Muscular DystrophiesPhase 2HOPE-2NCT03406780
A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
A Phase 2 study testing CAP-1002 in people with Muscular Dystrophies and Muscular Dystrophy, Duchenne, run by Capricor Inc. at 9 sites in United States.
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… Read more about this study
- Who is it for?
- Ages 10 years and older, men only
- Conditions the study is for
- Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic
- What is being tested?
- CAP-1002
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 20 participants
- Where
- 9 sitesUnited States
- Study dates
- 4 Apr 2018to 10 Mar 2020
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 participants at least 10 years of age at time of consent
- Participants 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
- Participants with 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, Gowers' sign, and gait impairment before 7 years of age) with confirmatory genetic testing performed at a Clinical Laboratory Improvement Amendments (CLIA)-certified laboratory.
- Participants with performance of the Upper Limb entry item score 2-5
- Participants if ambulatory, 10-meter walk/run velocity < 1 meter/second
- Participants with loss of independent ambulation by 18th birthday (standing unassisted or ability to take, at most, several steps independently is not considered ambulation)
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- Participants who 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
- Participants who received treatment with a systemic glucocorticoid is required for at least 12 months prior to randomization. The dose must remain stable for at least 6 months prior to randomization with the exception of 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
- Participants with current and up-to-date immunizations according to children and adolescent Centers for Disease Control immunization schedule, unless contraindicated, including the following: meningococcal and meningococcal B; tetanus, diphtheria \& acellular pertussis (Tdap); and pneumococcal polysaccharide vaccinations
- Participants with adequate venous access for parenteral IP infusions and routine blood collections in the judgement of the Investigator
- Participants assessed by the Investigator as willing and able to comply with the requirements of the trial
Who cannot join
- Participants with Left Ventricular Ejection Fraction (LVEF) < 35%
- Participants with elbow-flexion contractures > 30° in both extremities
- Participants with Body Mass Index (BMI) > 45
- Participants with documentation of exon 44 skip-amenable mutation(s) in the dystrophin gene
- Participants with documentation of dystrophin deletion mutation(s) encompassing and limited to exons 3-7
- Participants with percent predicted FVC (FVC%p) < 35%
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- Participants with inability to perform consistent FVC measurement within ±15% during paired testing at screening
- Participants with 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 at screening
- Participants with history of non DMD-related chronic respiratory disease requiring ongoing or intermittent treatment, including, but not limited to, asthma, bronchitis, and tuberculosis
- Participants with acute respiratory illness within 30 days prior to screening
- Participants with initiation of non-invasive ventilation within 30 days prior to screening, or the anticipated need to initiate non-invasive ventilation within the 12 months following screening
- Participants with planned or anticipated thoracic or spinal surgery within the 12 months following randomization
- Participants with planned or anticipated lower extremity surgery within the 12 months following randomization, if ambulatory
- Participants with known hypersensitivity to Dimethyl Sulfoxide (DMSO) or bovine products
- Participants with initiation of treatment with metformin or insulin within 3 months prior to randomization
- Participants with initiation of treatment with an FDA-approved exon skipping therapy for the treatment of DMD within 24 months prior to randomization or dose adjustments to the therapy within 12 months prior to randomization with the exception of weight-based dose adjustments.
- Participants who received treatment with Human Growth Hormone (HGH) within 3 months prior to randomization, unless on a stable dose (as determined by the site PI) for at least 24 months prior to randomization
- Participants who received Treatment with idebenone within 3 months prior to randomization
- Participants who received treatment with a cell therapy product within 12 months prior to randomization
- Participants who received treatment with an investigational product within 6 months prior to randomization
- Participants with history, or current use, of drugs or alcohol that could impair their ability to comply with participation in the trial
- Participants with inability to comply with the investigational plan and follow-up visit schedule for any reason, in the judgment of the investigator
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
United States9 sites
Children's Hospital Colorado
Aurora, Colorado, 80045
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
University of Iowa
Iowa City, Iowa, 52242
Children's Hospital Wisconsin
Milwaukee, Wisconsin, 53226
Nemours Children's Hospital
Orlando, Florida, 32827
University of California, Davis
Sacramento, California, 95817
University of Utah
Salt Lake City, Utah, 84112
Washington University
St Louis, Missouri, 63110
University of Massachusetts Medical Center
Worcester, Massachusetts, 01655
About this study
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
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.
Read the detailed description
- Approximately 20 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 by Performance of the Upper Limb, cardiac MRI, pulmonary function testing, North Star Ambulatory Assessment (ambulatory subjects only), strength testing, 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.
