At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- ATX-01 (drug), Placebo (drug)
- How long the study runs
- Study runs about 33 months (dates as stated)
- About the drug or intervention
- ATX-01 — drug: Solution for infusion · Placebo — drug: Solution for infusion
- Patient visit burden
- Not specified by the sponsor
In plain English
This study is testing a treatment called ATX-01 in people with myotonic dystrophy type 1 (DM1), a condition that causes muscle weakness and stiffness. DM1 is caused by a change in a gene called DMPK. The study is run by ARTHEx Biotech S.L.
Who can take part
- You have a confirmed diagnosis of DM1, shown by a genetic test finding more than 150 CTG repeats in the DMPK gene (tested from a blood sample)
- You can walk or run 10 metres on your own at the screening visit, without aids such as a stick, walker or splints — except ankle-foot splints are allowed
- You have grip myotonia (muscle stiffness when gripping) that lasts more than 3 seconds, confirmed by a central reader
Who may not be able to
- You have congenital DM1 (present from birth)
- You have significant weakness in lifting your foot up at the ankle, or significant thinning of a shin muscle, that would make a muscle biopsy (taking a small sample of muscle) impossible
- You have taken mexiletine or another medicine for muscle stiffness within 21 days or 5 half-lives (whichever is longer) before screening
What taking part involves
- • Taking part involves testing an experimental treatment called ATX-01
- • Not stated — ask the trial team about how the treatment is given and what doses are used
Time commitment: Not stated — ask the trial team about how many visits there are, how long the study lasts, and what taking part involves.
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Testing a treatment
- Ages
- 18 Years to 64 Years
- Who
- All
- Number of participants
- 56
- Started
- 2024-10-15
- Last checked
- 2026-02
Plain English Summary
What is this study?
- • Testing a new treatment for myotonic dystrophy 1
- • Phase1/Phase2 - 56 participants
- • The goal of this clinical trial is to test ATX-01 in participants with myotonic dystrophy type 1 (DM1)
Who can take part?
- • Ages 18 Years to 64 Years
- • Diagnosed with myotonic dystrophy 1
Where?
- • London - St. George's University Hospital
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
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.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 18 Years - 64 Years
- Who can join: All genders
What the study is looking for
- ✓Participants with a documented clinical diagnosis of DM1 (CTG expansion of \>150 repeats in DMPK gene measured in...
- ✓Ambulatory, defined as able to complete a 10-meter walk/run test at screening without the use of assistive devices...
- ✓Presence for \>3 seconds of grip myotonia as confirmed by a central reader
Who cannot take part
- ✗Participants with congenital DM1
- ✗Medical Research Council Muscle Scale score of less than 4 on ankle dorsiflexion or significant tibialis anterior...
- ✗Use of mexiletine or other agent for myotonia within 21 days or 5 half-lives, whichever is longer, prior to screening
See the full criteria
Where Is This Study? (1 UK site)
St. George's University Hospital
London SW17 0RE, United Kingdom
How to Get in Touch
Project Manager
Sponsor contactCONTACT