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NCT06300307 · ClinicalTrials.gov registry record · Phase 1

Study of ATX-01 in Participants With DM1

A Phase 1 study of Myotonic Dystrophy 1, sponsored by ARTHEx Biotech S.L..

Recruiting
Registry status
Phase 1
Development phase
56
Enrollment target
12
Study locations

NCT06300307: Recruiting Phase 1 study of Myotonic Dystrophy 1, sponsored by ARTHEx Biotech S.L..

NCT06300307 is a Phase 1 study of Myotonic Dystrophy 1 that is actively recruiting participants, run by ARTHEx Biotech S.L.. The registered enrollment target is 56 participants, below the 1,099-participant average among 4 other Myotonic Dystrophy 1 trials with a reported enrollment target (95% lower). The trial reports 12 study locations across 7 states. According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT06300307, a Phase 1 study of Myotonic Dystrophy 1, is actively recruiting participants, sponsored by ARTHEx Biotech S.L..

RECRUITING
Registry status
Phase 1
Development phase
56 participants
Enrollment target
12
Study locations

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.

Primary Outcome

To evaluate the safety and tolerability of ATX-01 in adult participants with DM1

Conditions Studied

Myotonic Dystrophy 1

Interventions

  • DRUG Placebo
  • DRUG ATX-01

Study Locations (12)

Other

  • Institute of Myology - Paris
  • The NeMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan - Milan
  • Fondazione Policlinico A. Gemelli- IRCCS - Rome
  • Radboudumc - Nijmegen
  • Hospital Universitario Donostia - Donostia / San Sebastian
  • St. George's University Hospital - London

California

  • UCLA - Los Angeles

Florida

  • University of Florida - Gainesville

Iowa

  • University of Iowa Health Care - Department of Neurology - Iowa City

Kansas

  • University of Kansas Medical Center, Department of Neurology - Fairway

Virginia

  • Virginia Commonwealth University - Richmond

Quebec

  • Centre Intégré Universitaire de Santé et Services Sociaux du Saguenay-Lac-St-Jean - Chicoutimi

Trial Details

FieldValue
Enrollment Target 56 participants
Start Date 2024-10-15
Est. Completion 2027-07
Phase Phase 1
ARTHEx Biotech S.L.

1 total trials

What NCT06300307 shows while recruiting

NCT06300307 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 56 participants, a relatively small participant target, below the 1,099-participant average among 4 other Myotonic Dystrophy 1 trials with a reported enrollment target (95% lower).

The record links to 1 condition, with Myotonic Dystrophy 1 appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.

NCT06300307 lists 12 locations in 7 states (Other, California, Florida).

Frequently Asked Questions

What is clinical trial NCT06300307 about?

NCT06300307 is a clinical study titled "Study of ATX-01 in Participants With DM1". 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 ...

What is the current status of trial NCT06300307?

This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 56 participants. The study started on 2024-10-15. Estimated completion is 2027-07.

What conditions does trial NCT06300307 study?

This clinical trial studies the following conditions: Myotonic Dystrophy 1.

What interventions are being tested in trial NCT06300307?

The interventions under investigation include: Placebo (DRUG), ATX-01 (DRUG).

Who is sponsoring clinical trial NCT06300307?

This trial is sponsored by ARTHEx Biotech S.L., which has 1 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT06300307 being conducted?

This trial has 12 study locations across California, Florida, Iowa, Kansas, Virginia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

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More recruiting context for NCT06300307

Source: ClinicalTrials.gov NCT06300307, the US trial registry maintained by the National Library of Medicine. NCT06300307 (small enrollment · multi site footprint · recruiting) retrieved and formatted by PlainTrial, see methodology.