- Conditions
- Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic, Muscular Diseases, Neuromuscular Diseases, Nervous System Diseases
- Being tested
- CAP-1002, Placebo
- Keywords
- Duchenne Muscular Dystrophy, Cell Therapy, Performance of the Upper Limb, Pulmonary Function, Ambulatory, Non-Ambulatory, Glucocorticoids
- Medical subject headings
- Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic, Muscular Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CAP-1002Biological
Also called Cardiosphere-Derived Cells, CDCs, deramiocel
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, anti-inflammatory, and pro-angiogenic.
PlaceboDrug
Placebo
| Study group | Role | What they receive |
|---|---|---|
| CAP-1002 | Experimental | Patients will receive 150 million Cardiosphere-derived Cells (CDCs) via intravenous infusion every 3 months for a total of 4 doses. Receives: Biological: CAP-1002 |
| Placebo | Placebo comparator | Patients will receive a placebo solution via intravenous infusion every 3 months for a total of 4 doses. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Functional Capacity as Assessed by the Mid-level (Elbow) Dimension Score of the Performance of Upper Limb (PUL) Version 1.2 at Month 12
Change from baseline in functional capacity as assessed by the mid-level (elbow) dimension of PUL version 1.2 at Month 12 expressed as percentile ranked change.
PUL 1.2 scale assesses motor performance in the upper limb. PUL 1.2 included 22 items. One entry item to define the starting functional level, and 21 items subdivided into: Shoulder Level (score 0 to 16); Elbow Level (score 0 to 34); Distal Level Dimension (score 0 to 24). The total score range was from 0 to 74. For all items, the higher the score, the better the outcome. A negative change indicates worst the outcome. Change from baseline and baseline values were converted to a percentile rank over all timepoints and at baseline using a non-parametric version of the prespecified model, generated by calculating the percentile rank of each change-from-baseline value relative to all observed change-from-baseline values for the same outcome (across all patients and all post-baseline observation times).
Measured: Baseline, Month 12
Number of Participants Experiencing Acute Respiratory Decompensation
Acute respiratory decompensation was defined as an unexplained rapid deterioration of the participant's condition with increasing shortness of breath requiring oxygen supplementation.
Measured: Baseline through Month 12
Number of Participants With Hypersensitivity Reactions
Hypersensitivity reaction was defined as a clinical syndrome including, but not limited to, fever, leukocytosis, or rash with onset less than or equal to (<=) 2 hours post-infusion and lasting less than (<) 24 hours, in the absence of clinical signs of concomitant infection.
Measured: Baseline through Month 12
Number of Participants With All-cause Mortality
Number of participants who died due to any cause were reported.
Measured: Baseline through Month 12
Number of Participants With Serious Adverse Events (SAEs)
A SAE was defined as an AE that results in any of the following outcomes: Death; life-threatening adverse event; Inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions; congenital anomaly/birth defect.
Measured: Baseline through Month 12
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Related to Investigational Product (IP) or Administration Procedure
TEAE was defined as an AE that was not present prior to the initiation of line placement procedure for the IP infusion or was present but worsened in intensity or frequency. The Investigator assessed the relationship (causality) of an AE to the investigational product and administration procedure.
Measured: Baseline through Month 12
Number of Participants With Immune Sensitization Syndrome
Immune sensitization syndrome is defined as a) clinical signs and symptoms that are consistent with systemic inflammation (e.g.,fever, leukocytosis, rash, arthralgia), with an onset >=24 hours after infusion of the investigational product, in the absence of clinical signs of concomitant infection, and b) an elevation of anti-Human Leukocyte Antigen (anti-HLA) antibodies against the Donor-Specific Antibody (DSA) cells, which is detected <=30 days after the onset of syndrome, that meets the following criteria: i) 2000 mean fluorescence intensity if mean fluorescence intensity is <=1000 at baseline, or ii) >=2 times the baseline value.
Measured: Baseline through Month 12
Secondary outcomes (3)
Number of Participants With TEAEs and Severity of TEAEs
TEAE was defined as an AE that was not present prior to the initiation of line placement procedure for the IP infusion or was present but worsened in intensity or frequency. Severity of TEAEs were determined by following criteria:: Mild (Grade 1): Transient or mild discomfort; no limitation in activity; no medical intervention/therapy required; Moderate (Grade 2): Mild to moderate limitation in activity - some assistance may be needed; no or minimal medical intervention/therapy required; Severe (Grade 3): Marked limitation in activity, some assistance usually required; medical intervention/therapy required and often requiring hospitalization or prolongation of hospitalization; Life-Threatening or Disabling (Grade 4): Extreme limitation in activity, significant assistance required; significant medical intervention/therapy required; hospitalization, prolongation of hospitalization, or hospice care; Fatal (Grade 5): death.
