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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03687242 | Study to Evaluate the Safety and Efficacy of SPR001 in Subjects With Classic Congenital Adrenal Hyperplasia | PHASE2 | COMPLETED | 11 | — | — | Sep 6, 2018 | Aug 9, 2019 | Apr 1, 2025 | 8 | United States |
| NCT03257462 | Study of SPR001 in Adults With Classic Congenital Adrenal Hyperplasia | PHASE2 | COMPLETED | 24 | — | — | Jul 12, 2017 | Mar 29, 2019 | Oct 22, 2025 | 9 | United States |
Incidence of treatment-emergent adverse events including any serious adverse events, dose-limiting toxicities, and adverse events leading to discontinuation of study drug.
Incidence of treatment-emergent adverse events, changes from Baseline to End-of-study in clinical laboratory parameters, physical examination findings, vital signs, ECG parameters
Change in 17-hydroxyprogesterone from Baseline to End-of-study. Results are expressed as mean percent change from baseline. Reductions in 17-OHP are indicators of better disease control.
| Arm | Type | Description |
|---|---|---|
| SPR001 | EXPERIMENTAL | SPR001 at Dose A |
| Cohort A | EXPERIMENTAL | The first cohort of 9 patients will be administered SPR001 at dose strength of Dose A daily for 2 weeks, and escalating through Dose B per day for 2 weeks and Dose C per day for 2 weeks. |
| Cohort B | EXPERIMENTAL | Cohort B will begin enrollment after Cohort A has been fully enrolled. Starting dose selection and the stepwise dosing paradigm for Cohort B will be determined by an interim review of safety and PK/PD data from from Cohort A. |
| Cohort C | EXPERIMENTAL | Cohort C will begin enrollment after Cohort B has been fully enrolled. Starting dose selection and the stepwise dosing paradigm for Cohort C will be determined by an interim review of safety and PK/PD data from from Cohort A and B. |
| Name | Type | Description |
|---|---|---|
| SPR001 | DRUG | Open label SPR001 |
Inclusion Criteria: * Is approved by the Sponsor's Medical Monitor * Is on a stable regimen of glucocorticoid replacement for ≥30 days before baseline that is expected to remain stable throughout the study * If screening for this study occurs \>3 months after the subject's final follow-up visit in ...