Phase 12a Study of ATX-01 Infusion to Assess Safety and Tolerability in Adults Aged 18 to 64 With Classic Myotonic Dystrophy Type 1

What this Trial Is About

Researchers are evaluating the safety and tolerability of ATX-01 in adults aged 18 to 64 with classic myotonic dystrophy type 1 DM1. This phase 12a trial aims to compare ATX-01, a new synthetic single-stranded oligonucleotide that inhibits microRNA miR-23b, with a placebo. The study focuses on whether ATX-01 is safe, well tolerated, and its effects on patients with DM1. The trial includes two parts a single-ascending dose phase where participants receive one dose of ATX-01 or placebo, and a multiple-ascending dose phase where participants receive three doses. Both treatments are given intravenously. The study groups consist of those receiving ATX-01 and those receiving a matching placebo. Participants will be monitored for up to 120 days to assess adverse events, laboratory tests, heart activity via ECG, vital signs, and any changes in suicidal thoughts or behavior. Other measurements include blood levels of ATX-01, hand opening time, ankle strength, and daily living activity impacts. Safety and tolerability are carefully tracked throughout the study period under a randomized, triple-blind design.

Study of ATX-01 in Participants With DM1