months prior to study participation, except for either weight-based dose adjustment or a decrease in steroid dose of ≤ 10% for toxicity. For patients on chronic deflazacort, treatment with an equivalent dose of prednisone or prednisolone for a period of ≤ 30 days to bridge lack of availability of deflazacort during the 6 months prior to randomization is acceptable.
  • Current and up-to-date immunizations according to children and adolescent Centers for Disease Control and Prevention immunization schedule at the discretion of the Investigator.
  • Adequate venous access for parenteral IP infusions and routine blood collection.
  • Assessed by the Investigator as willing and able to comply with the requirements of the trial.
  • Sexually active subjects and their partners who are fertile must agree to use effective method(s) of contraception.

Who cannot join

  • Left ventricular ejection fraction (LVEF) less than or equal to 35% prior to randomization.
  • Elbow-flexion contractures > 30° in both extremities.
  • Body mass index (BMI) > 45.
  • Percent predicted forced vital capacity (FVC%) < 35% within 6 months prior to randomization.
  • Inability to perform consistent PUL 2.0 measurement within ± 2 points without shoulder domain or within ± 3 points with shoulder domain during paired testing at screening.
  • Risk of near-term respiratory decompensation in the judgment of the Investigator, or the need for initiation of day and night non-invasive ventilator support as defined by serum bicarbonate ≥ 29 mmol/L at screening.
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  • History of non DMD-related chronic respiratory disease requiring ongoing or intermittent treatment, including, but not limited to, asthma, bronchitis, and tuberculosis.
  • Acute respiratory illness within 30 days prior to screening and during screening.
  • Initiation of nocturnal non-invasive ventilation within 30 days prior to screening.
  • Planned or anticipated thoracic or spinal surgery within the 6 months following randomization.
  • Planned or anticipated lower extremity surgery within the 6 months following randomization, if ambulatory.
  • Known hypersensitivity to dimethyl sulfoxide (DMSO) or bovine products.
  • Initiation of treatment with metformin or insulin within 3 months prior to randomization.
  • Initiation of treatment with an FDA-approved exon skipping therapy for the treatment of DMD and/or non-weight based adjustments within 12 months prior to randomization.
  • Treatment with human growth hormone within 3 months prior to randomization, unless on a stable dose allowing for weight-based dose adjustments (as determined by the site Investigator) for at least 24 months prior to randomization.
  • Treatment with a cell therapy product within 12 months prior to randomization; any prior exposure to deramiocel will be excluded.
  • Treatment with an investigational product within 6 months prior to randomization.
  • History, or current use, of drugs or alcohol that could impair the 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.
  • Inability to undergo a cardiac MRI. For Cohort B Only - Subjects with a known hypersensitivity to gadolinium may forgo the LGE assessment but must complete a cardiac MRI without contrast. For Cohort B Only - Subjects who are unable to tolerate gadolinium due to renal insufficiency as measured by an estimated Glomerular Filtration Rate (eGFR) less than 60 mL/min/1.73 m2 may forgo the LGE assessment but must complete a cardiac MRI without contrast.
  • For Cohort B: Subjects with PUL entry score 6, Exon 44 skipping amenable, or Exon 3 through 7 deletions are excluded from participation.

Trial locations and contacts

20 sites in 1 country.

20 sites in 1 country

United States20 sites

  • Akron Children's Hospital

    Akron, Ohio, 44308

  • Rare Disease Research, LLC

    Atlanta, Georgia, 30329

  • Children's Hospital Colorado

    Aurora, Colorado, 80045

  • Boston Children's Hospital

    Boston, Massachusetts, 02115

  • University of Virginia Children's Hospital

    Charlottesville, Virginia, 22903

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229

  • University of Missouri Health Care

    Columbia, Missouri, 65212

  • Children's Health Specialty Care Pavilion

    Dallas, Texas, 75207

  • Rare Disease Research NC LLC

    Hillsborough, North Carolina, 27278

  • University of Iowa Hospitals and Clinics

    Iowa City, Iowa, 52242

  • UCSD Altman Clinical and Translational Research Institute

    La Jolla, California, 92037

  • Arkansas Children's Hospital

    Little Rock, Arkansas, 72202

  • Children's Hospital of Los Angeles, Division of Neurology

    Los Angeles, California, 90027

  • Children's Wisconsin

    Milwaukee, Wisconsin, 53226

  • Phoenix Children's Hospital

    Phoenix, Arizona, 85016

  • University of California, Davis

    Sacramento, California, 95817

  • University of Utah Hospital

    Salt Lake City, Utah, 84112

  • Seattle Children's

    Seattle, Washington, 98105

  • Saint Louis Children's Hospital

    St Louis, Missouri, 63110

Other trials in United States

About this study

Official title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial Evaluating the Efficacy and Safety of Human Allogeneic Cardiosphere-Derived Cells for the Treatment of Duchenne Muscular Dystrophy

