Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
Study of ATX-01 in Participants With DM1
NCT06300307 · View on ClinicalTrials.gov ↗
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 Studied
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
| Field | Value |
|---|---|
| Enrollment Target | 56 participants |
| Start Date | 2024-10-15 |
| Est. Completion | 2027-07 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06300307
The ClinicalTrials.gov registry entry for NCT06300307 describes a study currently listed as recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 56 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is ARTHEx Biotech S.L., which has 1 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT06300307 reports 12 study locations spanning 7 distinct geographic areas — top geographies include Other, California, Florida. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT06300307?
The interventions under investigation include: Placebo (DRUG), ATX-01 (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
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. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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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