Study of ATX-01 in Participants With DM1

NCT06300307 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: CT-ATX-01-DM1-1.1
• Study Type: interventional
• Target: 56
• Locations: 7 countries
• Sites: 12

Brief 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

Outcome Measures

Primary Outcomes (1)

• Incidence of adverse events

  To evaluate the safety and tolerability of ATX-01 in adult participants with DM1

  Up to 120 days

Secondary Outcomes (6)

• Incidence of clinically significant changes in laboratory assessments, electrocardiograms (ECGs), vital signs, suicidal ideation and behavior

  Up to 120 days

• Maximum observed plasma concentration (Cmax) of ATX-01

  Up to 48 hours post-dose

• Area under the plasma concentration-time curve (AUC) of ATX-01

  Up to 48 hours post-dose

• Video hand opening time

  Change from baseline up to 120 days

• Change from baseline in ankle dorsiflexion strength by quantitative myometry

  Change from baseline up to 120 days

Study Arms (2)

ATX-01

EXPERIMENTAL

ATX-01 is a formulation of the anti-microRNA 23b (anti-miR-23b), known as X82108, a novel type of antisense oligonucleotide

Drug: ATX-01

Placebo

PLACEBO COMPARATOR

Placebo to ATX-01

Drug: Placebo

Interventions

ATX-01 DRUG

Solution for infusion

ATX-01

Placebo DRUG

Solution for infusion

Placebo

Eligibility Criteria

• Age: 18 Years - 64 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• ARTHEx Biotech S.L.: lead

Study Sites (12)

UCLA

Los Angeles, California, 90095, United States

RECRUITING

University of Florida

Gainesville, Florida, 32610, United States

RECRUITING

University of Iowa Health Care - Department of Neurology

Iowa City, Iowa, 52242, United States

RECRUITING

University of Kansas Medical Center, Department of Neurology

Fairway, Kansas, 66205, United States

RECRUITING

Virginia Commonwealth University

Richmond, Virginia, 23298, United States

RECRUITING

Centre Intégré Universitaire de Santé et Services Sociaux du Saguenay-Lac-St-Jean

Chicoutimi, Quebec, G7H 5H6, Canada

RECRUITING

Institute of Myology

Paris, 75651, France

RECRUITING

The NeMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan

Milan, 20162, Italy

RECRUITING

Fondazione Policlinico A. Gemelli- IRCCS

Rome, 00168, Italy

RECRUITING

Radboudumc

Nijmegen, 6525, Netherlands

RECRUITING

Hospital Universitario Donostia

Donostia / San Sebastian, 20014, Spain

RECRUITING

St. George's University Hospital

London, SW17 0RE, United Kingdom

RECRUITING

MeSH Terms

Conditions

Myotonic Dystrophy

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Myotonic Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Project Manager

CONTACT

+34676229821; clinical@arthexbiotech.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 26, 2024
• First Posted: March 8, 2024
• Study Start: October 15, 2024
• Primary Completion (Estimated): July 1, 2027
• Study Completion (Estimated): July 1, 2027
• Last Updated: February 10, 2026

Record last verified: 2026-02

Locations

ES (1); GB (1); NL (1); FR (1); US (5); CA (1); IT (2)