58 completed, 19 unknown, 18 recruiting, 10 terminated. 42 Phase 2, 16 Phase 1, 12 Phase 3, 11 Not applicable, 3 Phase 4.
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Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Development of Healthcare Transition for Patients With Congenital Adrenal Hyperplasia exposure to healthcare transition protocol - Congenital Adrenal Hyperplasia | Not yet recruiting | University of Alabama at Birmingham | 40 | 2027-01-01 | 2026-10-14 | |
| Efficacy, Safety, Tolerability, and Pharmacokinetics of NBIP-01435 in Adult Participants With Classic Congenital Adrenal Hyperplasia (CAH) NBIP-01435 - Congenital Adrenal Hyperplasia | Phase 2 | Not yet recruiting | Neurocrine Biosciences | 36 | 2026-12-01 | 2026-09-30 |
| An Extension Trial of Asedebart in Participants With Congenital Adrenal Hyperplasia Asedebart - Adrenal Hyperplasia, Congenital | Phase 2 | Not yet recruiting | H. Lundbeck A/S | 45 | 2026-10-01 | 2026-10-05 |
| Metyrapone Study for Patients Diagnosed With Mild Autonomous Cortisol Secretion - MACS Metyrapone 250 mg Oral Tablets, Placebo - Mild Autonomous Cortisol Secretion (MACS), Autonomous Cortisol Secretion (ACS) | Phase 2 | Recruiting | Mayo Clinic | 90 | 2026-10-01 | 2026-09-25 |
| Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of OMS1620 OMS1620, Placebo - Congenital Adrenal Hyperplasia (CAH) | Phase 1 | Not yet recruiting | OMass Therapeutics Australia Proprietary Ltd | 84 | 2026-09-01 | 2026-09-09 |
| Evaluation of Circadian Hormonal Balance: LC-MS/MS Measurement of Salivary and Serum Steroids in Patients With Congenital Adrenal Hyperplasia Congenital Adrenal Hyperplasia, Adrenal Insufficiency | Not yet recruiting | Assistance Publique - Hôpitaux de Paris | 50 | 2026-09-01 | 2026-07-21 | |
| Hydrocortisone Modified-release in Adults: Real-world Monitoring of Longitudinal Outcomes in coNgenital Adrenal hYperplasia CAH - Congenital Adrenal Hyperplasia | Not yet recruiting | IRCCS San Raffaele | 100 | 2026-07-31 | 2026-06-08 | |
| Pediatric Congenital Adrenal Hyperplasia Management: Regret and Long-term Outcomes questionnaire - Classic Congenital Adrenal Hyperplasia, 21-Hydroxylase Deficiency | Recruiting | University Hospital, Lille | 40 | 2026-07-08 | 2026-08-24 | |
| Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Crinecerfont in Participants With Classic Congenital Adrenal Hyperplasia (CAH) Who Are Less Than 4 Years Old Crinecerfont - Congenital Adrenal Hyperplasia | Phase 2 | Recruiting | Neurocrine Switzerland GmbH | 20 | 2026-07-01 | 2026-09-16 |
| Parenting and CAH - 21-hydroxylase Deficiency phone questionnaire - CAH - 21-Hydroxylase Deficiency | Recruiting | Assistance Publique - Hôpitaux de Paris | 200 | 2026-03-16 | 2026-08-31 | |
| Isturisa Treatment in Mild Autonomous Cortisol Secretion( MACS) Osilodrostat (Isturisa) - Mild Autonomous Cortisol Secretion (MACS) | Phase 4 | Recruiting | Johns Hopkins University | 10 | 2026-02-02 | 2026-10-23 |
| A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH) Atumelnant, Placebo - Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia | Phase 2 / Phase 3 | Recruiting | Crinetics Pharmaceuticals Inc. | 153 | 2026-01-22 | 2026-10-16 |
| Impact of 1 mg Osilodrostat Therapy on Mild Autonomous Cortisol Secretion (MACS) Osilodrostat 1 MG - Mild Autonomous Cortisol Secretion, Autonomous Cortisol Secretion (ACS) | Phase 2 | Recruiting | Mayo Clinic | 15 | 2026-01-14 | 2026-02-11 |
| A Study to Evaluate Atumelnant in Adults With Congenital Adrenal Hyperplasia Atumelnant, Placebo - Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia | Phase 3 | Recruiting | Crinetics Pharmaceuticals Inc. | 150 | 2025-12-11 | 2026-10-16 |
| A Study of a Virtual Education-Based Transition Intervention to Improve Transition Readiness in Adolescent and Young Adults With Congenital Adrenal Hyperplasia virtual educational based transition intervention self-paced - Congenital Adrenal Hyperplasia | Not applicable | Withdrawn | National Institutes of Health Clinical Center (CC) | 2025-12-09 | 2025-12-10 | |
| Pharmacokinetics, Safety and Tolerability of Crinecerfont in Participants With Congenital Adrenal Hyperplasia Who Are Less Than 2 Years Old Crinecerfont - Congenital Adrenal Hyperplasia | Phase 2 | Active not recruiting | Neurocrine Switzerland GmbH | 7 | 2025-09-30 | 2026-08-28 |
