Not applicableEnrolling By InvitationNCT07159581What this trial is testingGene Therapy for Wilson Disease Evaluated by 64Cu PET/CTWho this might be right forWilson Disease Thomas Damgaard Sandahl 5
Not applicableEnded earlyNCT01550705What this trial is testingEffect of Isoniazid on Protoporphyrin Levels in Erythropoietic ProtoporphyriaWho this might be right forErythropoietic Protoporphyria (EPP)X Linked Erythropoietic Protoporphyria University of Utah 11
Early research (Phase 1)Study completedNCT01874028What this trial is testingAssess the Effects in the Body of a Single Dose of Trientine Dihydrochloride in Wilson's Disease PatientsWho this might be right forWilson's Disease Univar BV 20
Post-approval studies (Phase 4)Study completedNCT05488067What this trial is testingAtorvastatin Therapy on Xanthoma in Alagille SyndromeWho this might be right forAlagille SyndromeAtorvastatinXanthoma Children's Hospital of Fudan University 15
Not applicableTemporarily pausedNCT04306939What this trial is testingGenomic Resources for Enhancing Available Therapies (GREAT1.0) StudyWho this might be right forChronic PancreatitisInflammatory Bowel DiseasesHepatitis+24 more University of Pittsburgh 120,000
Early research (Phase 1)Study completedNCT02452372What this trial is testingGivosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP)Who this might be right forAcute Intermittent Porphyria Alnylam Pharmaceuticals 40
Testing effectiveness (Phase 2)WithdrawnNCT01927913What this trial is testingTreatment of Iron Overload Requiring Chelation TherapyWho this might be right forTransfusional Iron OverloadIron OverloadIron Chelation+4 more Shire
Not applicableEnded earlyNCT04628871What this trial is testingLong Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIXWho this might be right forHemophilia BMucopolysaccharidosis IMucopolysaccharidosis II Sangamo Therapeutics 13
Large-scale testing (Phase 3)Study completedNCT00418795What this trial is testingPorphozym in the Treatment of Acute Attacks in AIPWho this might be right forAcute Intermittent Porphyria Zymenex A/S 36
Early research (Phase 1)Study completedNCT03505853What this trial is testingInvestigate the Interaction Between Givosiran and a 5-probe Drug Cocktail in Patients With Acute Intermittent Porphyria (AIP)Who this might be right forAcute Intermittent Porphyria (AIP)Acute Hepatic Porphyria (AHP)Porphyria, Acute Intermittent+1 more Alnylam Pharmaceuticals 10
Testing effectiveness (Phase 2)Study completedNCT05355766What this trial is testingLong-term Clinical Study of CN128 in Thalassemia With Sever Liver Iron Overloaded PatientsWho this might be right forThalassemiaIron Overload Hangzhou Zede Pharma-Tech Co., Ltd. 16
Testing effectiveness (Phase 2)Study completedNCT03856866What this trial is testingHydroxychloroquine Administration for Reduction of PexophagyWho this might be right forZellweger SyndromePeroxisome Biogenesis Disorders The Hospital for Sick Children 3
Not applicableEnded earlyNCT00001223What this trial is testingDiagnosis and Treatment of Patients With Cystic FibrosisWho this might be right forCystic FibrosisLiver Disease or AbnormalitiesDiabetes+1 more National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) 79
Early research (Phase 1)Study completedNCT02082860What this trial is testingPhase I Gene Therapy Clinical Trial Using the Vector rAAV2/5-PBGD for the Treatment of Acute Intermittent PorphyriaWho this might be right forAcute Intermittent Porphyria Digna Biotech S.L. 8
Not applicableStudy completedNCT04640324What this trial is testingEffect of PNPLA3, TM6SF2 and MBOAT7 Genetic Variants on Non-alcoholic Fatty Liver Disease Therapeutic Outcome.Who this might be right forNon-Alcoholic Fatty Liver DiseaseInsulin Resistance University of Campania Luigi Vanvitelli 92
Testing effectiveness (Phase 2)Study completedNCT00600938What this trial is testingEvaluating Use of Deferasirox as Compared to Deferoxamine in Treating Cardiac Iron OverloadWho this might be right forTransfusional Iron OverloadTransfusional Hemosiderosis Novartis Pharmaceuticals 197
Post-approval studies (Phase 4)UnknownNCT05305872What this trial is testingGandouling in the Treatment of Wilson's DiseaseWho this might be right forWilson's Disease Jun Li 240
Early research (Phase 1)Ended earlyNCT00004386What this trial is testingPhase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase DeficiencyWho this might be right forOrnithine Transcarbamylase Deficiency Disease Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Not applicableStudy completedNCT00001203What this trial is testingDeferoxamine for the Treatment of HemochromatosisWho this might be right forDiabetes MellitusHeart DiseaseHemochromatosis+1 more National Heart, Lung, and Blood Institute (NHLBI) 151
Testing effectiveness (Phase 2)Study completedNCT00362180What this trial is testingMeasure Liver Fat Content After ISIS 301012 (Mipomersen) AdministrationWho this might be right forLipid Metabolism, Inborn ErrorsHyperlipidemiasMetabolic Diseases+11 more Kastle Therapeutics, LLC 38