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Study of ATX-01 in Participants With DM1 — ArthemiR  

Myotonic Dystrophy 1 Clinical Trial

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

Clinical Trial 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.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT06300307
Study type Interventional
Source ARTHEx Biotech S.L.
Contact Project Manager
Phone +34676229821
Email clinical@arthexbiotech.com
Status Recruiting
Phase Phase 1/Phase 2
Start date May 24, 2024
Completion date December 2025
View Details
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