44 completed, 11 recruiting, 10 terminated, 8 active not recruiting. 90 Phase 2, 14 Phase 1, 7 Phase 3.
Follow Urogenital Abnormalities: new trials by email Literature Review of Urogenital Abnormalities trials Research Report on Urogenital Abnormalities
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 |
|---|---|---|---|---|---|---|
| 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 |
| Pre-pubertal Low Dose Transdermal Estradiol in Turner Syndrome Estradiol transdermal patch 0.025mg/24 hours- 1/8th fractionated patch - Turner Syndrome, Primary Ovarian Insufficiency | Phase 2 | Not yet recruiting | Children's National Research Institute | 15 | 2026-09-01 | 2026-08-25 |
| 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 |
| EXACT Study: A Blinded Study in Patients With Alport Syndrome to Evaluate Exaluren Efficacy and Safety Exaluren - Alport Syndrome, X-Linked, Alport Syndrome, Autosomal Recessive | Phase 2 | Recruiting | Eloxx Pharmaceuticals, Inc. | 24 | 2026-06-30 | 2026-09-30 |
| A Study Comparing Different Treatment Approaches for the Initiation of Puberty in Girls With Turner Syndrome Using a TRIFECTA-DARED Approach for Rare Diseases Oral estradiol valerate, Transdermal 17β estradiol - Turner Syndrome | Phase 2 | Not yet recruiting | Universiti Kebangsaan Malaysia Medical Centre | 24 | 2026-02-15 | 2026-02-02 |
| 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 | 2026-11-10 |
| 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 Learn About How Well BAY 3401016 Works in Adults With Alport Syndrome BAY 3401016, Placebo - Alport Syndrome | Phase 2 | Active not recruiting | Bayer | 60 | 2025-11-19 | 2026-10-29 |
| A Long-Term Study of Bivamelagon in Participants With Hypothalamic Obesity (HO) Bivamelagon - Hypothalamic Obesity | Phase 2 | Enrolling by invitation | Rhythm Pharmaceuticals, Inc. | 25 | 2025-11-10 | 2026-02-10 |
| 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 |
| Efficacy and Safety of Phentermine/Topiramate in Youth With Hypothalamic Obesity Phentermine / Topiramate Extended Release Oral Capsule [Qsymia], Placebo - Hypothalamic Obesity, Hypothalamic Tumor | Phase 2 | Active not recruiting | Seattle Children's Hospital | 24 | 2025-03-01 | 2026-10-16 |
| 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 |
| Human Umbilical Cord Mesenchymal Stem Cells for Alport Syndrome hUC-MSC, Placebo control drug - Alport Syndrome | Phase 2 / Phase 3 | Not yet recruiting | Guangzhou Women and Children's Medical Center | 40 | 2025-01-01 | 2024-12-31 |
| Vonafexor ALPort Syndrome Efficacy & Safety TRIAl-1 (ALPESTRIA-1) Vonafexor - Alport Syndrome | Phase 2 | Completed | Enyo Pharma | 26 | 2024-08-01 | 2026-02-09 |
| A Study to Assess Efficacy and Safety of LB54640 in Patients With Hypothalamic Obesity Bivamelagon, Placebo - Hypothalamic Obesity | Phase 2 | Completed | Rhythm Pharmaceuticals, Inc. | 28 | 2024-07-11 | 2026-10-30 |
| Autologous Muscle Stem Cell Therapy for Treatment of Congenital Urinary Incontinence in Epispadias Patients Primary human muscle stem cells (Satori-01), Placebo - Urinary Incontinence, Epispadias, Male | Phase 1 / Phase 2 | Not yet recruiting | Simone Spuler, MD | 21 | 2024-06-01 | 2023-08-03 |
| Vosoritide for Short Stature in Turner Syndrome Vosoritide - Turner Syndrome, Short Stature | Phase 2 | Unknown | Roopa Kanakatti Shankar, MBBS, MS | 20 | 2024-04-12 | 2024-06-24 |
| NRSTS2021, A Risk Adapted Study Evaluating Maintenance Pazopanib, Limited Margin, Dose-Escalated Radiation Therapy and Selinexor in Non-Rhabdomyosarcoma Soft Tissue Sarcoma (NRSTS) Surgical resection, Proton beam radiation therapy, Pazopanib - Adipocytic Neoplasm, Liposarcoma | Phase 1 / Phase 2 | Recruiting | St. Jude Children's Research Hospital | 139 | 2024-03-27 | 2026-08-03 |
Showing 20 of 90. Sign in to see all of them.
