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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
An Extension Trial of Asedebart in Participants With Congenital Adrenal HyperplasiaCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenogenital syndrome, congenital adrenal hyperplasia, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, metabolic disease, steroid inherited metabolic disorderNot yet recruitingPhase 22026-10-05NCT07859293
Efficacy, Safety, Tolerability, and Pharmacokinetics of NBIP-01435 in Adult Participants With Classic Congenital Adrenal Hyperplasia (CAH)Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenogenital syndrome, congenital adrenal hyperplasia, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, metabolic disease, steroid inherited metabolic disorderNot yet recruitingPhase 22026-09-30NCT07850739
A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenogenital syndrome, classic congenital adrenal hyperplasia, congenital adrenal hyperplasia, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, metabolic disease, steroid inherited metabolic disorderRecruitingPhase 2 / Phase 32026-09-30NCT07159841
A Study to Evaluate Atumelnant in Adults With Congenital Adrenal HyperplasiaCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenogenital syndrome, classic congenital adrenal hyperplasia, congenital adrenal hyperplasia, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, metabolic disease, steroid inherited metabolic disorderRecruitingPhase 32026-09-30NCT07144163
Metyrapone Study for Patients Diagnosed With Mild Autonomous Cortisol Secretion - MACSCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cushing syndrome, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Paraneoplastic Endocrine Syndromes, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenal gland hyperfunction, adrenogenital syndrome, congenital adrenal hyperplasia, disorder of sexual differentiation, ectopic ACTH secretion syndrome, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, metabolic disease, neoplasm, paraneoplastic syndrome, steroid inherited metabolic disorderRecruitingPhase 22026-09-25NCT07138274
Metyrapone for Mild Autonomous Cortisol Secretion (MACS)Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cushing syndrome, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Paraneoplastic Endocrine Syndromes, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenal gland hyperfunction, adrenogenital syndrome, congenital adrenal hyperplasia, disorder of sexual differentiation, ectopic ACTH secretion syndrome, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, metabolic disease, neoplasm, paraneoplastic syndrome, steroid inherited metabolic disorderActive, not recruitingPhase 22026-09-25NCT06106295
Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Crinecerfont in Participants With Classic Congenital Adrenal Hyperplasia (CAH) Who Are Less Than 4 Years OldCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenogenital syndrome, congenital adrenal hyperplasia, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, metabolic disease, steroid inherited metabolic disorderRecruitingPhase 22026-09-16NCT07536269
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of OMS1620Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenogenital syndrome, congenital adrenal hyperplasia, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, metabolic disease, steroid inherited metabolic disorderNot yet recruitingPhase 12026-09-09NCT07811089
Natural History Study of Patients With Excess AndrogenCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Familial Testotoxicosis, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenocortical insufficiency, adrenogenital syndrome, classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency, congenital adrenal hyperplasia, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, metabolic disease, precocious puberty, steroid inherited metabolic disorderRecruitingNot stated2026-09-08NCT00250159
Parenting and CAH - 21-hydroxylase DeficiencyCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenogenital syndrome, congenital adrenal hyperplasia, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, metabolic disease, steroid inherited metabolic disorderRecruitingNot stated2026-08-31NCT06900153
Pharmacokinetics, Safety and Tolerability of Crinecerfont in Participants With Congenital Adrenal Hyperplasia Who Are Less Than 2 Years OldCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenogenital syndrome, congenital adrenal hyperplasia, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, metabolic disease, steroid inherited metabolic disorderActive, not recruitingPhase 22026-08-28NCT07187375
Pediatric Congenital Adrenal Hyperplasia Management: Regret and Long-term OutcomesCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenogenital syndrome, classic congenital adrenal hyperplasia, classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency, congenital adrenal hyperplasia, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, metabolic disease, steroid inherited metabolic disorderRecruitingNot stated2026-08-24NCT07781267
Development of Healthcare Transition for Patients With Congenital Adrenal HyperplasiaCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenogenital syndrome, congenital adrenal hyperplasia, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, metabolic disease, steroid inherited metabolic disorderNot yet recruitingNot stated2026-08-21NCT07611786
Evaluation of Circadian Hormonal Balance: LC-MS/MS Measurement of Salivary and Serum Steroids in Patients With Congenital Adrenal Hyperplasia46,XX disorder of sex development, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hyperandrogenism, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenocortical insufficiency, adrenogenital syndrome, classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency, congenital adrenal hyperplasia, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, metabolic disease, steroid inherited metabolic disorderNot yet recruitingNot stated2026-07-21NCT07718230
An Extension Study to Evaluate Safety and Efficacy of Atumelnant in Participants With Congenital Adrenal HyperplasiaCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenogenital syndrome, classic congenital adrenal hyperplasia, congenital adrenal hyperplasia, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, metabolic disease, steroid inherited metabolic disorderRecruitingPhase 22026-06-29NCT06712823
