10 completed, 2 terminated, 1 active not recruiting, 1 not yet recruiting. 16 Phase 1, 8 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 16 | Completed 10, Terminated 2, Active not recruiting 1, Not yet recruiting 1, Recruiting 1, Withdrawn 1 |
| Phase 2 | 8 | Completed 6, Active not recruiting 1, Recruiting 1 |
Follow Steroid Metabolism, Inborn Errors: new trials by email Literature Review of Steroid Metabolism, Inborn Errors trials Research Report on Steroid Metabolism, Inborn Errors
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 |
|---|---|---|---|---|---|---|
| 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 |
| Neoadjuvant ADI-PEG 20 + Ifosfamide + Radiotherapy in Soft Tissue Sarcoma ADI PEG20, Ifosfamide, Radiotherapy - Soft Tissue Sarcoma, Sts | Phase 1 | Terminated | Washington University School of Medicine | 6 | 2024-03-14 | 2026-09-03 |
| A Trial of Lu AG13909 in Participants With Congenital Adrenal Hyperplasia Lu AG13909 - Congenital Adrenal Hyperplasia | Phase 1 / Phase 2 | Recruiting | H. Lundbeck A/S | 42 | 2022-12-19 | 2026-03-09 |
| A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH) AAV BBP-631 - Congenital Adrenal Hyperplasia | Phase 1 / Phase 2 | Active not recruiting | Adrenas Therapeutics Inc | 8 | 2021-07-01 | 2025-12-23 |
| Smith-Lemli-Opitz Syndrome and Cholic Acid Cholic Acid - Smith-Lemli-Opitz Syndrome | Phase 1 / Phase 2 | Completed | University of Nebraska | 12 | 2021-03-27 | 2024-04-30 |
| Subcutaneous Hydrocortisone Children With Congenital Adrenal Hyperplasia Subcutaneous hydrocortisone - Congenital Adrenal Hyperplasia, Hyperplasia | Phase 1 | Completed | University of Minnesota | 11 | 2019-01-01 | 2025-06-25 |
| Androgen Reduction in Congenital Adrenal Hyperplasia, Phase 1 Abiraterone acetate - Congenital Adrenal Hyperplasia | Phase 1 | Terminated | University of Texas Southwestern Medical Center | 4 | 2017-08-01 | 2025-11-04 |
| Safety, Pharmacokinetics and Pharmacodynamics of NBI-77860 in Adolescent Females With Congenital Adrenal Hyperplasia NBI-77860 - Congenital Adrenal Hyperplasia | Phase 1 | Withdrawn | Neurocrine Biosciences | 2015-02-01 | 2015-06-11 | |
| Ultradian Subcutaneous Hydrocortisone Infusion in Addison Disease and Congenital Adrenal Hyperplasia Solu-Cortef, Cortef - Addison Disease, Adrenal Hyperplasia Congenital | Phase 1 / Phase 2 | Completed | Haukeland University Hospital | 8 | 2014-03-27 | 2024-06-07 |
| A Phase 1 Open-Label Study to Evaluate the Metabolism and Excretion of CC-223 and the Effect of Food on the Pharmacokinetics of CC-223 in Healthy Male Adult Subjects CC-223 - Safety and Pharmacokinetics in Healthy Volunteer Subjects | Phase 1 | Completed | Celgene | 18 | 2012-06-01 | 2019-11-12 |
| A Two-part, Study to Compare the Pharmacokinetics and Dose Proportionality of up to 6 Chronocort Formulations Chronocort - Adrenal Insufficiency, Congenital Adrenal Hyperplasia | Phase 1 | Completed | Neurocrine UK Limited | 28 | 2011-02-01 | 2026-09-14 |
| An Open Label Study in Healthy Volunteers to Compare Chronocort® to Hydrocortisone Hydrocortisone, Chronocort - Congenital Adrenal Hyperplasia, Adrenal Insufficiency | Phase 1 | Completed | Neurocrine UK Limited | 30 | 2010-03-01 | 2026-09-14 |
