FDA Allows Expanded Enrollment in Clinical Study of Signature Biologics’ Investigational Cell Therapy for Duchenne Muscular Dystrophy
Study may enroll up to 30 additional subjects; sponsor is seeking an additional clinical site before recruitment resumes
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Signature Biologics, a clinical-stage cell therapy company committed to rigorous clinical research and to the DMD community, today announced that the U.S. Food and Drug Administration (FDA) has allowed expanded enrollment in a clinical study of the company’s investigational umbilical cord–derived mesenchymal stem cell (UC-MSC) therapy in subjects with Duchenne muscular dystrophy (DMD).
The study is conducted under an Investigational New Drug (IND) application held by its contracted CRO which serves as study sponsor. Signature Biologics manufactures the investigational product and funds the study.
The initial cohort of five subjects has completed enrollment. The study is open-label and historically controlled. The protocol includes functional assessments — 4 Stair Climb, 10-meter walk test, and the North Star Ambulatory Assessment — along with the PedsQL™ DMD Module and the PedsQL™ Multidimensional Fatigue Scale, metabolic laboratory testing, and inflammatory cytokine panels. No efficacy results from the study are being reported at this time.
FDA has allowed enrollment of up to 30 additional subjects. The sponsor is currently seeking an additional clinical site, and recruitment will resume once that site is activated. Qualified investigators interested in participating may contact Stefanie Johnson: press@signaturebiologics.com. Current study status and eligibility criteria are maintained in the ClinicalTrials.gov record, NCT06579352.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a rare, progressive genetic disorder caused by mutations in the dystrophin gene that lead to the gradual loss of muscle strength and function. It overwhelmingly affects boys, with symptoms typically appearing in early childhood and progressing over time to affect mobility, respiration, and cardiac function.
About the Study
The study is registered on ClinicalTrials.gov under identifier NCT06579352. The CRO is the study sponsor. Signature Biologics manufactures the investigational product used in the study and funds the study.
Important Information About Investigational Status
The UC-MSC therapy described above is for investigational use only and has not been approved by the FDA or any other regulatory authority. Its safety and effectiveness have not been established. No efficacy results from this study are reported in this release. Observations from a small number of subjects in an open-label study are not a measure of, and may not predict, the results of the expanded study. Nothing in this release should be interpreted as a claim of safety, efficacy, or approval, or as an FDA endorsement of the investigational product.
About Signature Biologics
Signature Biologics is a clinical-stage biotechnology company dedicated to the discovery, development, and commercialization of advanced therapeutics that leverage the biological properties of human tissues and cells. For more information, visit www.signaturebiologics.com.
Forward-Looking Statements
This release contains forward-looking statements regarding the planned expansion of enrollment, site activation, and the anticipated conduct of the study. These statements are subject to risks and uncertainties, including the outcome of ongoing clinical investigation and regulatory review, and actual results may differ materially. Signature Biologics undertakes no obligation to update these statements except as required by law.
View source version on businesswire.com: https://www.businesswire.com/news/home/20260811207495/en/
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