Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03525886 | Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 in Adults With Congenital Adrenal Hyperplasia | PHASE2 | COMPLETED | 18 | — | — | Apr 10, 2018 | Apr 7, 2020 | May 3, 2022 | 6 | United States |
Percent changes in 17-OHP were assessed through the collection of samples from 0600 hours to 1000 hours both prior to study drug administration (i.e., at baseline) and after 14 days of study drug dosing. The 3 samples collected during this morning window at each visit were averaged and used to determine the percent change from baseline.
| Arm | Type | Description |
|---|---|---|
| Cohort 1 (50 mg QHS) | EXPERIMENTAL | NBI-74788 50 mg once daily at bedtime (QHS) administered orally for 14 consecutive days. |
| Cohort 2 (100 mg QHS) | EXPERIMENTAL | NBI-74788 100 mg once daily at bedtime (QHS) administered orally for 14 consecutive days. |
| Cohort 3 (100 mg QPM) | EXPERIMENTAL | NBI-74788 100 mg once daily in the evening (QPM) administered orally for 14 consecutive days. |
| Cohort 4 (100 mg BID) | EXPERIMENTAL | NBI-74788 100 mg twice daily (BID) administered orally for 14 consecutive days. |
| Name | Type | Description |
|---|---|---|
| NBI-74788 | DRUG | Capsule, administered daily. |
Inclusion Criteria: 1. Be in good general health. 2. Have a medically confirmed diagnosis of classic 21-hydroxylase deficiency CAH. 3. Be on a stable regimen of steroidal treatment for CAH that is expected to remain stable throughout the study. 4. Subjects of childbearing potential must be instruct...