Study of ATX-01 in Participants With DM1

Launched by ARTHEX BIOTECH S.L. · Mar 2, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called ATX-01 for people with myotonic dystrophy type 1 (DM1). The main goal is to find out if ATX-01 is safe and how well people can tolerate it. Participants will be given either ATX-01 or a placebo (a treatment that looks like the real drug but has no active ingredients) to compare the effects. The trial has two parts: in the first part, participants will receive one dose, and in the second part, they will receive three doses of ATX-01 or the placebo.

To join the trial, participants need to have a confirmed diagnosis of DM1 and be able to walk without assistance for a short distance. They should also have a certain level of muscle stiffness. However, people with a specific type of DM1 that starts at birth or those with certain muscle weaknesses or treatments in the recent past may not be eligible. If you decide to participate, you can expect careful monitoring and support throughout the trial, helping researchers learn more about this potential new treatment for DM1.

Gender

ALL

Eligibility criteria

• 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