Phase 2
Completed N=20
A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
Muscular Dystrophies · Muscular Dystrophy, Duchenne · Muscular Disorders, Atrophic · Muscular Diseases
Source: ClinicalTrials.gov NCT03406780 ↗
Enrolled (actual)
20
Serious AEs
10.0%
Results posted
Feb 2025
Primary outcomePrimary: 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; 29.3 Percentile Rank
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 3 months for a total of 4 doses during a 12-month period.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY 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; 29.3 | — |
| PRIMARY Number of Participants Experiencing Acute Respiratory Decompensation |
0; 0 | — |
| PRIMARY Number of Participants With Hypersensitivity Reactions |
3; 0 | — |
| PRIMARY Number of Participants With All-cause Mortality |
0; 0 | — |
| PRIMARY Number of Participants With Serious Adverse Events (SAEs) |
2; 0 | — |
| PRIMARY Number of Participants With Treatment-emergent Adverse Events (TEAEs) Related to Investigational Product (IP) or Administration Procedure |
5; 5 | — |
| PRIMARY Number of Participants With Immune Sensitization Syndrome |
0; 0 | — |
| SECONDARY Number of Participants With TEAEs and Severity of TEAEs |
7; 12; 1; 6; 4; 5 | — |
| SECONDARY Change From Baseline in the Mid-level (Elbow) Dimension Score of the PUL 1.2 at Months 3, 6, and 9 |
86.6; 66.8; 77.6; 49.9; 68.1; 48.4 | — |
| SECONDARY Change From Baseline in Regional Systolic Left Ventricular (LV) Wall Thickening, as Assessed by Cardiac Magnetic Resonance Imaging (MRI) at Months 6 and 12 |
46.3; 45.8; 40.9; 40.7; 50.4; 39.9 | — |
Eligibility Criteria
Inclusion Criteria
- 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 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%
- 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
Data sourced from ClinicalTrials.gov (NCT03406780). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.