Testing ATX-01 for myotonic dystrophy type 1

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

This study is testing a new drug called ATX-01 to see if it is safe and well-tolerated in people with myotonic dystrophy type 1.

Quick facts

• Phase: Phase1; Phase2
• Study type: Interventional
• Enrollment: 56 (estimated)
• Ages: 18 Years to 64 Years
• Sex: All
• Sponsor: ARTHEx Biotech S.L. (Industry-sponsored)
• Locations: 12 sites (Los Angeles, California and 11 other locations)
• Trial ID: NCT06300307 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to evaluate the safety and tolerability of ATX-01 in participants diagnosed with myotonic dystrophy type 1 (DM1). The study consists of two parts: a single-ascending dose phase where participants receive either ATX-01 or a placebo, and a multiple-ascending dose phase where participants receive three doses of ATX-01 or placebo. ATX-01 is a novel anti-miR that targets the microRNA miR-23b, which is believed to play a role in the pathology of DM1. The trial will assess how well participants tolerate the drug compared to a placebo.

Who should consider this trial

Good fit: Ideal candidates are individuals with a confirmed diagnosis of DM1 who are ambulatory and exhibit grip myotonia.

Not a fit: Patients with congenital DM1 or significant muscle atrophy that prevents participation may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with myotonic dystrophy type 1.

How similar studies have performed: While this approach is novel, similar studies targeting microRNA in neuromuscular disorders have shown promise.

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

Where this trial is running

Los Angeles, California and 11 other locations

• UCLA — Los Angeles, California, United States (Recruiting)
• University of Florida — Gainesville, Florida, United States (Recruiting)
• University of Iowa Health Care - Department of Neurology — Iowa City, Iowa, United States (Recruiting)
• University of Kansas Medical Center, Department of Neurology — Fairway, Kansas, United States (Recruiting)
• Virginia Commonwealth University — Richmond, Virginia, United States (Recruiting)
• Centre Intégré Universitaire de Santé et Services Sociaux du Saguenay-Lac-St-Jean — Chicoutimi, Quebec, Canada (Recruiting)
• Institute of Myology — Paris, France (Recruiting)
• The NeMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan — Milan, Italy (Recruiting)
• Fondazione Policlinico A. Gemelli- IRCCS — Rome, Italy (Recruiting)
• Radboudumc — Nijmegen, Netherlands (Recruiting)
• Hospital Universitario Donostia — Donostia / San Sebastian, Spain (Recruiting)
• St. George's University Hospital — London, United Kingdom (Recruiting)

Study contacts

• Study coordinator: Project Manager

Conditions Myotonic Dystrophy 1