Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Tildacerfont
SPR001 · 2 trials · 3 indications
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 ...
SPR001 is an investigational small molecule being developed for classic congenital adrenal hyperplasia, an endocrine disorder. It has been studied in adults 18 years and older with classic CAH, including cases associated with 21-hydroxylase deficiency. Both of its Phase 2 trials enrolled adults in the United States.
SPR001 targets the hCRF1 receptor, a receptor in the corticotropin-releasing factor receptor family. By acting on this receptor, the small molecule is designed to modulate the hormonal signaling pathway relevant to congenital adrenal hyperplasia. The hCRF1 receptor is the stated molecular target for SPR001.
SPR001 is being developed by Spruce Biosciences, Inc., which trades under the ticker SPRB. The company is the sponsor behind the clinical program evaluating SPR001 in classic congenital adrenal hyperplasia, an endocrine condition affecting adults.
SPR001 is in Phase 2 development. Two Phase 2 trials have been completed, and no trials are currently active. SPR001 is investigational and has not been approved by the FDA for congenital adrenal hyperplasia or any other indication.
SPR001 has been evaluated in two completed Phase 2 trials: NCT03687242, a study of safety and efficacy in subjects with classic congenital adrenal hyperplasia, which enrolled 11 participants, and NCT03257462, a study in adults with classic congenital adrenal hyperplasia, which enrolled 24 participants. Both were conducted in the United States.
The two completed Phase 2 trials of SPR001 enrolled a combined total of 35 participants. NCT03687242 enrolled 11 subjects and NCT03257462 enrolled 24 subjects. Both studies were controlled, enrolled adults aged 18 years and older, and included all sexes.