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Taking people in United StatesPhase 1/2

Study of ATX-01 in Participants With DM1

Key facts

Who runs it
ARTHEx Biotech S.L.
Where
Fairway, Gainesville, Iowa City, Los Angeles, and Richmond, United StatesSee the 5 sitesAlso in 6 other countries, 7 sites
Trial code
Status
Recruiting
Phase
Phase 1/2
People taking part
56 people, planned
Started
15 Oct 2024
Expected to end
Jul 2027
Type of study
Interventional, 2 groupsOne group gets a placebo (a dummy treatment), for comparison.
Treatments tested
  • ATX-01
  • Placebo
Record updated
10 February 2026

Who can take part

Age
18 years to 64 years
Sex
Women and men
Healthy volunteers
Cannot take part

The registry criteria, word for word

Show all 6 criteriaShow less

Who can take part

  • 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

Who cannot take part

  • Use of mexiletine or other agent for myotonia within 21 days or 5 half-lives, whichever is longer, prior to screening
  • 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

Check if you might fit

Answer for yourself. This is not a decision: only the study team can say whether you can take part.

  • Are you between 18 and 64 years old?

Where in United States

Also in 6 other countries, 7 sites

  • University of Kansas Medical Center, Department of NeurologyFairway, United StatesContact: Cassidy NelsonTaking people
  • University of FloridaGainesville, United StatesContact: Brandy Donahue-QuailsTaking people
  • University of Iowa Health Care - Department of NeurologyIowa City, United StatesContact: Heena OlaldeTaking people
  • UCLALos Angeles, United StatesContact: Michael YanTaking people
  • Virginia Commonwealth UniversityRichmond, United StatesContact: Jodie HowellTaking people

About the trial

What the trial wants to find out

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.

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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.

The 2 groups in the trial

  • ATX-01Treatment being tested

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

  • PlaceboPlacebo, for comparison

    Placebo to ATX-01

Questions to ask the study team

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?

Contact the study team: see the registry record → Contacts and Locations

Print a page for my doctor

Data and sources

Registry record
Listed on ClinicalTrials.gov and on the EU's official list of trials (CTIS).
Official 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
Fit questions
Built from NCT06300307, who can take part
Record updated
10 February 2026