ATX-01 for Myotonic Dystrophy
(ArthemiR Trial)
Trial Summary
What is the purpose of this trial?
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.
Will I have to stop taking my current medications?
The trial requires that you stop using mexiletine or any other medication for myotonia at least 21 days before screening.
How is the drug ATX-01 different from other treatments for myotonic dystrophy?
ATX-01 is a novel treatment being explored for myotonic dystrophy, a condition with limited existing therapies. Unlike other treatments, ATX-01 may offer a new approach, potentially involving unique mechanisms or formulations, as current options like tideglusib, mexiletine, or metformin are repurposed drugs.12345
Eligibility Criteria
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.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Single-Ascending Dose
Participants receive one dose of ATX-01 or placebo
Multiple-Ascending Dose
Participants receive three doses of ATX-01 or placebo
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- ATX-01
ATX-01 is already approved in United States, European Union for the following indications:
- None approved; Orphan Drug Designation for Myotonic Dystrophy Type 1 (DM1)
- None approved; Orphan Drug Designation for Myotonic Dystrophy Type 1 (DM1)
Find a Clinic Near You
Who Is Running the Clinical Trial?
ARTHEx Biotech S.L.
Lead Sponsor