The U.S. Food and Drug Administration (FDA) has allowed the enrollment of up to 30 additional boys with Duchenne muscular dystrophy (DMD) in a clinical study testing an experimental stem cell therapy.
The Phase 2 trial (NCT06579352) testing the treatment from Signature Biologics has enrolled five boys, ages 5 to 10, at two sites in Texas, according to a company press release.
No results have been reported to date from this first group. The additional participants may join the study once another clinical site becomes available. Qualified investigators interested in taking part in the trial may email Stefanie Johnson at the company.
Duchenne is caused by mutations in the DMD gene that result in virtually no production of dystrophin, a protein that protects muscles from damage during movement. Without enough functional dystrophin, patients experience the progressive muscle weakness and wasting that are symptoms of muscular dystrophy.
Signature’s experimental cell therapy uses mesenchymal stem cells derived from the umbilical cord. These cells have important features as potential treatments, including ease of collection and growth in the lab, a low likelihood of triggering an immune response, no relevant safety concerns, and the ability to differentiate into many types of functional cells. They can also release biological signals that may reduce inflammation and promote tissue repair.
Trial involves safety, functional assessments
The clinical study aims to assess the cell therapy’s safety, as well as its effects on functional assessments, lab tests, and inflammation-related biomarkers in boys with DMD who are able to walk. Participants must have a North Star Ambulatory Assessment score higher than 13 and lower than 30 (maximum is 34), and be able to complete a timed rise from the floor (the time it takes a person to stand up from lying on the back) in less than 10 seconds. They must also be receiving a stable dose of glucocorticoids and have not responded adequately to an established DMD treatment or be a poor candidate for one.
Participants will receive four treatment cycles, with the stem cell therapy administered via intravenous (into-the-vein) infusions for three consecutive days. The treatment cycles will be given three months apart, and participants will be evaluated at specific follow-up intervals through one year.
Functional assessments include the 4-Stair Climb test, which measures how quickly a child can climb four stairs; the 10-meter walk test, which assesses walking speed; and the North Star Ambulatory Assessment, which evaluates overall motor abilities.
The PedsQL DMD Module and the PedsQL Multidimensional Fatigue Scale will also be used to assess health-related quality of life and fatigue.
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