A Phase 2 interventional study of CAP-1002 and Placebo in Muscular Dystrophies, Muscular Dystrophy, Duchenne and Muscular Disorders, Atrophic, sponsored by Capricor Inc.. Completed at 9 sites in United States. Open to male participants aged 10 Years and older. Per ClinicalTrials.gov, last updated 2025-05-28.
Sponsored by Capricor Inc. · Phase 2, Interventional, and Treatment
Among 232 studies in Muscular Dystrophy, Duchenne with a site in United States — 35 open to participants now, 60 under way, 8 of them phase 3. Muscular Dystrophy, Duchenne · United States.
Among the 232 studies in Muscular Dystrophy, Duchenne with a site in United States, this one is the 96th largest by enrollment of the 171 interventional ones that give a count, is one of 21 that started in 2018, is one of 57 in phase 2, and was the 125th registered.
481 studies on the registry are indexed under Muscular Dystrophy, Duchenne; 107 are open to participants now.
Browse Muscular Dystrophy, Duchenne studies →35 of the 232 studies in Muscular Dystrophy, Duchenne with a site in United States are open now. Recruiting first, the newest first:
Vanderbilt University Medical Center · Sites in United States
Ages 18+ · Men only
Sarepta Therapeutics, Inc. · Sites in United States
Ages 4+ · Men only
University of Florida · Sites in United States
Ages 6+ · Men only
Vanderbilt University Medical Center · Sites in United States
Ages 8–18 · Men only
University of Rochester · Sites in United States
Ages 0 days–3 yrs · Men only
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 66 Muscular Dystrophy, Duchenne studies recruiting now, 30 (45%) set no upper age limit and 34 (52%) stop at 75 or younger; 59 take children under 18; 12 take healthy volunteers.
This study: 10 Years and older · male · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
232 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 57 · Phase 2 90 · Phase 3 40 · Phase 4 5
First in 2000; this study’s year is marked. 21 started in 2018.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 20 is below the median of 26 across 332 interventional studies indexed under Muscular Dystrophy, Duchenne.
Median enrollment 25 across the 171 interventional studies, 75 across the 55 observational. Of the 160 that completed or were terminated, 78 (49%) have results posted.
This tab as a report to print or save as PDF → With an account.
130 of 232 have an industry lead sponsor. The 10 most frequent:
Capricor Inc. is the lead sponsor of 11 studies on the registry; none are open to participants now.
Capricor Inc. has 5 studies filed under Muscular Dystrophy, Duchenne — fewer than ten, so there is no comparison to draw.
19 of the studies in Muscular Dystrophy, Duchenne with a site in United States that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT05881408Phase 3Sarepta Therapeutics, Inc.A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) | 2027-05-31 |
| NCT06290713University of FloridaVasodilator and Exercise Study for DMD (VASO-REx) | 2026-11 |
| NCT06450639Hoffmann-La RocheA Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD) | 2026-11-18 |
| NCT03368742Solid Biosciences Inc.Microdystrophin Gene Transfer Study in Adolescents and Children With DMD | 2026-11-30 |
| NCT03655223RTI InternationalEarly Check: Expanded Screening in Newborns | 2026-11-30 |
Add all 19 to your calendar (.ics) → With an account.
Among the 232 studies in Muscular Dystrophy, Duchenne with a site in United States, from their registry updates:
This tab as a report to print or save as PDF → With an account.
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.
481 studies on the registry are indexed under Muscular Dystrophy, Duchenne; 107 are open to participants now.
This study's enrollment of 20 is below the median of 26 across 332 interventional studies indexed under Muscular Dystrophy, Duchenne.
Browse Muscular Dystrophy, Duchenne studies →Capricor Inc. is the lead sponsor of 11 studies on the registry; none are open to participants now.
Compared within Muscular Dystrophy, Duchenne, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Muscular Dystrophies. The counts are of registry records indexed under each term, refreshed daily. How we classify →
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
BMI (body mass index)
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Patients will receive 150 million Cardiosphere-derived Cells (CDCs) via intravenous infusion every 3 months for a total of 4 doses.
Biological: CAP-1002
Patients will receive a placebo solution via intravenous infusion every 3 months for a total of 4 doses.
Drug: Placebo
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.
Also known as: Cardiosphere-Derived Cells, CDCs, deramiocel
Placebo
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).
Time frame: 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.
Time frame: 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.
Time frame: Baseline through Month 12
Number of Participants With All-cause Mortality
Number of participants who died due to any cause were reported.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: Baseline through Month 12
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.
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
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).
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
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).
Time frame: Baseline, Months 6 and 12
Change From Baseline in Left Ventricular Ejection Fraction at Month 6 and 12
Change from baseline in Left Ventricular Ejection Fraction assessed by cardiac magnetic resonance imaging (MRI) and expressed as percentile ranked change. 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).
Time frame: Month 6 and 12
Change From Baseline in Left Ventricular End-diastolic Volume at Month 6 and 12
Change from baseline in Left Ventricular End-diastolic Volume assessed by cardiac magnetic resonance imaging (MRI) expressed as percentile ranked change. 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).
