[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100602427":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":21,"centralContacts":26,"locations":26,"responsibleParty":27,"collaborators":26,"id":29,"slug":30,"hasResults":31,"nctId":32,"briefTitle":33,"officialTitle":33,"acronym":26,"eligibilityCriteria":34,"healthyVolunteers":31,"sex":35,"minAge":36,"maxAge":37,"enrollmentInfo":38,"targetDuration":26,"studyType":41,"phases":42,"briefSummary":44,"conditions":45,"keywords":47,"overallStatus":51,"whyStopped":52,"lastUpdateSubmitDate":53,"lastUpdatePostDateStruct":54,"startDateStruct":56,"completionDateStruct":59,"leadSponsor":61,"locationsCount":26},{"fullName":5,"class":6},"Wake Forest University Health Sciences","OTHER",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"bitopertin","EXPERIMENTAL","bitopertin 60 mg per day",[13],"Drug: bitopertin",[15],{"type":16,"name":9,"description":17,"armGroupLabels":18,"otherNames":19},"DRUG","bitopertin 60 mg will be taken once daily by mouth",[9],[20],"DISC-1459",[22],{"name":23,"affiliation":24,"role":25},"Herbert Bonkovsky, MD","Atrium Health Wake Forest Baptist","PRINCIPAL_INVESTIGATOR",null,{"type":28,"investigatorFullName":26,"investigatorTitle":26,"investigatorAffiliation":26,"oldNameTitle":26,"oldOrganization":26},"SPONSOR","100602427","phase-1-effect-of-bitopertin-on-the-liver-and-on-levels-of-protoporphyrin-ix-in-bile-blood-liver-and-stool-in-patients-with-erythropoietic-protoporphyriax-linked-protoporphyria-and-increased-liver-stiffness-andor-liver-enzymes-at-baseline-100602427",false,"NCT07123363","Effect of Bitopertin on the Liver and on Levels of Protoporphyrin IX in Bile, Blood, Liver, and Stool in Patients With Erythropoietic Protoporphyria\u002FX-linked Protoporphyria and Increased Liver Stiffness and\u002For Liver Enzymes at Baseline","Inclusion Criteria:\n\n* Diagnosis of EEP or XLP that has been biochemically and genetically verified prior diagnosis of EPP or XLP\n* Age 18-75 years\n* Increased liver stiffness at baseline, defined as stiffness measurement \\[E value\\] by: Fibroscan \\>7 kPa; MRE that is \\>3.0 kPa; Velacur\u002FSonicincyte that is \\>6.0 kPa; and\u002For elevated liver enzymes: serum ALT \\> 2 X upper limit of normal (ULN), but not greater than 5 X ULN and\u002For serum AP \\> 2 X ULN, but not greater than 5 X ULN\n* INR \\\u003C 1.4\n* Compensated liver disease at baseline, defined as lack of clinically evident ascites, encephalopathy, hepatocellular carcinoma, clinically evident icterus or jaundice, peripheral edema.\n* Willingness and ability to volunteer and provide informed consent for a long-term study that will include liver biopsies and collection of bile from the second portion of the duodenum, at baseline and at the end of study. In addition, follow-up visits will be planned every 26 weeks throughout the study, with plans for repeat routine\u002Fsafety labs at each visit and for measures of liver stiffness and markers of hepatic status and fibrosis (Fib-4, APRI, ELF, Fibrotest) performed once every 6 months.\n\nExclusion Criteria:\n\n* Chronic hepatitis B, C, D, or E;\n* Human immunodeficiency (HIV) infection;\n* Alcohol use \\> 14 units\u002Fweek for men or \\> 7 units\u002Fweek for women;\n* Pregnancy or breast feeding among women;\n* Any known active malignancy other than small and localized squamous or basal cell carcinoma of the skin;\n* Advanced or decompensated heart, lung, kidney, liver, or neuro-psychiatric disease;\n* History of diagnosed depression or suicidality;\n* History of diagnosed substance abuse or poor impulse control;\n* Any other conditions that, in the opinion of the Investigator, renders the individual unfit to participate","ALL","18 Years","75 Years",{"count":39,"type":40},0,"ACTUAL","INTERVENTIONAL",[43],"PHASE1","The primary goal of this study is to assess safety and tolerability of bitopertin in subjects with Erythropoietic Protoporphyria (EPP) or X-linked Protoporphyria (XLP) and evidence of compensated liver disease.",[46],"Protoporphyria",[48,49,50],"Erythropoietic Protoporphyria","X-linked Protoporphyria","Bitopertin","WITHDRAWN","department leadership decided not to proceed with study","2026-01-26",{"date":55,"type":40},"2026-01-29",{"date":57,"type":58},"2026-01","ESTIMATED",{"date":60,"type":58},"2035-12",{"name":5,"class":6}]