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Recruiting NCT06300307

Study of ATX-01 in Participants With DM1

Phase I / Phase II Interventional Myotonic Dystrophy 1

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: ATX-01, Placebo.
Who it may be relevant to
Registry conditions: Myotonic Dystrophy 1. Basic parameters: 18 years — 64 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Canada, France, Italy, Netherlands +2
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

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

Overview

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.

Interventions

  • Drug ATX-01
    Solution for infusion
  • Drug Placebo
    Solution for infusion

Primary outcome measures

  • Incidence of adverse events [Time frame: Up to 120 days]
Secondary outcome measures (6)
  • Incidence of clinically significant changes in laboratory assessments, electrocardiograms (ECGs), vital signs, suicidal ideation and behavior [Time frame: Up to 120 days]
  • Maximum observed plasma concentration (Cmax) of ATX-01 [Time frame: Up to 48 hours post-dose]
  • Area under the plasma concentration-time curve (AUC) of ATX-01 [Time frame: Up to 48 hours post-dose]
  • Video hand opening time [Time frame: Change from baseline up to 120 days]
  • Change from baseline in ankle dorsiflexion strength by quantitative myometry [Time frame: Change from baseline up to 120 days]
  • Change from baseline in Impact on Activities of Daily Living questionnaire item scores [Time frame: Change from baseline up to 120 days]

Eligibility criteria

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

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Triple blind
Primary purpose
Treatment

Study locations

United States · 5 centers
  • UCLA — Los Angeles
  • University of Florida — Gainesville
  • University of Iowa Health Care - Department of Neurology — Iowa City
  • University of Kansas Medical Center, Department of Neurology — Fairway
  • Virginia Commonwealth University — Richmond
Italy · 2 centers
  • The NeMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan — Milan
  • Fondazione Policlinico A. Gemelli- IRCCS — Rome
Canada · 1 center
  • Centre Intégré Universitaire de Santé et Services Sociaux du Saguenay-Lac-St-Jean — Chicoutimi
France · 1 center
  • Institute of Myology — Paris
Netherlands · 1 center
  • Radboudumc — Nijmegen
Spain · 1 center
  • Hospital Universitario Donostia — Donostia / San Sebastian
United Kingdom · 1 center
  • St. George's University Hospital — London

Identifiers

NCT: NCT06300307 · CT-ATX-01-DM1-1.1

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