HOPE-3 is a two cohort, Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating the efficacy and safety of a cell therapy called deramiocel (CAP-1002) in study participants with Duchenne muscular dystrophy (DMD) and impaired skeletal muscle function. Non-ambulatory and ambulatory boys and young men who meet eligibility criteria will be randomly assigned to receive either deramiocel or placebo every 3 months for a total of 4 doses during the first 12 months of the study. All participants will be eligible to receive 4 doses of deramiocel for an additional 12 months as part of an open-label extended assessment period. After completion of the first open-label extension (Months 12-24), subjects who have completed Month 24 are eligible to continue onto a Long-Term Open-Label Extension period that will provide treatment with deramiocel until commercial availability, or until sponsor's decision to terminate the trial, or the participant withdraws consent.

Read the detailed description

Up to 102 eligible study subjects will participate in this two cohort study. Cohort A will enroll approximately 58 subjects randomized in a 1:1 ratio (active:placebo). Subjects randomized to the active treatment group in Cohort A will receive CAP-1002A (deramiocel manufactured at Capricor's manufacturing facility in Los Angeles, CA). Once Cohort A enrollment is completed, Cohort B enrollment will begin. Cohort B will enroll approximately 44 participants randomized in a 1:1 ratio (active:placebo). Subjects randomized to the active treatment group in Cohort B will receive CAP-1002B (deramiocel manufactured at Capricor's manufacturing facility in San Diego, CA). Both cohorts will include visits at Screening, Baseline/Day 1, Month 1, and Months 3, 6, 9, and 12. Subjects will receive IV infusions of deramiocel or placebo on Day 1 and Months 3, 6, and 9. All subjects will then be eligible to receive additional IV infusions of deramiocel at Month 12, 15, 18, and 21 as part of the open-label phase of the study. All subjects who complete the first open-label extension phase of the study will be eligible to receive additional IV infusions of deramicoel every 3 months in a Long Term Open-Label Extension phase until commercial availability of deramiocel, or until sponsor's decision to terminate the trial, or the participant withdraws consent.

A primary analysis of efficacy and safety will be performed on the double-blind placebo-controlled phase of the study for both Cohort A and Cohort B combined at Month 12 following 4 administrations of deramiocel or placebo.

The primary efficacy endpoint is the mean change from baseline in upper limb function as assessed by Performance of the Upper Limb test, version 2.0 [PUL 2.0] Total Score at the 12-month time point. Secondary endpoints evaluated at the 12-month time point include assessment of changes in cardiac muscle function and structure by cardiac magnetic resonance imaging [cMRI], changes in hand-to-mouth function [eat 10-bites assessed by the Duchenne Video Assessment (DVA)], quality of life assessments, and biomarker analysis for creatine kinase MB isoenzyme (CK-MB).

Safety evaluations will include adverse events, concomitant medications, physical exam, vital signs, and clinical laboratory testing.

An analysis of extended safety and efficacy will be performed in the subsequent open-label phases of the study.

Being tested
Deramiocel (CAP-1002), Placebo
Keywords
Duchenne Muscular Dystrophy, Cell Therapy, Performance of the Upper Limb, Ambulatory, Non-Ambulatory
Medical subject headings
Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic, Muscular Diseases, Neuromuscular Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn, Nervous System Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Deramiocel (CAP-1002)Biological

    Also called Cardiosphere-Derived Cells (CDCs)

    Cohort A: CAP-1002A manufactured in Los Angeles, CA; Cohort B: CAP-1002B manufactured in San Diego, CA; Open-Label Extension: CAP-1002A or CAP-1002B

  • PlaceboBiological

    Placebo

Study groups in NCT05126758
Study groupRoleWhat they receive
Deramiocel (CAP-1002)ExperimentalCohort A: Approximatetly 29 subjects will receive deramiocel (CAP-1002A) active treatment consisting of 150 million cardiosphere-derived cells (CDCs) via intravenous infusion every 3 months Cohort B: Approximately 22 participants will receive deramiocel (CAP-1002B) active treatment consisting of 150 million cardiosphere-derived cells (CDCs) via intravenous infusion every 3 months Open-Label Extension: After completion of the double-blind, placebo-controlled phase, subjects will receive deramiocel (CAP-1002) active treatment consisting of 150 million cardiosphere-derived cells (CDCs) via intravenous infusion every 3 months for 12 months followed by the long-term open label extension phase which continues deramiocel administration every 3 months until sponsor decision.
Receives: Biological: Deramiocel (CAP-1002)
PlaceboPlacebo comparatorCohort A: Approximately 29 subjects will receive a Placebo solution via intravenous infusion every 3 months Cohort B: Approximately 22 participants will receive a Placebo solution via intravenous infusion every 3 months
Receives: Biological: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change in the upper limb function

    Mean percent change from baseline in 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. Percent change from baseline is calculated as change from baseline divided by baseline score at the subject level.

