Testing ATX-01 for myotonic dystrophy type 1

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• 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

Study contacts

• Study coordinator: Project Manager
• Email: clinical@arthexbiotech.com
• Phone: +34676229821

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Understanding long-term progression in myotonic dystrophy type 1 (DM1)

  This study will follow adults with DM1 for at least four years to see how walking, hand function, muscle strength, heart rhythms, and lung function change over time.

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• Spanish National Registry for Myotonic Dystrophy Type 1

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  Recruiting at 8 sites Fundació Institut Germans Trias i Pujol · Multiple Locations, Andalusia and 7 other locations · NCT07385443

• Long-term PGN-EDODM1 extension for people with myotonic dystrophy type 1

  This open-label extension will try continued PGN-EDODM1 in people with myotonic dystrophy type 1 who completed a prior PGN-EDODM1 study to see how safe and tolerable it is over time.

  Recruiting at 3 sites Phase 2 PepGen Inc · Calgary, Alberta and 2 other locations · NCT07220603

• Wearable monitoring of walking and arm movement in adults with neuromuscular or metabolic movement disorders

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• Investigating PGN-EDODM1 for Myotonic Dystrophy Type 1

  This study is testing a new medication called PGN-EDODM1 to see if it is safe and well-tolerated in people with myotonic dystrophy type 1.

  Recruiting at 12 sites Phase 2 PepGen Inc · Calgary, Alberta and 11 other locations · NCT06667453

• Collecting data on muscle disorders using wearable devices

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.