| Cholesterol in ASD: Characterization and Treatment Cholesterol - Autism, Asperger Disorder | Phase 1 / Phase 2 | Completed | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 44 | 2009-07-28 | 2018-07-05 |
| Comparison of Two Forms of Hydrocortisone in Patients With Congenital Adrenal Hyperplasia Chronocort, Cortef - Congenital Adrenal Hyperplasia, 21-Hydroxylase Deficiency | Phase 1 / Phase 2 | Completed | Neurocrine UK Limited | 20 | 2007-08-01 | 2022-04-26 |
| Treatment of the Cholesterol Defect in Smith-Lemli-Opitz Syndrome crystalline cholesterol oil-based suspension - Smith-Lemli-Opitz Syndrome | Phase 1 / Phase 2 | Completed | Boston Children's Hospital | 23 | 1998-01-01 | 2017-10-20 |
| Congenital Adrenal Hyperplasia: Calcium Channels as Therapeutic Targets Nifedipine - Congenital Adrenal Hyperplasia | Phase 1 / Phase 2 | Completed | National Center for Research Resources (NCRR) | 2005-06-24 |
Adrenal Hyperplasia, Congenital (11) · Smith-Lemli-Opitz Syndrome (3) · Adrenal Gland Diseases (2) · Adrenal Insufficiency (2) · Adrenocortical Hyperfunction (2) · Adrenogenital Syndrome (2) · Congenital Abnormalities (2) · Congenital adrenal hyperplasia due to 21 hydroxylase deficiency (2) · Disorders of Sex Development (2) · Genetic Diseases, Inborn (2) · Hyperplasia (2) · Urogenital Abnormalities (2) · Addison Disease (1) · Asperger Syndrome (1)
Nutritional and Metabolic Diseases (16) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (15) · Metabolic Diseases (15) · Metabolism, Inborn Errors (15) · Genetic Diseases, Inborn (14) · Congenital Abnormalities (13) · Female Urogenital Diseases (11) · Female Urogenital Diseases and Pregnancy Complications (11) · Male Urogenital Diseases (11) · Urogenital Diseases (11) · Endocrine System Diseases (10) · Gonadal Disorders (10) · Adrenal Gland Diseases (9) · Adrenogenital Syndrome (9)
Hydrocortisone (3) · ADI PEG20 (1) · Abiraterone Acetate (1) · CC-223 (1) · Cholesterol (1) · Cholic Acid (1) · Ifosfamide (1) · Mesna (1) · NBI 77860 (1) · Nifedipine (1) · Radiotherapy (1) · hydrocortisone hemisuccinate (1)
Neurocrine UK Limited (3) · Adrenas Therapeutics Inc (1) · Boston Children's Hospital (1) · Celgene (1) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (1) · H. Lundbeck A/S (1) · Haukeland University Hospital (1) · National Center for Research Resources (NCRR) (1) · Neurocrine Biosciences (1) · OMass Therapeutics Australia Proprietary Ltd (1) · University of Minnesota (1) · University of Nebraska (1) · University of Texas Southwestern Medical Center (1) · Washington University School of Medicine (1)
United States (12) · Denmark (1) · France (1) · Georgia (1) · Ireland (1) · Italy (1) · Norway (1) · Poland (1) · Sweden (1) · United Kingdom (1)
Ellen R Elias, MD (1) · Forbes D Porter, M.D. (1) · Girish Sharma (1) · Katerina Simunkova, MD, PhD (1) · Kyriaki Sarafoglou, MD (1) · Maria Palmisano, M.D. (1) · Mia Weiss, M.D. (1) · Mira Irons, M.D. (1) · Perrin C White, MD (1) · Salvatore Febbraro (1) · William B Rizzo, MD (1)
PMID 14605787, PMID 14662594, PMID 33278125, PMID 37172949, PMID 8209913, PMID 9024564, PMID 9024565
M01RR001070 (1), U01HD083493 (1)