Time frame: Month 6 and 12
Change From Baseline in Left Ventricular End-Diastolic Volume (Indexed) at Month 6 and 12
Change from baseline in Left Ventricular End-diastolic Volume (Indexed) assessed by cardiac magnetic resonance imaging (MRI) expressed as percentile ranked change. The End-diastolic Volume (Indexed) (EDVI) is calculated as follows: EDVI= EDV (End-diastolic Volume)/BSA (body surface area) = xxx mL/m\^2. 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).
Time frame: Month 6 and 12
Change From Baseline in Left Ventricular End-Systolic Volume at Month 6 and 12
Change from baseline in Left Ventricular End-systolic Volume assessed by cardiac magnetic resonance imaging (MRI) expressed as percentile ranked change. 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).
Time frame: Month 6 and 12
Change From Baseline in Left Ventricular End-Systolic Volume (Indexed) at Month 6 and 12
Change from baseline in Left Ventricular End-systolic Volume (Indexed) assessed by cardiac magnetic resonance imaging (MRI) expressed as percentile ranked change. The End-systolic Volume (Indexed) (ESVI) is calculated as follows: ESVI= ESV (End-systolic Volume)/BSA (body surface area) = xxx mL/m\^2. 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).
Time frame: Month 6 and 12
Participants took part in this study at 7 investigational sites in the United States from 04 April 2018 to 10 March 2020.
| Milestone | CAP-1002 | Placebo |
|---|---|---|
| Started | 8 | 12 |
| Treated | 8 | 12 |
| Completed | 7 | 9 |
| Not completed | 1 | 3 |
| Withdrew: Withdrawal by subject | 1 | 1 |
| Withdrew: Other | 0 | 2 |
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).
| Percentile Rank | CAP-1002 | Placebo |
|---|---|---|
| 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 | 65.5 ± 10.82 | 29.3 ± 9.23 |
Acute respiratory decompensation was defined as an unexplained rapid deterioration of the participant's condition with increasing shortness of breath requiring oxygen supplementation.
| Participants | CAP-1002 | Placebo |
|---|---|---|
| Number of Participants Experiencing Acute Respiratory Decompensation | 0 | 0 |
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.
| Participants | CAP-1002 | Placebo |
|---|---|---|
| Number of Participants With Hypersensitivity Reactions | 3 | 0 |
Number of participants who died due to any cause were reported.
| Participants | CAP-1002 | Placebo |
|---|---|---|
| Number of Participants With All-cause Mortality | 0 | 0 |
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.
| Participants | CAP-1002 | Placebo |
|---|---|---|
| Number of Participants With Serious Adverse Events (SAEs) | 2 | 0 |
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.
| Participants | CAP-1002 | Placebo |
|---|---|---|
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) Related to Investigational Product (IP) or Administration Procedure | 5 | 5 |
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.
| Participants | CAP-1002 | Placebo |
|---|---|---|
| Number of Participants With Immune Sensitization Syndrome | 0 | 0 |
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.
| participants | CAP-1002 | Placebo |
|---|---|---|
| Participants with TEAEs | 7 | 12 |
| Participants with Mild (Grade 1) TEAEs | 1 | 6 |
| Participants with Moderate (Grade 2) TEAEs | 4 | 5 |
| Participants with Severe (Grade 3) TEAEs | 1 | 1 |
| Participants with Life-threatening or Disabling (Grade 4) TEAEs | 1 | 0 |
| Participants with Fatal (Grade 5) TEAEs | 0 | 0 |
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).
| Percentile Rank | CAP-1002 | Placebo |
|---|---|---|
| At Month 3 | 86.6 ± 10.51 | 66.8 ± 8.40 |
| At Month 6 | 77.6 ± 11.19 | 49.9 ± 8.38 |
| At Month 9 | 68.1 ± 10.83 | 48.4 ± 8.97 |
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).
| Percentile Rank | CAP-1002 | Placebo |
|---|---|---|
| Systolic Wall Thickening Anterior LV at Month 6 | 46.3 ± 10.84 | 45.8 ± 7.13 |
| Systolic Wall Thickening Anterior LV at Month 12 | 40.9 ± 8.90 | 40.7 ± 8.88 |
| Systolic Wall Thickening Lateral LV at Month 6 | 50.4 ± 18.47 | 39.9 ± 12.32 |
| Systolic Wall Thickening Lateral LV at Month 12 | 49.2 ± 17.00 | 27.4 ± 14.26 |
| Systolic Wall Thickening Inferior LV at Month 6 | 57.3 ± 12.37 | 59.7 ± 8.52 |
| Systolic Wall Thickening Inferior LV at Month 12 | 60.3 ± 10.99 | 45.7 ± 9.94 |
| Systolic Wall Thickening Septal LV at Month 6 | 50.1 ± 16.89 | 49.2 ± 11.34 |
| Systolic Wall Thickening Septal LV at Month 12 | 50.8 ± 15.28 | 49.9 ± 12.97 |
Change from baseline in Left Ventricular Ejection Fraction assessed by cardiac magnetic resonance imaging (MRI) and expressed as percentile ranked change. 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).
