56 Participants Needed

ATX-01 for Myotonic Dystrophy

(ArthemiR Trial)

Recruiting at 8 trial locations
PM
Overseen ByProject Manager
Age: 18 - 65
Sex: Any
Trial Phase: Phase 1 & 2
Sponsor: ARTHEx Biotech S.L.
Approved in 2 JurisdictionsThis treatment is already approved in other countries

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

I can walk 10 meters without help, except for ankle-foot braces.
Presence for >3 seconds of grip myotonia as confirmed by a central reader
I have been diagnosed with DM1, confirmed by a specific genetic test.

Exclusion Criteria

I was born with myotonic dystrophy type 1.
I cannot move my ankle upwards well or have severe muscle loss in the front of my lower leg.
I haven't taken mexiletine or similar drugs for myotonia in the last 21 days or 5 half-lives, whichever is longer.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Single-Ascending Dose

Participants receive one dose of ATX-01 or placebo

1 week

Multiple-Ascending Dose

Participants receive three doses of ATX-01 or placebo

3 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

16 weeks

Treatment Details

Interventions

  • ATX-01
Trial Overview 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.
Participant Groups
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

ATX-01 is already approved in United States, European Union for the following indications:

🇺🇸
Approved in United States as ATX-01 for:
  • None approved; Orphan Drug Designation for Myotonic Dystrophy Type 1 (DM1)
🇪🇺
Approved in European Union as ATX-01 for:
  • 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

Trials
1
Recruited
60+

Findings from Research

A nationwide registry for myotonic dystrophies (DM) in Japan has successfully enrolled 976 patients, primarily with DM1, providing a crucial resource for future clinical trials and therapeutic development.
The study found that longer CTG repeat lengths in DM1 patients are associated with earlier disease onset and significantly influence clinical outcomes like grip strength and lung function, highlighting the importance of genetic factors in disease progression.
Characteristics of myotonic dystrophy patients in the national registry of Japan.Sugimoto, M., Kuru, S., Takada, H., et al.[2022]
In a study of 46 patients with myotonic dystrophy type 1 (DM1), the Muscular Impairment Rating Scale (MIRS) showed significant correlations with myopathological changes, particularly in nuclear translocation and ragged red fibers, indicating that MIRS can reflect underlying muscle pathology.
The findings suggest that while MIRS is associated with certain myopathological features, it does not correlate with all aspects of muscle fiber changes, such as connective tissue proliferation, highlighting its specific utility in assessing DM1 severity.
[Correlation of muscular impairment rating scale with myopathological changes in myotonic dystrophy type 1].Wang, W., Liu, Q., Wang, ZX., et al.[2014]
Myotonic dystrophy type 1 (DM1) affects at least half a million people and has various therapeutic approaches currently under development, with nearly two dozen drugs being explored.
Among these, the repurposed drugs tideglusib, mexiletine, and metformin show the most promise for potential marketing authorization, with eight therapies already in clinical trials.
Myotonic dystrophy type 1 drug development: A pipeline toward the market.Pascual-Gilabert, M., López-Castel, A., Artero, R.[2022]

References

Characteristics of myotonic dystrophy patients in the national registry of Japan. [2022]
[Correlation of muscular impairment rating scale with myopathological changes in myotonic dystrophy type 1]. [2014]
Myotonic dystrophy type 1 drug development: A pipeline toward the market. [2022]
Assessing upper extremity capacity as a potential indicator of needs related to household activities for rehabilitation services in people with myotonic dystrophy type 1. [2015]
Functioning and disability in adults with myotonic dystrophy type 1. [2015]
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