Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1/PHASE2
56 participants
INTERVENTIONAL
2024-10-15
2027-07-31
Brief Summary
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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.
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
TRIPLE
Study Groups
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ATX-01
ATX-01 is a formulation of the anti-microRNA 23b (anti-miR-23b), known as X82108, a novel type of antisense oligonucleotide
ATX-01
Solution for infusion
Placebo
Placebo to ATX-01
Placebo
Solution for infusion
Interventions
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ATX-01
Solution for infusion
Placebo
Solution for infusion
Eligibility Criteria
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Inclusion Criteria
* 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 orthoses, except for ankle-foot orthoses
* Presence for \>3 seconds of grip myotonia as confirmed by a central reader
Exclusion Criteria
* Medical Research Council Muscle Scale score of less than 4 on ankle dorsiflexion or significant tibialis anterior atrophy that prevents a muscle biopsy
* Use of mexiletine or other agent for myotonia within 21 days or 5 half-lives, whichever is longer, prior to screening
18 Years
64 Years
ALL
No
Sponsors
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ARTHEx Biotech S.L.
INDUSTRY
Responsible Party
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Locations
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UCLA
Los Angeles, California, United States
University of Florida
Gainesville, Florida, United States
University of Kansas Medical Center, Department of Neurology
Fairway, Kansas, United States
Virginia Commonwealth University
Richmond, Virginia, United States
Centre Intégré Universitaire de Santé et Services Sociaux du Saguenay-Lac-St-Jean
Chicoutimi, Quebec, Canada
Institute of Myology
Paris, , France
The NeMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan
Milan, , Italy
Fondazione Policlinico A. Gemelli- IRCCS
Rome, , Italy
Radboudumc
Nijmegen, , Netherlands
Hospital Universitario Donostia
Donostia / San Sebastian, , Spain
St. George's University Hospital
London, , United Kingdom
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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CT-ATX-01-DM1-1.1
Identifier Type: -
Identifier Source: org_study_id
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