{"id":"gtx-102","safety":{"commonSideEffects":[{"rate":null,"effect":"Injection site reactions"},{"rate":null,"effect":"Headache"},{"rate":null,"effect":"Flu-like symptoms"},{"rate":null,"effect":"Elevated liver enzymes"}]},"_chembl":null,"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"GTX-102 works by binding to FUS (fused in sarcoma) mRNA and promoting its degradation through RNase H-mediated cleavage, thereby reducing pathogenic FUS protein levels. This approach targets the underlying genetic cause in FUS-related diseases, particularly FUS-associated amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). By lowering FUS protein expression, the drug aims to slow or halt neurodegeneration caused by FUS mutations.","oneSentence":"GTX-102 is an antisense oligonucleotide that reduces production of FUS protein by targeting FUS mRNA in patients with FUS-associated neurological disease.","_ai_confidence":"medium"},"_scrapedAt":"2026-03-28T01:14:45.135Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"FUS-associated amyotrophic lateral sclerosis (ALS)"},{"name":"FUS-associated frontotemporal dementia (FTD)"}]},"trialDetails":[{"nctId":"NCT07157254","phase":"PHASE2","title":"A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)","status":"RECRUITING","sponsor":"Ultragenyx Pharmaceutical Inc","startDate":"2025-10-13","conditions":"Angelman Syndrome","enrollment":60},{"nctId":"NCT06617429","phase":"PHASE3","title":"Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)","status":"ACTIVE_NOT_RECRUITING","sponsor":"Ultragenyx Pharmaceutical Inc","startDate":"2024-12-03","conditions":"Angelman Syndrome","enrollment":129},{"nctId":"NCT04259281","phase":"PHASE1, PHASE2","title":"A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome","status":"COMPLETED","sponsor":"Ultragenyx Pharmaceutical Inc","startDate":"2020-02-24","conditions":"Angelman Syndrome","enrollment":74},{"nctId":"NCT06415344","phase":"PHASE3","title":"Long-term Extension of GTX-102 in Angelman Syndrome","status":"ENROLLING_BY_INVITATION","sponsor":"Ultragenyx Pharmaceutical Inc","startDate":"2024-07-31","conditions":"Angelman Syndrome","enrollment":255},{"nctId":"NCT05531890","phase":"PHASE1","title":"Comparative Bioavailability of Betamethasone Oral Solution Metered Spray (GTX-102) in Healthy Subjects","status":"UNKNOWN","sponsor":"Grace Therapeutics Inc.","startDate":"2022-09-13","conditions":"Ataxia Telangiectasia","enrollment":48}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":0,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":["apazunersen"],"phase":"phase_3","status":"active","brandName":"GTX-102","genericName":"GTX-102","companyName":"Ultragenyx Pharmaceutical Inc","companyId":"ultragenyx-pharmaceutical-inc","modality":"Small molecule","firstApprovalDate":"","aiSummary":"GTX-102 is a small molecule that acts as an estrogen receptor alpha agonist. It is being studied in a Phase 3 clinical trial for the treatment of Angelman Syndrome and Ataxia Telangiectasia.","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":1,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}