What side effects are associated with this type of intervention?

Common treatments for Muscular Dystrophy, particularly cell therapies like CAP-1002, generally show a favorable safety profile with minimal serious adverse effects reported. However, some potential side effects include respiratory tract infections, cough, fever, headache, joint pain, and oropharyngeal pain. Other treatments, such as glucocorticoids, can cause weight gain, bone thinning, high blood pressure, and mood swings. Eteplirsen, another treatment, has shown no serious adverse effects but may cause mild side effects like headache and fever. Ataluren can cause vomiting, decreased appetite, weight loss, and gastrointestinal discomfort.

What prior FDA approvals exist?

The FDA has approved several treatments for Duchenne muscular dystrophy (DMD) that aim to improve muscle function. Eteplirsen, golodirsen, and viltolarsen are exon-skipping drugs that increase dystrophin production in patients with specific genetic mutations. Ataluren is another approved drug that promotes read-through of premature stop codons in the dystrophin gene. These treatments, while not cell therapies like CAP-1002, share the goal of enhancing muscle function in DMD patients. CAP-1002, currently under investigation, is a cell therapy designed to improve skeletal muscle function, representing a novel approach compared to the existing exon-skipping and gene therapy treatments.

What other types of research exist?

Promising alternatives to CAP-1002 for treating Duchenne muscular dystrophy include: <ol> <li>Ataluren (PTC124): An orally administered drug that promotes ribosomal read-through of nonsense mutations, potentially benefiting 10-15% of DMD patients with such mutations. It has shown some efficacy in increasing dystrophin production and improving certain functional outcomes in clinical trials.</li> <li>Casimersen: An exon-skipping therapy that increases dystrophin production in patients with specific genetic mutations.</li> </ol> It has shown a mean increase in dystrophin levels in clinical trials. 3. Stem Cell Therapies: Various stem cell-based approaches, including mesenchymal stem cells and myogenic stem cell transplantation, are being explored for their potential to repair and regenerate damaged muscle tissue in DMD patients.

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Cell Therapy

Cell Therapy for Duchenne Muscular Dystrophy (HOPE-3 Trial)

Phase 3

Recruiting

Led By Craig McDonald, MD

Research Sponsored by Capricor Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Adequate venous access for parenteral IP infusions and routine blood collection.

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 specific characteristics will be capped at no more than 10% of the total study population.

Must not have

Elbow-flexion contractures > 30° in both extremities.

History of non DMD-related chronic respiratory disease requiring ongoing or intermittent treatment.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up at months 1, 3, 6, 9, 12

Awards & highlights

Pivotal Trial

Summary

This trial tests a cell therapy called CAP-1002 in boys and young men with Duchenne muscular dystrophy. The therapy involves giving special cells through an IV to help improve muscle function. The goal is to see if this treatment can repair or regenerate damaged muscles.

Who is the study for?

This trial is for boys and young men at least 10 years old with genetically confirmed Duchenne muscular dystrophy (DMD). They must have been on stable glucocorticoids for a year, have up-to-date immunizations, and adequate venous access. Ambulatory participants should take more than 10 seconds to walk/run 10 meters; non-ambulatory ones should have lost independent walking between ages 10-18.

What is being tested?

The HOPE-3 trial tests CAP-1002 cell therapy's effectiveness in improving muscle function in DMD patients. Participants are randomly assigned to receive either CAP-1002 or placebo every three months over a year, followed by an open-label extension where all get CAP-1002.

What are the potential side effects?

Potential side effects of CAP-1002 may include reactions related to the infusion process, immune system responses due to the cells being introduced into the body, and possible discomfort at injection sites.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have good veins for IV treatments and blood tests.

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My upper limb function scores are within the required range for the study.

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I have been diagnosed with Duchenne Muscular Dystrophy.

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It takes me more than 10 seconds to walk or run 10 meters.

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My DMD diagnosis was confirmed by genetic testing in a certified lab.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I cannot fully straighten my arms due to stiffness.

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I have a chronic lung condition not related to muscular dystrophy that needs treatment.

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I am expecting to have chest or spine surgery within the next 6 months.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ at months 1, 3, 6, 9, 12

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~at months 1, 3, 6, 9, 12

This trial's timeline: 3 weeks for screening, Varies for treatment, and at months 1, 3, 6, 9, 12 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change in the upper limb function

Secondary study objectives

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

Change in cardiac muscle function and structure by assessment of left ventricular end-diastolic volume

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

+7 more

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: CAP-1002Experimental Treatment1 Intervention

Cohort A: Approximatetly 29 subjects will receive 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 CAP-1002B active treatment consisting of 150 million cardiosphere-derived cells (CDCs) via intravenous infusion every 3 months

Group II: PlaceboPlacebo Group1 Intervention

Cohort 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

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

CAP-1002

2018

Completed Phase 2

~90

0 / 8Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Muscular Dystrophy (MD) include glucocorticoids, exon-skipping drugs, and cell therapies. Glucocorticoids help reduce inflammation and slow muscle degeneration. Exon-skipping drugs, like casimersen, promote the production of functional dystrophin protein by skipping faulty exons during gene expression. Cell therapies, such as CAP-1002, involve the infusion of donor or autologous stem cells to repair and regenerate damaged muscle tissue. CAP-1002 specifically aims to improve skeletal muscle function in Duchenne Muscular Dystrophy (DMD) patients by delivering cells that can differentiate into muscle cells and support muscle repair. These treatments are crucial for MD patients as they target the underlying causes of muscle degeneration, potentially improving muscle function and quality of life.

Evolving paradigms in clinical pharmacology and therapeutics for the treatment of Duchenne muscular dystrophy.Muscle atrophy reversed by growth factor activation of satellite cells in a mouse muscle atrophy model.Cell therapy in Duchenne muscular dystrophy treatment: clinical trials overview.