Hydrocortisone Modified-release in Adults: Real-world Monitoring of Longitudinal Outcomes in coNgenital Adrenal hYperplasiaCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenogenital syndrome, congenital adrenal hyperplasia, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, metabolic disease, steroid inherited metabolic disorderNot yet recruitingNot stated2026-06-08NCT07622030
Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal HyperplasiaCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenogenital syndrome, congenital adrenal hyperplasia, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, metabolic disease, steroid inherited metabolic disorderActive, not recruitingPhase 32026-05-11NCT04490915
Syndromes With Neonatal Salt Loss: Not Only Congenital Adrenal Hyperplasia Due to 21-hydroxylase Deficiency (21OH-ISC)Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenogenital syndrome, congenital adrenal hyperplasia, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, metabolic disease, steroid inherited metabolic disorderRecruitingNot stated2026-03-16NCT07473804
A Trial of Lu AG13909 in Participants With Congenital Adrenal HyperplasiaCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenogenital syndrome, congenital adrenal hyperplasia, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, metabolic disease, steroid inherited metabolic disorderRecruitingPhase 1 / Phase 22026-03-09NCT05669950
Isturisa Treatment in Mild Autonomous Cortisol Secretion( MACS)Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Paraneoplastic Endocrine Syndromes, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenogenital syndrome, cardiovascular disorder, congenital adrenal hyperplasia, disorder of sexual differentiation, ectopic ACTH secretion syndrome, endocrine system disorder, gonadal disorder, hereditary disease, hypertensive disorder, inborn errors of metabolism, metabolic disease, neoplasm, paraneoplastic syndrome, steroid inherited metabolic disorder, vascular disorderRecruitingPhase 42026-03-05NCT07247058
Impact of 1 mg Osilodrostat Therapy on Mild Autonomous Cortisol Secretion (MACS)Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cushing syndrome, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Paraneoplastic Endocrine Syndromes, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenal gland hyperfunction, adrenogenital syndrome, congenital adrenal hyperplasia, disorder of sexual differentiation, ectopic ACTH secretion syndrome, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, metabolic disease, neoplasm, paraneoplastic syndrome, steroid inherited metabolic disorderRecruitingPhase 22026-02-11NCT07104812
Institutional Registry of Rare DiseasesAbnormalities, Multiple, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cardiovascular Abnormalities, Ciliopathies, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cushing syndrome, Digestive System Abnormalities, Digestive System Diseases, Disease Attributes, Eosinophilia, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Gaucher disease, Hemic and Lymphatic Diseases, Hereditary Complement Deficiency Diseases, Hirschsprung disease, Hypersensitivity, Delayed, Hypersensitivity, Immediate, Immunoglobulin G4-Related Disease, Imprinting Disorders, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Male Urogenital Diseases, Mast Cell Activation Disorders, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Connective and Soft Tissue, Neoplasms, Germ Cell and Embryonal, Neoplasms, Multiple Primary, Neoplasms, Nerve Tissue, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Overweight, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Prader-Willi syndrome, Primary Immunodeficiency Diseases, Rare Diseases, Skin Abnormalities, Skin and Connective Tissue Diseases, Urogenital Abnormalities, Urogenital Diseases, Wilson disease, adrenal gland disorder, adrenal gland hyperfunction, adrenogenital syndrome, amyloidosis, angioedema, angiomatosis, aortic valve disorder, aortic valve stenosis, autoimmune disease, basal ganglia disorder, brain disorder, cardiomyopathy, cardiovascular disorder, central nervous system disorder, chromosomal disorder, colonic disorder, congenital adrenal hyperplasia, connective tissue neoplasm, demyelinating disease, digestive system disorder, disorder of sexual differentiation, ectodermal dysplasia syndrome, endocrine system disorder, eosinophilic esophagitis, esophageal disorder, esophagitis, gastroenteritis, gonadal disorder, heart disorder, heart valve disorder, hematologic disorder, hemorrhagic disease, hereditary angioedema, hereditary disease, hereditary hemorrhagic telangiectasia, hereditary neoplastic syndrome, hereditary skin disorder, hypertensive disorder, hypertrophic cardiomyopathy, immune system disorder, inborn error of immunity, inborn errors of metabolism, inborn metal metabolism disorder, inflammatory bowel disease, inherited neurodegenerative disorder, intellectual disability, intestinal disorder, leukocyte disorder, liver disorder, lung disorder, lymphatic system disorder, lymphoproliferative syndrome, lysosomal lipid storage disorder, lysosomal storage disease, malignant endocrine neoplasm, mastocytosis, megacolon, metabolic disease, movement disorder, multiple endocrine neoplasia, neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neuroendocrine neoplasm, nutritional disorder, obesity disorder, overnutrition, paraganglioma, pheochromocytoma, primitive neuroectodermal tumor, proteostasis deficiencies, pulmonary hypertension, respiratory system disorder, sarcoidosis, skin disorder, skin vascular disease, sphingolipidosis, steroid inherited metabolic disorder, subvalvular aortic stenosis, telangiectasis, urticaria, vascular disorder, vascular hemostatic disease, vascular malformation, von Hippel-Lindau diseaseRecruitingNot stated2026-01-14NCT06573723
A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenogenital syndrome, congenital adrenal hyperplasia, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, metabolic disease, steroid inherited metabolic disorderActive, not recruitingPhase 1 / Phase 22025-12-23NCT04783181
Congenital Adrenal Hyperplasia Once Daily Hydrocortisone TreatmentCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenogenital syndrome, congenital adrenal hyperplasia, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, metabolic disease, steroid inherited metabolic disorderRecruitingPhase 42025-09-16NCT03760835
Fertility And Sexual Function In CAH: CALLIOPECongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Abnormalities, Urogenital Diseases, adrenal gland disorder, adrenogenital syndrome, congenital adrenal hyperplasia, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, metabolic disease, steroid inherited metabolic disorderRecruitingNot stated2025-08-01NCT07099456