NCT06300307

Study of ATX-01 in Participants With DM1

A Phase 1/2a Double-Blind, Placebo-controlled, Single- and Multiple Ascending Dose Study to Assess the Safety, Tolerability, PK, PD and Efficacy of IV Administration of ATX-01 In Male and Female Participants Aged 18 to 64 With Classic DM1

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 56 (estimated)
• Sponsor: ARTHEx Biotech S.L. · Industry
• Sex: All
• Age: 18 Years – 64 Years
• Healthy volunteers: Not accepted

Summary

The goal of this clinical trial is to test ATX-01 in participants with myotonic dystrophy type 1 (DM1). The main question it aims to answer is if ATX-01 is safe and well tolerated. The trial will compare the safety and tolerability of ATX-01 and a matching placebo. There will be a single-ascending dose part of the trial and a multiple-ascending dose part. In the single-ascending dose, participants will receive one dose of ATX-01 or placebo. In the multiple-ascending dose part, participants will receive three doses of ATX-01 or placebo. ATX-01 is a novel anti-miR (synthetic single stranded oligonucleotide) that inhibits a microRNA called miR-23b.

Conditions

• Myotonic Dystrophy 1

Interventions

Type	Name	Description
DRUG	ATX-01	Solution for infusion
DRUG	Placebo	Solution for infusion

Timeline

• Start date: 2024-10-15
• Primary completion: 2027-07-01
• Completion: 2027-07-01
• First posted: 2024-03-08
• Last updated: 2026-02-10

Locations

12 sites across 7 countries: United States, Canada, France, Italy, Netherlands, Spain, United Kingdom

Regulatory

• FDA-regulated drug study