Signature Biologics has announced that the U.S. Food and Drug Administration (FDA) has allowed expanded enrollment in a clinical study evaluating the company’s investigational umbilical cord-derived mesenchymal stem cell (UC-MSC) therapy for Duchenne muscular dystrophy (DMD).
The study is being conducted under an Investigational New Drug (IND) application held by the contracted CRO serving as the study sponsor. Signature Biologics manufactures the investigational product and funds the study.
The initial cohort of five participants has completed enrollment. The open-label, historically controlled study includes functional assessments such as the 4 Stair Climb, 10-Meter Walk Test, and North Star Ambulatory Assessment (NSAA), as well as PedsQL DMD and fatigue assessments, metabolic laboratory testing, and inflammatory cytokine panels.
According to the company, the FDA has permitted enrollment of up to 30 additional participants. Recruitment will resume after an additional clinical site is activated.
Importantly, no efficacy results are being reported at this time. Families and researchers should therefore distinguish the FDA’s authorization to expand enrollment from evidence of clinical effectiveness.
Read More: Explore Duchenne Clinical Trials