Measured: Baseline through Month 12
Change From Baseline in the Mid-level (Elbow) Dimension Score of the PUL 1.2 at Months 3, 6, and 9
Change From Baseline in the Mid-level (Elbow) Dimension Score of the PUL 1.2 at Months 3, 6, and 9 expressed as percentile ranked change.
PUL 1.2 scale assesses motor performance in the upper limb. PUL 1.2 included 22 items. One entry item to define the starting functional level, and 21 items subdivided into: Shoulder Level (score 0 to 16); Elbow Level (score 0 to 34); Distal Level Dimension (score 0 to 24). The total score range was from 0 to 74. For all items, the higher the score, the better the outcome. A negative change indicates worst the outcome. Change from baseline and baseline values were converted to a percentile rank over all timepoints and at baseline using a non-parametric version of the prespecified model, generated by calculating the percentile rank of each change-from-baseline value relative to all observed change-from-baseline values for the same outcome (across all patients and all post-baseline observation times).
Measured: Baseline, Months 3, 6, and 9
Change From Baseline in Regional Systolic Left Ventricular (LV) Wall Thickening, as Assessed by Cardiac Magnetic Resonance Imaging (MRI) at Months 6 and 12
Change from baseline in regional systolic LV wall thickening (anterior, lateral, inferior, septal) expressed as percentile ranked change, as assessed by cardiac MRI at Months 6 and 12.
Change from baseline and baseline values were converted to a percentile rank over all timepoints and at baseline using a non-parametric version of the prespecified model, generated by calculating the percentile rank of each change-from-baseline value relative to all observed change-from-baseline values for the same outcome (across all patients and all post-baseline observation times).
Measured: Baseline, Months 6 and 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jan 2018
- Study start4 Apr 2018
- Primary completion10 Mar 2020
- Study completion10 Mar 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Muscular DystrophiesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Capricor Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Craig McDonald, MD, Principal investigator, University of California, Davis
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03406780
- Sponsor's protocol ID
- CAP-1002-DMD-02
- Acronym
- HOPE-2
- Status last verified
- May 2025
- First submitted
- 15 Jan 2018
- Last updated
- 28 May 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 35279258
Study at a glance
From the registry record, last updated by the sponsor on 28 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03406780
- Acronym / sponsor ID
- HOPE-2 / CAP-1002-DMD-02
- Conditions
- Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic, Muscular Diseases, Neuromuscular Diseases, Nervous System Diseases
- Keywords
- Duchenne Muscular Dystrophy, Cell Therapy, Performance of the Upper Limb, Pulmonary Function, Ambulatory, Non-Ambulatory, Glucocorticoids
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Capricor Inc.Industry (pharma, biotech, devices)
- Study start
- 4 Apr 2018 (actual)
- Primary completion
- 10 Mar 2020 (actual)
- Study completion
- 10 Mar 2020 (actual)
- First posted
- 23 Jan 2018 (actual)
- Results
- Posted 24 Feb 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 20 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 by Performance of the Upper Limb, cardiac MRI, pulmonary function testing, North Star Ambulatory Assessment (ambulatory subjects only), strength testing, 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| CAP-1002 | Experimental | Biological: CAP-1002Patients will receive 150 million Cardiosphere-derived Cells (CDCs) via intravenous infusion every 3 months for a total of 4 doses. |
| Placebo | Placebo comparator | Drug: PlaceboPatients will receive a placebo solution via intravenous infusion every 3 months for a total of 4 doses. |
Who may qualify
- Ages
- 10 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (11 listed)
- Male participants at least 10 years of age at time of consent
- Participants 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
- Participants with 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, Gowers' sign, and gait impairment before 7 years of age) with confirmatory genetic testing performed at a Clinical Laboratory Improvement Amendments (CLIA)-certified laboratory.
