Not applicableEnded earlyNCT04768348What this trial is testingNatural History Clinical Study in Adult PKUWho this might be right forPhenylketonuriasPKU Homology Medicines, Inc 7
Not applicableLooking for participantsNCT05522374What this trial is testingTIRCON International NBIA RegistryWho this might be right forNeurodegeneration With Brain Iron Accumulation (NBIA)Pantothenate Kinase-associated Neurodegeneration (PKAN)Beta-Propeller Protein-Associated Neurodegeneration (BPAN)+8 more LMU Klinikum 2,000
Not applicableStudy completedNCT05101902What this trial is testingDetermine Eligibility for CAH-301 (A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH) [NCT04783181])Who this might be right forCongenital Adrenal Hyperplasia Adrenas Therapeutics Inc 65
Not applicableStudy completedNCT04237480What this trial is testingInfluence of Kidney Performance and Magnesium on Formation of (OH)2-vitamin D3Who this might be right forHydroxylase Deficiency University Hospital, Basel, Switzerland 50,000
Not applicableUnknownNCT00694525What this trial is testingRole of the Protein Osteoprotegerin in the Bone Health of Women With Congenital Adrenal HyperplasiaWho this might be right forAdrenal Hyperplasia, Congenital Office of Rare Diseases (ORD) 40
Large-scale testing (Phase 3)Active Not RecruitingNCT04806451What this trial is testingGlobal Safety and Efficacy Registration Study of Crinecerfont in Pediatric Participants With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study)Who this might be right forCongenital Adrenal Hyperplasia Neurocrine Biosciences 103
Not applicableApproved For MarketingNCT00484991What this trial is testingSapropterin Expanded Access ProgramWho this might be right forPhenylketonuria BioMarin Pharmaceutical
Early research (Phase 1)Study completedNCT00748059What this trial is testingThe Pathophysiology of Orthostatic HypotensionWho this might be right forAutonomic Nervous System DiseasesOrthostatic HypotensionDopamine Beta-Hydroxylase Deficiency+1 more Vanderbilt University Medical Center 10
Not applicableStudy completedNCT00542841What this trial is testingExamining Genetic Differences Among People With 21-Hydroxylase DeficiencyWho this might be right for21-hydroxylase Deficiency Maria I. New 99
Not applicableLooking for participantsNCT06754423What this trial is testingElectronic Registry of Male Patients With Congenital Adrenal Hyperplasia 21-hydroxylase DeficiencyWho this might be right forCongenital Adrenal Hyperplasia IRCCS Azienda Ospedaliero-Universitaria di Bologna 30
Not applicableStudy completedNCT06624657What this trial is testingHigh-intensity Interval Training and Vitamin D Effects on Bone Metabolism Among Women Diagnosed With OsteoporosisWho this might be right forOsteoporosisExercise OvertrainingVitamin D 25-Hydroxylase Deficiency King Saud University 120
Large-scale testing (Phase 3)Study completedNCT00782340What this trial is testingA Clinical Study for Patients With Neurogenic Orthostatic Hypotension (NOH) Using DroxidopaWho this might be right forSymptomatic Neurogenic Orthostatic Hypotension (NOH)Non-diabetic NeuropathyPrimary Autonomic Failure+1 more Chelsea Therapeutics 263
Post-approval studies (Phase 4)Study completedNCT02586623What this trial is testingSustained Effect of Droxidopa in Symptomatic Neurogenic Orthostatic HypotensionWho this might be right forSymptomatic Neurogenic Orthostatic Hypotension H. Lundbeck A/S 453
Not applicableUnknownNCT01862380What this trial is testingAdrenocortical Functions in Women With Nonclassical 21-hydroxylase Deficiency.Who this might be right forAdrenal Hyperplasia, Congenital21-hydroxylase Deficiency Assistance Publique - Hôpitaux de Paris 41
Not applicableApproved For MarketingNCT00889135What this trial is testingLong Term Treatment With L-DOPSWho this might be right forDopamine Beta Hydroxylase (DBH) Deficiency Vanderbilt University
Not applicableStudy completedNCT02176603What this trial is testingObservational Study of Endothelial Dysfunction in PhenylketonuriaWho this might be right forPhenylketonuriaEndothelial Dysfunction Charite University, Berlin, Germany 51
Testing effectiveness (Phase 2)WithdrawnNCT03548246What this trial is testingAndrogen Reduction in Congenital Adrenal HyperplasiaWho this might be right forCongenital Adrenal Hyperplasia University of Texas Southwestern Medical Center
Not applicableStudy completedNCT05687474What this trial is testingBaby Detect : Genomic Newborn ScreeningWho this might be right forCongenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more Centre Hospitalier Universitaire de Liege 6,824
Large-scale testing (Phase 3)Study completedNCT01132326What this trial is testingClinical Study of Droxidopa in Patients With Neurogenic Orthostatic Hypotension (NOH) (Droxi-304)Who this might be right forPrimary Autonomic FailureDopamine Beta Hydroxylase DeficiencyNon-Diabetic Neuropathy+1 more Chelsea Therapeutics 350
Not applicableUnknownNCT00617292What this trial is testingDetermining the Long-Term Effects of Prenatal Dexamethasone Treatment in Children With 21-Hydroxylase Deficiency and Their MothersWho this might be right forAdrenal Hyperplasia, Congenital Office of Rare Diseases (ORD) 233