Early research (Phase 1)Ended earlyNCT04110496What this trial is testingSafety and Tolerability of RTX-134 in Adults With PhenylketonuriaWho this might be right forPhenylketonurias Rubius Therapeutics 1
Not applicableStudy completedNCT06694662What this trial is testingPKU EASY Microtabs Plus - Acceptability and ToleranceWho this might be right forPhenylketonuria (PKU) Galen Limited 10
Not applicableStudy completedNCT03323307What this trial is testingLow Cost OCT for Point of CareWho this might be right forOptical Coherence Tomography (OCT) of the Retina Duke University 70
Not applicableNot Yet RecruitingNCT06929637What this trial is testingAmino Acid Infusion in Kidney Transplant RecipientsWho this might be right forShort-term Graft FunctionMetabolic EffectsNutritional Effect+3 more University Hospital, Martin 100
Not applicableLooking for participantsNCT01659749What this trial is testingEducational, Social Support, and Nutritional Interventions and Their Cumulative Effect on Pregnancy Outcomes and Quality of Life in Teen and Adult Women With PhenylketonuriaWho this might be right forPregnancyPhenylketonuria Emory University 200
Not applicableWithdrawnNCT06227429What this trial is testingA Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 PatientsWho this might be right forHereditary Tyrosinemia, Type I Swedish Orphan Biovitrum
Early research (Phase 1)UnknownNCT00004307What this trial is testingStudy of Treatment and Metabolism in Patients With Urea Cycle DisordersWho this might be right forAmino Acid Metabolism, Inborn Errors National Center for Research Resources (NCRR) 66
Not applicableStudy completedNCT00808106What this trial is testingClinical, Cellular, and Molecular Investigation Into Oculocutaneous AlbinismWho this might be right forOculocutaneous Albinism National Human Genome Research Institute (NHGRI) 206
Large-scale testing (Phase 3)Study completedNCT00814073What this trial is testingMasitinib in Severe Indolent or Smoldering Systemic MastocytosisWho this might be right forIndolent Systemic Mastocytosis AB Science 135
Not applicableEnded earlyNCT03343756What this trial is testingHepaStem Long-Term Safety RegistryWho this might be right forUrea Cycle DisorderCrigler-Najjar SyndromeAcute on Chronic Liver Failure Cellaion SA 44
Post-approval studies (Phase 4)Study completedNCT02677870What this trial is testingThe Effectiveness of Kuvan in Amish PKU PatientsWho this might be right forPhenylketonuria University Hospitals Cleveland Medical Center 7
Not applicableUnknownNCT05827666What this trial is testingThe Effect of Dairy and Dairy-Free Alternative Beverages on Post-Exercise Anabolism in Active YouthWho this might be right forDietary ProteinsIndicator Amino Acid Oxidation University of Toronto 24
Not applicableLooking for participantsNCT07082309What this trial is testingThe Effect of Ketone Monoesters on Skeletal Muscle Protein Synthesis and Whole-body Protein Metabolism.Who this might be right forHealthy Participants University of Exeter 12
Not applicableLooking for participantsNCT05769621What this trial is testingA Retrospective Study to Characterize Participants With Propionic AcidemiaWho this might be right forPropionic Acidemia ModernaTX, Inc. 60
Not applicableStudy completedNCT00031421What this trial is testingNeonatal CMV-Ganciclovir Follow-up StudyWho this might be right forCytomegalovirus Infections National Institute of Allergy and Infectious Diseases (NIAID) 8
Early research (Phase 1)Study completedNCT02750332What this trial is testingBioavailability Food-Effect Study of an Oral Nitisinone Formulation to Treat Hereditary Tyrosinemia (HT-1)Who this might be right forHereditary Tyrosinemia, Type I Cycle Pharmaceuticals Ltd. 20
Not applicableStudy completedNCT00002299What this trial is testingThe Safety and Effectiveness of Ganciclovir Plus Interferon Beta in Preventing the Return of Cytomegalovirus (CMV) of the Eyes in Patients With AIDSWho this might be right forCytomegalovirus RetinitisHIV Infections Hoffmann-La Roche
Not applicableStudy completedNCT02157844What this trial is testingFat Metabolism in OSA and COPDWho this might be right forObstructive Sleep ApneaChronic Obstructive Pulmonary DiseaseObesity Texas A&M University 62
Post-approval studies (Phase 4)UnknownNCT01640678What this trial is testingAutologous Cell Suspension Grafting Using ReCell in Vitiligo and Piebaldism PatientsWho this might be right forPiebaldismSegmental Vitiligo Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) 10
Early research (Phase 1)Study completedNCT00001328What this trial is testingGene Therapy for the Treatment of Brain TumorsWho this might be right forBrain NeoplasmNeoplasm Metastasis National Institute of Neurological Disorders and Stroke (NINDS) 15