- Participants with performance of the Upper Limb entry item score 2-5
- Participants if ambulatory, 10-meter walk/run velocity < 1 meter/second
Key exclusion criteria (22 listed)
- Participants with Left Ventricular Ejection Fraction (LVEF) < 35%
- Participants with elbow-flexion contractures > 30° in both extremities
- Participants with Body Mass Index (BMI) > 45
- Participants with documentation of exon 44 skip-amenable mutation(s) in the dystrophin gene
- Participants with documentation of dystrophin deletion mutation(s) encompassing and limited to exons 3-7
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CAP-1002Also: Cardiosphere-Derived Cells, CDCs, deramiocelThe 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, anti-inflammatory, and pro-angiogenic. | Biological | CAP-1002 |
| PlaceboPlacebo | Drug | Placebo |
Primary outcome
Change From Baseline in Functional Capacity as Assessed by the Mid-level (Elbow) Dimension Score of the Performance of Upper Limb (PUL) Version 1.2 at Month 12
Time frame: Baseline, Month 12
Change from baseline in functional capacity as assessed by the mid-level (elbow) dimension of PUL version 1.2 at Month 12 expressed as percentile ranked change. PUL 1.2 scale assesses motor performance in the upper limb. PUL 1.2 included 22 items. One entry item to define the starting functional level, and 21 items subdivided into: Shoulder Level (score 0 to 16); Elbow Level (score 0 to 34); Distal Level Dimension (score 0 to 24). The total score range was from 0 to 74. For all items, the higher the score, the better the outcome. A negative change indicates worst the outcome. Change from baseline and baseline values were converted to a percentile rank over all timepoints and at baseline using a non-parametric version of the prespecified model, generated by calculating the percentile rank of each change-from-baseline value relative to all observed change-from-baseline values for the same outcome (across all patients and all post-baseline observation times).
Number of Participants Experiencing Acute Respiratory Decompensation
Time frame: Baseline through Month 12
Acute respiratory decompensation was defined as an unexplained rapid deterioration of the participant's condition with increasing shortness of breath requiring oxygen supplementation.
Number of Participants With Hypersensitivity Reactions
Time frame: Baseline through Month 12
Hypersensitivity reaction was defined as a clinical syndrome including, but not limited to, fever, leukocytosis, or rash with onset less than or equal to (<=) 2 hours post-infusion and lasting less than (<) 24 hours, in the absence of clinical signs of concomitant infection.
Number of Participants With All-cause Mortality
Time frame: Baseline through Month 12
Number of participants who died due to any cause were reported.
Number of Participants With Serious Adverse Events (SAEs)
Time frame: Baseline through Month 12
A SAE was defined as an AE that results in any of the following outcomes: Death; life-threatening adverse event; Inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions; congenital anomaly/birth defect.
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Related to Investigational Product (IP) or Administration Procedure
Time frame: Baseline through Month 12
TEAE was defined as an AE that was not present prior to the initiation of line placement procedure for the IP infusion or was present but worsened in intensity or frequency. The Investigator assessed the relationship (causality) of an AE to the investigational product and administration procedure.
Number of Participants With Immune Sensitization Syndrome
Time frame: Baseline through Month 12
Immune sensitization syndrome is defined as a) clinical signs and symptoms that are consistent with systemic inflammation (e.g.,fever, leukocytosis, rash, arthralgia), with an onset >=24 hours after infusion of the investigational product, in the absence of clinical signs of concomitant infection, and b) an elevation of anti-Human Leukocyte Antigen (anti-HLA) antibodies against the Donor-Specific Antibody (DSA) cells, which is detected <=30 days after the onset of syndrome, that meets the following criteria: i) 2000 mean fluorescence intensity if mean fluorescence intensity is <=1000 at baseline, or ii) >=2 times the baseline value.
3 secondary outcomes
Number of Participants With TEAEs and Severity of TEAEs
Time frame: Baseline through Month 12
Change From Baseline in the Mid-level (Elbow) Dimension Score of the PUL 1.2 at Months 3, 6, and 9
Time frame: Baseline, Months 3, 6, and 9
Change From Baseline in Regional Systolic Left Ventricular (LV) Wall Thickening, as Assessed by Cardiac Magnetic Resonance Imaging (MRI) at Months 6 and 12
Time frame: Baseline, Months 6 and 12
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 May 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 35279258McDonald 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.
- Company websiteLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Craig McDonald, MD principal investigator
University of California, Davis
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing CAP-1002 in people with Muscular Dystrophies and Muscular Dystrophy, Duchenne, run by Capricor Inc. at 9 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 11 inclusion and 22 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: finished, as of the sponsor's last update on 28 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 24 Feb 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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 From Baseline in Functional Capacity as Assessed by the Mid-level (Elbow) Dimension Score of the Performance of Upper Limb (PUL) Version 1.2 at Month 12 (time frame: Baseline, Month 12). The study lists 7 primary outcomes in all. 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 NCT03406780, 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 May 2025.