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NCT Number: NCT06300307

Study of ATX-01 in Participants With DM1

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.

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Key information

Conditions

Myotonic Dystrophy 1 Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Musculoskeletal Diseases Myotonic Disorders Myotonic Dystrophy Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases

Age range

18 year–64 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Centre Intégré Universitaire de Santé et Services Sociaux du Saguenay-Lac-St-Jean, Chicoutimi, Quebec, Canada

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

ATX-01

Drug

Solution for infusion

Placebo

Drug

Solution for infusion

Primary outcomes

  1. Incidence of adverse events

    Time frame: Up to 120 days

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

Secondary outcomes

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

    Time frame: Up to 120 days

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

  2. Maximum observed plasma concentration (Cmax) of ATX-01

    Time frame: Up to 48 hours post-dose

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

    Time frame: Up to 48 hours post-dose

  4. Video hand opening time

    Time frame: Change from baseline up to 120 days

    To evaluate the efficacy of ATX-01 on myotonia in participants with DM1

  5. Change from baseline in ankle dorsiflexion strength by quantitative myometry

    Time frame: Change from baseline up to 120 days

    To evaluate the effects of ATX-01 in participants with DM1 on ankle dorsiflexion strength

  6. Change from baseline in Impact on Activities of Daily Living questionnaire item scores

    Time frame: Change from baseline up to 120 days

    The Impact on Activities of Daily Living questionnaire is a 7-item patient-reported outcome designed to evaluate the impact of ATX-01 on activities of daily living in participants with DM1.

Study contacts

Contact information is provided by the study sponsor or research team.

Project Manager

CONTACT

[email protected]

+34676229821

Sponsors and collaborators

Lead sponsor

ARTHEx Biotech S.L.

Industry

Registry information

Official study title

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

Acronym: ArthemiR

Important dates

Study start
2024
Primary completion
2027
Study completion
2027
First posted
Mar 8, 2024
Registry last updated
Feb 10, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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