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This is a Phase 1, multicenter, open-label, single-dose study to evaluate the safety and tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NBI-77860 in subjects with congenital adrenal hyperplasia (CAH). The study will be conducted in approximately 15 adolescent females (12-18 years of age) with a documented medical diagnosis of classic 21-hydroxylase deficiency CAH. The study will include three independent dose cohorts of NBI-77860 (approximately 5 subjects per dose cohort). Ascending doses will be evaluated as part of a sequential-cohort design.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| NBI-77860 Dose Group1 | Experimental | NBI-77860 administered orally on Night 1 at bedtime (at approximately 2200 hours). |
|
| NBI-77860 Dose Group 2 | Experimental | NBI-77860 administered orally on Night 1 at bedtime (at approximately 2200 hours). Dosing will not commence until all safety and PK results from the dose group 1 have been reviewed to ensure there are no safety concerns and that maximum tolerated dose (MTD) has not been reached. |
|
| NBI-77860 Dose Group 3 | Experimental | NBI-77860 administered orally on Night 1 at bedtime (at approximately 2200 hours). Dosing will not commence until all safety and PK results from the dose group 2 have been reviewed to ensure there are no safety concerns and that maximum tolerated dose (MTD) has not been reached. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| NBI-77860 | Drug |
| ||
| NBI-77860 |
| Measure | Description | Time Frame |
|---|---|---|
| Number of participants with adverse events following one oral dose of NBI-77860 | Up to 8 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Area Under Concentration Curve (AUC) of NBI-77860 and its metabolites following one oral dose of NBI-77860 | Night 1 and Days 2, 7, 14, 21 and 35 (or early termination) | |
| Concentrations of 17-hydroxyprogesterone (17-OHP) following one oral dose of NBI-77860 |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Ann Arbor | Michigan | 48109 | United States | |||
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| ID | Term |
|---|---|
| D000312 | Adrenal Hyperplasia, Congenital |
| C535979 | Congenital adrenal hyperplasia due to 21 hydroxylase deficiency |
| D047808 | Adrenogenital Syndrome |
| D006965 | Hyperplasia |
| D000307 | Adrenal Gland Diseases |
| D000013 | Congenital Abnormalities |
| D000308 | Adrenocortical Hyperfunction |
| D012734 | Disorders of Sex Development |
| D004700 | Endocrine System Diseases |
| D030342 | Genetic Diseases, Inborn |
| D006058 | Gonadal Disorders |
| D008659 | Metabolic Diseases |
| D008661 | Metabolism, Inborn Errors |
| D010335 | Pathologic Processes |
| D043202 | Steroid Metabolism, Inborn Errors |
| D014564 | Urogenital Abnormalities |
| ID | Term |
|---|---|
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
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| ID | Term |
|---|---|
| C556497 | NBI 77860 |
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| NBI-77860 | Drug |
|
| Screening, Night 1, Day 2 (10, 12 and 24 hours postdose) and 35 (or early termination) |
| Concentrations of adrenocorticotropin hormone (ACTH) following one oral dose of NBI-77860 | Screening, Night 1, Day 2 (10, 12 and 24 hours postdose) and 35 (or early termination) |
| Seattle |
| Washington |
| 98105 |
| United States |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D009750 | Nutritional and Metabolic Diseases |
| D013568 | Pathological Conditions, Signs and Symptoms |