MedTrace Logo

Study of ATX-01 in Participants With DM1

NCT06300307 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 56

Last updated 2026-02-10

No results posted yet for this study

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.

Conditions

  • Myotonic Dystrophy 1

Interventions

DRUG

ATX-01

Solution for infusion

DRUG

Placebo

Solution for infusion

Sponsors & Collaborators

  • ARTHEx Biotech S.L.

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
64 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-10-15
Primary Completion
2027-07-31
Completion
2027-07-31
FDA Drug
Yes

Countries

  • United States
  • Canada
  • France
  • Italy
  • Netherlands
  • Spain
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06300307 on ClinicalTrials.gov