What the trial was testing
The HOPE-2 enrolled 20 patients with muscular dystrophy. The study was sponsored by Capricor Inc. and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was initial testing (phase 2). Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
CAP-1002 slowed arm function decline by 2.6 points on a standard test compared to placebo.
Lancet (London, England) · 2022 · NCT03406780
These findings — that cAP-1002 slowed the loss of upper body strength compared to placebo — were published in the Lancet (London, England) and represent the headline result of the study.
Researchers tracked outcomes across 20 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with muscular dystrophy, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
This was mid-stage testing and CAP-1002 is not yet FDA-approved for Duchenne muscular dystrophy. The results suggest it may help preserve arm function and heart health in later stages of the disease. If you or your child has Duchenne, ask your doctor about clinical trials testing CAP-1002 or other cell-based treatments.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open muscular dystrophy trials
Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients
Phase 2a trial of SAT-3247 in ambulatory DMD patients aged ≥ 7 and \< 10 years. The trial has two parts. In Part 1, the trial will study two doses of SAT-3247 in a randomized, double-blind, placebo-controlled weekday regimen for 12 weeks to determine the optimal dose, safety, tolerability, and preliminary efficacy. In Part 2, the trial will study two doses of SAT-3247 for an additional 9 months.
Research on the Relationship Between Scoliosis, Pain, Quality of Life, and Trunk Muscle Compensation Patterns Among Patients with Duchenne Muscular Dystrophy.
* Objective: The objective of this observational study is to evaluate and quantify trunk muscle compensatory movement patterns in patients with Duchenne Muscular Dystrophy (DMD) using computer vision technology. Additionally, the study seeks to explore the relationship between these compensatory patterns and scoliosis, upper limb function, pain levels, and quality of life during functional upper limb movements. * Key Research Questions: 1\) Can trunk compensatory movement patterns be accurately measured using computer vision analysis? 2) Are these compensatory patterns correlated with scoliosis, upper limb function levels, pain, and quality of life? 3) Do these patterns and their correlations change over time? * Methodology: 1. Participants: Patients diagnosed with Duchenne Muscular Dystrophy will be recruited for this study. 2. Assessments: * Scoliosis Evaluation: 1. Cobb angle measurement via X-ray imaging. 2. Upper Limb Function Assessment: 3. Performance of the Upper Limb Module 2.0 (PUL 2.0). 4. Brooke Upper Extremity Functional Classification Score. 5. Korean version of the Duchenne Muscular Dystrophy Functional Ability Self-Assessment Tool (K-DMDSAT). * Pain Measurement: 1. Korean version of the PainDETECT Questionnaire (KPD-Q). 2. Short Form McGill Pain Questionnaire. 3. Quality of Life Assessment: * Duchenne Muscular Dystrophy Quality of Life Questionnaire (DMD-QoL). 1. Trunk Compensation Analysis: * Surface electromyography (sEMG) to measure muscle activation. * Video analysis using computer vision to quantify trunk compensatory movement patterns. * The following tasks will be evaluated using the dominant arm for sEMG and video analysis: i. Pouring water into a cup. ii. Lifting a cup to drink water. iii. Grooming the front of the hair. iv. Moving small blocks within one minute (Box and Block Test). v. Reaching toward nearby objects in the front, left, and right directions. * Front: Directly in front of the participant's line of sight. * Left and right: Approximately 45 degrees to the left and right from the participant's front. * Nearby objects: A water bottle or cup weighing approximately 250g, placed at arm's length. vi. Reaching toward distant objects in the front, left, and right directions. * Distant objects: A water bottle or cup weighing approximately 250g, placed at 1.5 times the participant's arm length. * The sEMG attachment sites are as follows: i. Muscles for assessing upper limb functional movements: 1. Deltoid 2. Pectoralis major 3. Trapezius 4. Biceps brachii ii. Muscles for assessing trunk compensatory actions: <!-- --> 1. Sternocleidomastoid 2. Longissimus muscle 3. External oblique abdominal muscle