Adrenal Hyperplasia, Congenital (30) · Nephritis, Hereditary (13) · Turner Syndrome (11) · Hyperandrogenism (10) · Sexual Infantilism (10) · Polycystic Ovary Syndrome (6) · Craniopharyngioma (4) · Disorders of Sex Development (4) · Hypospadias (4) · Infertility (4) · Insulin Resistance (4) · ACTH Syndrome, Ectopic (3) · Adrenal Gland Diseases (3) · Anovulation (3) · Cushing Syndrome (3)
Male Urogenital Diseases (90) · Urogenital Diseases (90) · Congenital Abnormalities (89) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (89) · Female Urogenital Diseases (89) · Female Urogenital Diseases and Pregnancy Complications (89) · Gonadal Disorders (70) · Endocrine System Diseases (69) · Disorders of Sex Development (65) · Genetic Diseases, Inborn (46) · Adrenogenital Syndrome (38) · Nutritional and Metabolic Diseases (37) · Metabolic Diseases (35) · Metabolism, Inborn Errors (30)
Hydrocortisone (6) · Metformin (4) · crinecerfont (4) · Human Growth Hormone (3) · Exenatide (2) · Gonadotropin-Releasing Hormone (2) · Growth Hormone (2) · Metyrapone (2) · Oxandrolone (2) · benazepril (2) · hydrocortisone hemisuccinate (2) · Abiraterone Acetate (1) · Acupuncture Therapy (1) · Anesthesia, General (1) · Atrasentan (1)
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (5) · Spruce Biosciences (5) · Rhythm Pharmaceuticals, Inc. (4) · Crinetics Pharmaceuticals Inc. (3) · Mayo Clinic (3) · Neurocrine Biosciences (3) · Novartis Pharmaceuticals (3) · Ain Shams University (2) · Eloxx Pharmaceuticals, Inc. (2) · H. Lundbeck A/S (2) · Haukeland University Hospital (2) · National Center for Research Resources (NCRR) (2) · Neurocrine Switzerland GmbH (2) · Neurocrine UK Limited (2) · Adrenas Therapeutics Inc (1)
United States (59) · United Kingdom (17) · Germany (14) · France (12) · Italy (12) · Spain (10) · China (9) · Australia (7) · Poland (7) · Brazil (6) · Sweden (5) · Argentina (4) · Canada (4) · Netherlands (4) · South Korea (4)
David Meeker, MD (3) · Irina Bancos, MD (2) · Shana E McCormack, MD (2) · Abd-Elmoneim Shawky Shams El-deen, MD (1) · Abdel-Wahab El-Okby, MD (1) · Ahmed Said Sayed Bayomy, MSc (1) · Andrea Isidori, MD, PhD (1) · Andrea Lenzi, MD (1) · Anne K Ebert, Prof. Dr. (1) · Ashley Shoemaker, M.D. (1)
PMID 40938675, PMID 33159213, PMID 34146101, PMID 38724717, PMID 19910321, PMID 20871859, PMID 21449786, PMID 23341491, PMID 24037890, PMID 24762109, PMID 25494662, PMID 27133664, PMID 27637014, PMID 27680873, PMID 28285751, PMID 29931143, PMID 30003563, PMID 30302881, PMID 30612112, PMID 31532522, PMID 32034419, PMID 32589738, PMID 33789284, PMID 34433653, PMID 35283460, PMID 36400597, PMID 36411058, PMID 37172949, PMID 37216921, PMID 37254256, PMID 38829703, PMID 39899371, PMID 41442197, PMID 42384439
2015 (2), U54HD028138 (2), 2017 (1), 2019 (1), 2023 (1), M01RR001070 (1), R01AT009484 (1), R01DK107605 (1)