| Syndromes With Neonatal Salt Loss: Not Only Congenital Adrenal Hyperplasia Due to 21-hydroxylase Deficiency (21OH-ISC) Neonatal Salt Loss, Congenital Adrenal Hyperplasia (CAH) | Recruiting | IRCCS Azienda Ospedaliero-Universitaria di Bologna | 25 | 2025-04-14 | 2026-03-16 | |
| An Extension Study to Evaluate Safety and Efficacy of Atumelnant in Participants With Congenital Adrenal Hyperplasia atumelnant (CRN04894) - Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia | Phase 2 | Recruiting | Crinetics Pharmaceuticals Inc. | 200 | 2025-02-25 | 2026-06-29 |
| Investigation of the Etiology of Hypertension and Endothelial Damage in Patients With Cytochrome P450 Oxidoreductase Deficiency Cytochrome P450 Oxidoreductase Deficiency, Hypertension | Completed | Ozge Bayrak Demirel | 51 | 2024-12-24 | 2025-09-08 | |
| Growing up With the Young Endocrine Support System (YESS!) YESS! game, Snake '97, Transition-toolkit - Congenital Adrenal Hyperplasia, Hypogonadotropic Hypogonadism | Not applicable | Not yet recruiting | dr. Laura C. G. de Graaff-Herder | 160 | 2024-12-01 | 2023-09-07 |
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Adrenal Hyperplasia, Congenital (99) · Smith-Lemli-Opitz Syndrome (18) · Congenital adrenal hyperplasia due to 21 hydroxylase deficiency (11) · Adrenal Insufficiency (10) · Cushing Syndrome (7) · Disorders of Sex Development (7) · Adrenal Gland Diseases (6) · ACTH Syndrome, Ectopic (5) · Ichthyosis, X-Linked (5) · Mineralocorticoid Excess Syndrome, Apparent (5) · Hypertension (4) · Pheochromocytoma (4) · Congenital Hypothyroidism (3) · Cystic Fibrosis (3) · Genetic Diseases, Inborn (3)
Nutritional and Metabolic Diseases (125) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (124) · Metabolic Diseases (124) · Metabolism, Inborn Errors (123) · Genetic Diseases, Inborn (122) · Congenital Abnormalities (119) · Female Urogenital Diseases (100) · Female Urogenital Diseases and Pregnancy Complications (100) · Male Urogenital Diseases (100) · Urogenital Diseases (100) · Endocrine System Diseases (98) · Gonadal Disorders (98) · Urogenital Abnormalities (98) · Adrenogenital Syndrome (97)
Hydrocortisone (13) · crinecerfont (6) · Dexamethasone (5) · Cholesterol (4) · Prednisone (4) · Prednisolone (3) · Surveys and Questionnaires (3) · hydrocortisone hemisuccinate (3) · Abiraterone Acetate (2) · Cortisone (2) · Doxorubicin (2) · Fludrocortisone (2) · Interviews as Topic (2) · Metyrapone (2) · Osilodrostat (2)
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (12) · Neurocrine UK Limited (8) · Assistance Publique - Hôpitaux de Paris (6) · Neurocrine Biosciences (6) · Spruce Biosciences (5) · Crinetics Pharmaceuticals Inc. (4) · Ain Shams University (3) · Haukeland University Hospital (3) · Hospices Civils de Lyon (3) · IRCCS Azienda Ospedaliero-Universitaria di Bologna (3) · Mayo Clinic (3) · Adrenas Therapeutics Inc (2) · Assiut University (2) · Boston Children's Hospital (2) · H. Lundbeck A/S (2)
United States (70) · France (21) · Italy (16) · United Kingdom (15) · Brazil (10) · Germany (10) · Netherlands (8) · Sweden (8) · Poland (7) · Spain (7) · Argentina (5) · Belgium (5) · Canada (5) · Australia (4) · Turkey (Türkiye) (4)
Forbes D Porter, M.D. (6) · Deborah P Merke, M.D. (3) · Ellen R Elias, MD (2) · Irina Bancos, MD (2) · Maria I. New, MD (2) · Perrin C White, MD (2) · Adrian Gadano, MD (1) · Alessandra Gambineri, MD (1) · Amir Hamrahian, MD (1) · Amy S Paller, MD (1) · Andrea Giustina, MD (1)
PMID 33527139, PMID 27053961, PMID 34146101, PMID 38828955, PMID 39271956, PMID 11511292, PMID 11739428, PMID 11912510, PMID 12648223, PMID 14605787, PMID 14662594, PMID 14734878, PMID 15238912, PMID 16250204, PMID 16895963, PMID 18459045, PMID 18571597, PMID 18948357, PMID 19241467, PMID 20515440, PMID 20719839, PMID 20871859, PMID 22157069, PMID 2288563, PMID 22904181, PMID 22990093, PMID 23337727, PMID 24878054, PMID 24884782, PMID 25494662, PMID 26014102, PMID 26075496, PMID 26760044, PMID 26811406, PMID 27177728, PMID 27336356, PMID 27454103, PMID 27565451, PMID 27680873, PMID 27801983, PMID 28472487, PMID 28576284, PMID 28887710, PMID 29229498, PMID 29510982, PMID 29682773, PMID 30003563, PMID 30465166, PMID 30817193, PMID 31206616
2023 (3), 2008 (1), 2019 (1), M01RR001070 (1), R01HD057930 (1), R01HL073980 (1), U01HD083493 (1), U54HD064382 (1), U54RR019484 (1)