[{"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:":42},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,1,0,[8],{"id":9,"slug":4,"hasResults":10,"nctId":11,"briefTitle":12,"officialTitle":13,"acronym":14,"eligibilityCriteria":15,"healthyVolunteers":10,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":26,"conditions":27,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":30,"lastUpdatePostDateStruct":31,"startDateStruct":34,"completionDateStruct":36,"leadSponsor":38,"locationsCount":41},"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":20,"type":21},56,"ESTIMATED","INTERVENTIONAL",[24,25],"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.",[28],"Myotonic Dystrophy 1","RECRUITING","2026-02-06",{"date":32,"type":33},"2026-02-10","ACTUAL",{"date":35,"type":33},"2024-10-15",{"date":37,"type":21},"2027-07",{"name":39,"class":40},"ARTHEx Biotech S.L.","INDUSTRY",12,""]