Home › Myotonic Dystrophy › 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
Who can join
Ages 18 to 64 · All sexes
Full eligibility criteria
Key Inclusion Criteria: * Participants with a documented clinical diagnosis of DM1 (CTG expansion of \>150 repeats in DMPK gene measured in peripheral blood mononuclear cells) * Ambulatory, defined as able to complete a 10-meter walk/run test at screening without the use of assistive devices such as canes, walkers, or orthoses, except for ankle-foot orthoses * Presence for \>3 seconds of grip myotonia as confirmed by a central reader Key Exclusion Criteria: * Participants with congenital DM1 * Medical Research Council Muscle Scale score of less than 4 on ankle dorsiflexion or significant tibialis anterior atrophy that prevents a muscle biopsy * Use of mexiletine or other agent for myotonia within 21 days or 5 half-lives, whichever is longer, prior to screening
About the study
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.
What is being tested
- ATX-01 (drug)
- Placebo (drug)
Sponsor: ARTHEx Biotech S.L. · Participants: 56 · Started: Oct 15, 2024
Contact the study team
- Project Manager · Phone: +34676229821
Official record on ClinicalTrials.gov — NCT06300307
Locations in the U.S.
Conditions
From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.