| Percentile Rank | CAP-1002 | Placebo |
|---|---|---|
| Month 6 | 57.9 ± 11.00 | 34.9 ± 7.46 |
| Month 12 | 54.5 ± 9.08 | 8.9 ± 9.29 |
Change from baseline in Left Ventricular End-diastolic Volume assessed by cardiac magnetic resonance imaging (MRI) expressed as percentile ranked change. 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).
| Percentile Rank | CAP-1002 | Placebo |
|---|---|---|
| Month 6 | 54.7 ± 12.89 | 33.4 ± 8.87 |
| Month 12 | 49.2 ± 12.55 | 48.6 ± 11.81 |
Change from baseline in Left Ventricular End-diastolic Volume (Indexed) assessed by cardiac magnetic resonance imaging (MRI) expressed as percentile ranked change. The End-diastolic Volume (Indexed) (EDVI) is calculated as follows: EDVI= EDV (End-diastolic Volume)/BSA (body surface area) = xxx mL/m\^2. 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).
| Percentile Rank | CAP-1002 | Placebo |
|---|---|---|
| Month 6 | 34.8 ± 14.11 | 58.1 ± 11.51 |
| Month 12 | 12.2 ± 13.29 | 60.0 ± 12.27 |
Change from baseline in Left Ventricular End-systolic Volume assessed by cardiac magnetic resonance imaging (MRI) expressed as percentile ranked change. 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).
| Percentile Rank | CAP-1002 | Placebo |
|---|---|---|
| Month 6 | 48.4 ± 12.16 | 51.1 ± 8.44 |
| Month 12 | 33.3 ± 10.97 | 79.2 ± 10.52 |
Change from baseline in Left Ventricular End-systolic Volume (Indexed) assessed by cardiac magnetic resonance imaging (MRI) expressed as percentile ranked change. The End-systolic Volume (Indexed) (ESVI) is calculated as follows: ESVI= ESV (End-systolic Volume)/BSA (body surface area) = xxx mL/m\^2. 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).
| Percentile Rank | CAP-1002 | Placebo |
|---|---|---|
| Month 6 | 38.6 ± 12.96 | 50.2 ± 10.73 |
| Month 12 | 15.8 ± 12.27 | 68.9 ± 11.77 |
Collected over Baseline up to Month 12. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| CAP-1002 | 0/8 (0%) | 2/8 (25%) | 7/8 (87.5%) |
| Placebo | 0/12 (0%) | 0/12 (0%) | 12/12 (100%) |
| Event | CAP-1002 | Placebo |
|---|---|---|
| Allergic Reaction To ExcipientImmune system disorders | 1/8 | 0/12 |
| HypersensitivityImmune system disorders | 1/8 | 0/12 |
| Event | CAP-1002 | Placebo |
|---|---|---|
| NasopharyngitisInfections and infestations | 1/8 | 4/12 |
| NauseaGastrointestinal disorders | 1/8 | 3/12 |
| DizzinessNervous system disorders | 2/8 | 0/12 |
| Back PainMusculoskeletal and connective tissue disorders | 0/8 | 2/12 |
| DysgeusiaNervous system disorders | 1/8 | 2/12 |
| RashSkin and subcutaneous tissue disorders | 0/8 | 2/12 |
| TachycardiaCardiac disorders | 1/8 | 0/12 |
| Deafness NeurosensoryEar and labyrinth disorders | 1/8 | 0/12 |
| Abdominal PainGastrointestinal disorders | 1/8 | 0/12 |
| HaematocheziaGastrointestinal disorders | 1/8 | 0/12 |
| Age, Continuous(years) | CAP-1002 | Placebo | Total |
|---|---|---|---|
| Mean | 14.1 ± 3.44 | 14.4 ± 3.03 | 14.3 ± 3.11 |
| Sex: Female, Male(Participants) | CAP-1002 | Placebo | Total |
|---|---|---|---|
| Female | 0 | 0 | 0 |
| Male | 8 | 12 | 20 |
| Ethnicity (NIH/OMB)(Participants) | CAP-1002 | Placebo | Total |
|---|---|---|---|
| Hispanic or Latino | 2 | 0 | 2 |
| Not Hispanic or Latino | 6 | 12 | 18 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | CAP-1002 | Placebo | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 1 | 1 |
| Asian | 1 | 3 | 4 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 |
| White | 7 | 8 | 15 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
Ran from Apr 2018 to primary completion in Mar 2020, 23 months.
Results were first posted in Feb 2025, 4 years 11 months after primary completion.
Of 40 industry-sponsored phase 2 interventional studies under Muscular Dystrophy, Duchenne, half reached primary completion within 31 months of starting (the median), counting those still running; a quarter within 20 and three quarters within 42. This study's 23 months is below that.
Counting every sponsor, half of 47 studies reached it within 31 months; a quarter within 20 and three quarters within 42.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in May 2025. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Deramiocel
Muscular Dystrophy, Duchenne →
Capricor Inc.