    Measured: At Month 12

Secondary outcomes (10)

  1. Change in cardiac muscle function and structure by assessment of left ventricular ejection fraction

    Mean change from baseline in left ventricular ejection fraction as assessed by Cardiac Magnetic Resonance (cMRI) and assessed centrally

    Measured: At Month 12

  2. Change in mid-level (elbow) upper limb function

    Mean percent change from baseline in mid-level [elbow] upper limb function in 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. Percent change from baseline is calculated as change from baseline divided by baseline score at the subject level.

    Measured: At Month 12

  3. Change in Global Statistical Test (Total GST) combining upper limb function, cardiac muscle function, and patient reported measure of disease severity

    Mean change from baseline in Total GST that combines PUL 2.0 score, LVEF assessment by cMRI, and the Patient Global Impression of Severity (PGI-S)

    Measured: At Month 12

  4. Change in the number of segments of myocardial scarring (Cohort B only)

    Change in the number of segments of myocardial scarring as observed by late gadolinium enhancement (LGE)

    Measured: at Month 12

  5. Change in cardiac muscle function and structure by assessment of left ventricular end-systolic volume

    Mean change from baseline in left ventricular indexed end-systolic volume as assessed by Cardiac Magnetic Resonance (cMRI)

    Measured: At Month 12

  6. Change in cardiac muscle function and structure by assessment of left ventricular ejection fraction in subjects with documented cardiomyopathy

    Mean change from baseline in left ventricular ejection fraction as assessed by Cardiac Magnetic Resonance (cMRI) in subjects with documented cardiomyopathy at baseline.

    Measured: At Month 12

  7. Change in a global statistical test (Cardiac GST) combining assessments of cardiac muscle function and structure

    Mean change from baseline in Cardiac GST that combines LVEF, LV end-systolic volume index, and LV end-diastolic volume index

    Measured: At Month 12

  8. Change in percentage of myocardial scarring (Cohort B only)

    Change in percentage of myocardial scarring as observed by late gadolinium enhancement (LGE)

    Measured: at Month 12

  9. Change in hand-to-mouth function in the context of functional eating

    Mean change from baseline in the functional ability of eating 10 bites evaluated by Duchenne Video Assessment (DVA) using a video record of patient performing the task at home. Physical therapists score the patient's quality of movement in the video using scorecards with pre-specified compensatory movement criteria.

    Measured: At Month 12

  10. Changes in cardiac inflammation biomarker, creatine kinase MB isoenzyme [CK-MB]

    Mean change from baseline in CK-MB blood levels - MB fraction (% of total CK).

    Measured: At Month 12

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted19 Nov 2021
  2. Study start22 Jun 2022
  3. Primary completion18 Jun 2025
  4. Study completionMar 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
Cohort A: placebo-controlled, double-blind, randomized 1:1 (active:placebo); Cohort B: placebo-controlled, double-blind, randomized 1:1 (active:placebo)
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
106 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05126758
Sponsor's protocol ID
CAP-1002-DMD-04
Acronym
HOPE-3
Status last verified
Jul 2026
First submitted
4 Nov 2021
Last updated
28 Aug 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • McDonald CM, Villa C, Soslow JH, Maharry K, Hogan N, Binks M, Berth KC, Elliott KA, Taylor M, Hor KN, Signorovich J, Henricson EK, Phan HC, Apkon S, Ghosh PS, Tian C, Veerapandiyan A, Ramos-Platt L, Gambetta K, Bernes SM, Varadhachary AS, Iannaccone ST, Laverty CG, Perlman SJ, Bass NE, Mosher K, Butterfield RJ, Mathews KD, Scharf RJ, Smith EC, Emerson JA, Mercuri E, Awadalla MS, Marban L, Marban E; HOPE-3 Investigators. Deramiocel heart-derived cellular therapy in advanced Duchenne muscular dystrophy (HOPE-3): a phase 3, randomised, double-blind, placebo-controlled trial. Lancet. 2026 Aug 22;408(10556):721-733. doi: 10.1016/S0140-6736(26)01385-1. Epub 2026 Jul 29.

    PubMed 42526472

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Deramiocel (CAP-1002) in people with Muscular Dystrophies and Muscular Dystrophy, Duchenne, run by Capricor Inc. at 20 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 12 inclusion and 21 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 28 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 20 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change in the upper limb function (time frame: At Month 12). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05126758, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Aug 2026.

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