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Anti-miR

ATX-01 for Myotonic Dystrophy (ArthemiR Trial)

Phase 1 & 2
Recruiting
Research Sponsored by ARTHEx Biotech S.L.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
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
Must not have
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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 120 days

Summary

"This trial will test a new drug called ATX-01 on people with myotonic dystrophy type 1 (DM1) to see if it is safe and well tolerated compared to a placebo

Who is the study for?
This trial is for people with myotonic dystrophy type 1 (DM1) who can walk 10 meters without help (except ankle-foot braces), have a specific genetic marker in their blood, and experience grip myotonia lasting more than three seconds.
What is being tested?
The study tests ATX-01, an experimental drug designed to inhibit a microRNA linked to DM1. Participants will receive either one dose of ATX-01 or placebo in the first part, followed by three doses in the second part.
What are the potential side effects?
Since this is an early-stage trial for ATX-01, potential side effects are being investigated. Common side effects may include reactions at the injection site, fatigue, headache, or nausea.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been diagnosed with DM1, confirmed by a specific genetic test.
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I can walk 10 meters without help, except for ankle-foot braces.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I was born with myotonic dystrophy type 1.
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I cannot move my ankle upwards well or have severe muscle loss in the front of my lower leg.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 120 days
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 120 days for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Incidence of adverse events
Secondary study objectives
Change from baseline in Impact on Activities of Daily Living questionnaire item scores
Change from baseline in ankle dorsiflexion strength by quantitative myometry
Incidence of clinically significant changes in laboratory assessments, electrocardiograms (ECGs), vital signs, suicidal ideation and behavior
+1 more

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: ATX-01Experimental Treatment1 Intervention
ATX-01 is a formulation of the anti-microRNA 23b (anti-miR-23b), known as X82108, a novel type of antisense oligonucleotide
Group II: PlaceboPlacebo Group1 Intervention
Placebo to ATX-01

Find a Location

Who is running the clinical trial?

ARTHEx Biotech S.L.Lead Sponsor
~34 spots leftby Dec 2025
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