Safety and Therapeutic Potential of the FDA-approved Drug Metformin for C9orf72 ALS/FTD
Status:
Recruiting
Trial end date:
2022-08-01
Target enrollment:
Participant gender:
Summary
The primary objective is to assess the safety and tolerability of Metformin in subjects with
C9orf72 amyotrophic lateral sclerosis administered for 24 weeks. The overall objective is to
determine if Metformin is safe in C9orf72 ALS patients and is a potentially viable
therapeutic treatment for C9-ALS that reduces repeat-associated non-canonical start codon -
in DNA (non-ATG) (RAN) proteins that are produced by the C9orf72 repeat expansion mutation.