Large-scale testing (Phase 3)Study completedNCT05166161What this trial is testingA Long-Term Safety Study of PTC923 in Participants With PhenylketonuriaWho this might be right forPhenylketonuria PTC Therapeutics 247
Not applicableStudy completedNCT04196959What this trial is testingEvaluation of TYR SphereWho this might be right forTyrosinemiasAlkaptonuriaTyrosinemia, Type I+2 more Vitaflo International, Ltd 18
Testing effectiveness (Phase 2)Ended earlyNCT01395394What this trial is testingPhenylketonuria, Oxidative Stress, and BH4Who this might be right forPhenylketonuria Emory University 12
Testing effectiveness (Phase 2)Study completedNCT00483314What this trial is testingHomocystinuria: Treatment With N-AcetylcysteineWho this might be right forHomocystinuria McGill University Health Centre/Research Institute of the McGill University Health Centre 5
Early research (Phase 1)Looking for participantsNCT07446400What this trial is testingA Trial to Examine the Interaction of Repinatrabit With Ethinyl Estradiol/Norethindrone, Metformin,Carbamazepine, Rosuvastatin, and Methotrexate When Administered TogetherWho this might be right forHealthy Volunteers Otsuka Pharmaceutical Development & Commercialization, Inc. 48
Not applicableStudy completedNCT00730080What this trial is testingSapropterin in Individuals With PhenylketonuriaWho this might be right forPhenylketonuria Washington University School of Medicine 45
Not applicableUnknownNCT04251494What this trial is testingA Study Into the Future Cardiovascular Disease (CVD) Risk of Phenylketonuria (PKU) Patients on a Low Phenylalanine Treatment Diet (LPD).Who this might be right forPhenylketonuriasCardiovascular Diseases Guy's and St Thomas' NHS Foundation Trust 32
Testing effectiveness (Phase 2)Study completedNCT06637514What this trial is testingJNT-517 in Adolescent Participants With PhenylketonuriaWho this might be right forPhenylketonuria (PKU) Otsuka Pharmaceutical Development & Commercialization, Inc. 14
Large-scale testing (Phase 3)Study completedNCT02323529What this trial is testingEfficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1Who this might be right forHereditary Tyrosinemia, Type I Swedish Orphan Biovitrum 18
Not applicableStudy completedNCT00744588What this trial is testingStress, Hypothalamic-pituitary-adrenal (HPA) Dysfunction, and Relapse in AlcoholismWho this might be right forAlcohol Dependence University of Texas Southwestern Medical Center 75
Not applicableUnknownNCT02978469What this trial is testingGo For It: to Improve Levels of Daily Physical Activity and to Reduce the Sedentary Life Style of Adolescents With CPWho this might be right forCerebral Palsy (CP) Assaf-Harofeh Medical Center 120
Testing effectiveness (Phase 2)Looking for participantsNCT05130437What this trial is testingAssess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 StudyWho this might be right forPropionic Acidemia ModernaTX, Inc. 50
Post-approval studies (Phase 4)Study completedNCT05539027What this trial is testingEfficacy of L-Ornithine L-Aspartate (LOLA) as an Adjunct to Branched Chain Amino Acids (BCAA) Enriched Solutions on Clinical Outcomes in ICU Patients With Hepatic EncephalopathyWho this might be right forHepatic Encephalopathy Ain Shams University 140
Early research (Phase 1)Ended earlyNCT04836494What this trial is testingA First in Human, Dose Escalation Study to Evaluate the Safety and Tolerability of BBP-671 in Healthy Volunteers and Patients With Propionic Acidemia or Methylmalonic AcidemiaWho this might be right forHealthy VolunteersPropionic AcidemiaMethylmalonic Acidemia+1 more CoA Therapeutics, Inc., a BridgeBio company 79
Not applicableStudy completedNCT00909012What this trial is testingQuantitative Requirements of Docosahexaenoic Acid for Neural Function in Children With PhenylketonuriaWho this might be right forPhenylketonuria Ludwig-Maximilians - University of Munich 114
Not applicableLooking for participantsNCT06953505What this trial is testingAt Home Ammonia Monitoring of Inborn Errors of Ammonia MetabolismWho this might be right forUrea Cycle DisordersOrganic AcidemiasFatty Acid Oxidation Disorder+1 more Sequitur Health Corp. 30
Post-approval studies (Phase 4)Study completedNCT00373165What this trial is testingProphylaxis With Ganciclovir Improves Graft Survival in Renal Allograft RecipientsWho this might be right forDNA Virus InfectionHerpesviridae InfectionsCytomegalovirus Infection Lower Saxony Center for Nephrology 150
Not applicableStudy completedNCT00000665What this trial is testingStudies of the Ocular Complications of AIDS (SOCA) CMV Retinitis Trial: Foscarnet-Ganciclovir ComponentWho this might be right forCytomegalovirus RetinitisHIV Infections National Institute of Allergy and Infectious Diseases (NIAID) 240
Not applicableStudy completedNCT03419819What this trial is testingEvaluation of PKU SphereWho this might be right forPhenylketonurias Vitaflo International, Ltd 28
Not applicableStudy completedNCT04679467What this trial is testingEvaluation of PKU Sphere in ItalyWho this might be right forPhenylketonuriasPKU Vitaflo International, Ltd 13