Study of Intramuscular AAV9-Follistatin Gene Therapy Alone or With VEGF Plasmid to Address Age-Related Muscle Loss in Adults Aged 35 to 75

What this Trial Is About

Researchers are evaluating the safety and tolerability of a gene therapy using AAV9-Follistatin alone or combined with a VEGF plasmid to address age-related muscle decline. This Phase 12a open-label study involves adults aged 35 to 75 years with muscle loss, focusing on safety and early signs of muscle mass and function improvement. Participants will be assigned sequentially to one of three groups low-dose AAV-Follistatin monotherapy, high-dose AAV-Follistatin monotherapy, or combination therapy with VEGF plasmid followed by AAV-Follistatin. Treatments are given through intramuscular injections in major muscles, with rapamycin administered for about two months to reduce immune reactions. The combination group receives VEGF plasmid twice before a delayed AAV-Follistatin dose. The study uses a cautious 33 dose-escalation design with safety reviews between cohorts. Participants undergo screening including medical history, physical exams, lab tests, and muscle assessments such as DXA and strength tests. The core study period lasts about 90 days 120 days for the combination group with frequent safety and function monitoring. Optional follow-up visits at 6 and 12 months are available. Researchers measure treatment side effects, immune responses, muscle performance, and safety outcomes, with independent oversight to manage risks. Total participation may extend up to 12 months.

CALM-AF-AI: Counteracting Age-related Loss of Muscle With AAV-Follistatin Combined With Angiogenesis-Inducing VEGF Plasmid Gene Therapy