{"id":"dcr-phxc","safety":{"commonSideEffects":[{"rate":null,"effect":"Injection site reactions"},{"rate":null,"effect":"Transient liver enzyme elevation"},{"rate":null,"effect":"Immune activation (interferon-related)"}]},"_chembl":null,"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"DCR-PHXC uses Dicerna's proprietary dicer-substrate RNA (dsRNA) technology to trigger RNA interference (RNAi) and degrade messenger RNA encoding the target protein. This approach aims to reduce pathogenic protein levels in patients with genetic or acquired diseases driven by PHXC overexpression or dysfunction.","oneSentence":"DCR-PHXC is an RNA interference therapeutic that silences the PHXC gene to reduce production of a disease-causing protein.","_ai_confidence":"low"},"_scrapedAt":"2026-03-28T01:08:43.913Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Phytanic acid storage disease (Refsum disease) or related lipid metabolism disorder"}]},"trialDetails":[{"nctId":"NCT04580420","phase":"PHASE2","title":"Safety & Efficacy of DCR-PHXC in Patients With PH1 and ESRD","status":"RECRUITING","sponsor":"Dicerna Pharmaceuticals, Inc., a Novo Nordisk company","startDate":"2021-04-15","conditions":"Primary Hyperoxaluria Type 1, End Stage Renal Disease","enrollment":28},{"nctId":"NCT05001269","phase":"PHASE2","title":"Nedosiran in Pediatric Patients From Birth to 11 Years of Age With PH and Relatively Intact Renal Function","status":"COMPLETED","sponsor":"Dicerna Pharmaceuticals, Inc., a Novo Nordisk company","startDate":"2022-02-22","conditions":"Primary Hyperoxaluria, Primary Hyperoxaluria Type 1, Primary Hyperoxaluria Type 2","enrollment":27},{"nctId":"NCT04042402","phase":"PHASE3","title":"Long Term Extension Study in Patients With Primary Hyperoxaluria","status":"ACTIVE_NOT_RECRUITING","sponsor":"Dicerna Pharmaceuticals, Inc., a Novo Nordisk company","startDate":"2019-07-09","conditions":"Primary Hyperoxaluria Type 1 (PH1), Primary Hyperoxaluria Type 2 (PH2), Kidney Diseases","enrollment":75},{"nctId":"NCT03392896","phase":"PHASE1","title":"Study of DCR-PHXC-101 in Normal Healthy Volunteers and Patients With Primary Hyperoxaluria","status":"COMPLETED","sponsor":"Dicerna Pharmaceuticals, Inc., a Novo Nordisk company","startDate":"2017-12-06","conditions":"Primary Hyperoxaluria","enrollment":43},{"nctId":"NCT04555486","phase":"PHASE1","title":"Study to Evaluate Safety, Tolerability, PK and PD of DCR-PHXC in PH Type 3 Patients","status":"COMPLETED","sponsor":"Dicerna Pharmaceuticals, Inc., a Novo Nordisk company","startDate":"2020-09-14","conditions":"Primary Hyperoxaluria Type 3","enrollment":6},{"nctId":"NCT03847909","phase":"PHASE2","title":"A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2","status":"COMPLETED","sponsor":"Novo Nordisk A/S","startDate":"2019-10-28","conditions":"Primary Hyperoxaluria Type 1 (PH1), Primary Hyperoxaluria Type 2 (PH2), Kidney Diseases","enrollment":35}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":1,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":["Nedosiran","nedosiran"],"phase":"phase_3","status":"active","brandName":"DCR-PHXC","genericName":"DCR-PHXC","companyName":"Dicerna Pharmaceuticals, Inc., a Novo Nordisk company","companyId":"dicerna-pharmaceuticals-inc-a-novo-nordisk-company","modality":"Small molecule","firstApprovalDate":"","aiSummary":"DCR-PHXC is an RNA interference therapeutic that silences the PHXC gene to reduce production of a disease-causing protein. Used for Phytanic acid storage disease (Refsum disease) or related lipid metabolism disorder.","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":5,"withResults":1},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}