Study of ATX-01 in Participants With DM1

a study on Myotonic Dystrophy

Summary

Eligibility
for people ages 18-64 (full criteria)
Location
at Los Angeles, California and other locations
Dates
study started
study ends around
Principal Investigator
by Perry Shieh, MD, PhD

Description

Summary

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.

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

Keywords

Myotonic Dystrophy 1, Myotonic Dystrophy, ATX-01

Eligibility

You can join if…

Open to people ages 18-64

  • 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

You CAN'T join if...

  • 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

Locations

  • UCLA accepting new patients
    Los Angeles California 90095 United States
  • University of Kansas Medical Center, Department of Neurology accepting new patients
    Fairway Kansas 66205 United States

Lead Scientist at UCLA

  • Perry Shieh, MD, PhD
    HS Clinical Professor, Neurology, Medicine. Authored (or co-authored) 95 research publications

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
ARTHEx Biotech S.L.
Links
Sign up for this study
ID
NCT06300307
Phase
Phase 1/2 Myotonic Dystrophy Research Study
Study Type
Interventional
Participants
Expecting 56 study participants
Last Updated
Contact us about this trial