[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"ARTHEx Biotech S.L.\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":43},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,1,0,[8],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":27,"conditions":28,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":31,"lastUpdatePostDateStruct":32,"startDateStruct":35,"completionDateStruct":37,"leadSponsor":39,"locationsCount":42},"100539166","phase-1-study-of-atx-01-in-participants-with-dm1-100539166",false,"NCT06300307","Study of ATX-01 in Participants With DM1","A Phase 1\u002F2a Double-Blind, Placebo-controlled, Single- and Multiple Ascending Dose Study to Assess the Safety, Tolerability, PK, PD and Efficacy of IV Administration of ATX-01 In Male and Female Participants Aged 18 to 64 With Classic DM1","ArthemiR","Key Inclusion Criteria:\n\n* Participants with a documented clinical diagnosis of DM1 (CTG expansion of \\>150 repeats in DMPK gene measured in peripheral blood mononuclear cells)\n* Ambulatory, defined as able to complete a 10-meter walk\u002Frun test at screening without the use of assistive devices such as canes, walkers, or orthoses, except for ankle-foot orthoses\n* Presence for \\>3 seconds of grip myotonia as confirmed by a central reader\n\nKey Exclusion Criteria:\n\n* Participants with congenital DM1\n* Medical Research Council Muscle Scale score of less than 4 on ankle dorsiflexion or significant tibialis anterior atrophy that prevents a muscle biopsy\n* Use of mexiletine or other agent for myotonia within 21 days or 5 half-lives, whichever is longer, prior to screening","ALL","18 Years","64 Years",{"count":21,"type":22},56,"ESTIMATED","INTERVENTIONAL",[25,26],"PHASE1","PHASE2","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.\n\nThere 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.\n\nATX-01 is a novel anti-miR (synthetic single stranded oligonucleotide) that inhibits a microRNA called miR-23b.",[29],"Myotonic Dystrophy 1","RECRUITING","2026-02-06",{"date":33,"type":34},"2026-02-10","ACTUAL",{"date":36,"type":34},"2024-10-15",{"date":38,"type":22},"2027-07",{"name":40,"class":41},"ARTHEx Biotech S.L.","INDUSTRY",12,""]