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New treatment option for Myotonic Dystrophy 1
Official title Study of ATX-01 in Participants With DM1
ClinicalTrials.gov ID: NCT06300307
What this study is testing
What is ATX-01?
ATX-01 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for myotonic dystrophy 1.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
- You might receive a placebo (an inactive treatment) instead of the study drug.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 64
You may be able to join if
- 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...
- Presence for \>3 seconds of grip myotonia as confirmed by a central reader Key
You likely can't join if
- 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...
- Use of mexiletine or other agent for myotonia within 21 days or 5 half-lives, whichever is longer, prior to screening
The study team makes the final eligibility decision.
Where it's taking place
- Los Angeles, California, United States
- Gainesville, Florida, United States
- Iowa City, Iowa, United States
- Fairway, Kansas, United States
- Richmond, Virginia, United States
- Chicoutimi, Quebec, Canada
- Paris, France
- Milan, Italy
- Rome, Italy
- Nijmegen, Netherlands
- Donostia / San Sebastian, Spain
- London, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Los Angeles, California, United States; Gainesville, Florida, United States; Iowa City, Iowa, United States; Fairway, Kansas, United States; Richmond, Virginia, United States; Chicoutimi, Quebec, Canada and 6 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.