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SPR001

Phase 2

Congenital Adrenal Hyperplasia | Small molecule | Endocrine |Spruce Biosciences, Inc.|Last Updated: Oct 22, 2025

Success Probability

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Market & Valuation

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Trial Design

CONTROLLED
Total Trials2
Total Enrollment35

FDA Designations

No designations recorded

Clinical trial landscape

SPR001 · 2 trials · 3 indications

Phase 2 2
NCT03687242Study to Evaluate the Safety and Efficacy of SPR001 in Subjects With Classic Congenital Adrenal HyperplasiaCongenital Adrenal Hyperplasia
COMPLETED11 Analytics
NCT03257462Study of SPR001 in Adults With Classic Congenital Adrenal HyperplasiaCongenital Adrenal Hyperplasia
COMPLETED24 Analytics
PHASE2COMPLETED
Study to Evaluate the Safety and Efficacy of SPR001 in Subjects With Classic Congenital Adrenal Hyperplasia
Congenital Adrenal HyperplasiaUnlock trial analytics
PHASE2COMPLETED
Study of SPR001 in Adults With Classic Congenital Adrenal Hyperplasia
Congenital Adrenal HyperplasiaUnlock trial analytics

Study Endpoints

Primary Endpoints

The Incidence of Treatment-emergent Adverse Events (Safety and Tolerability) in Subjects With CAH
Over 12 weeks

Incidence of treatment-emergent adverse events including any serious adverse events, dose-limiting toxicities, and adverse events leading to discontinuation of study drug.

Safety of SPR001 in Patients With CAH
6 weeks

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
Cohort A: Baseline/2a (Day -1-0), First dose/2b (Day 0-1), Visit 3 (Day 13-14), Visit 4 (Day 27-28), Visit 5 (Day 41-42). Cohort B and Cohort C: Visit 2 (Day 0-1), Visit 3 (Day 8), Visit 4 (Day 14-15), Visit 5 (Last dose +30d)

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.

Secondary Endpoints

Change From Baseline in 17-hydroxyprogesterone (17-OHP)
Week 12
Change From Baseline in Androstenedione (A4)
Week 12
Change From Baseline in Adrenocorticotropic Hormone (ACTH)
Week 12
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
SPR001EXPERIMENTALSPR001 at Dose A
Cohort AEXPERIMENTALThe 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 BEXPERIMENTALCohort 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 CEXPERIMENTALCohort 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.

Interventions

NameTypeDescription
SPR001DRUGOpen label SPR001
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites8

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 ...

Countries:United States
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Frequently asked questions about SPR001

What is SPR001 used for in Congenital Adrenal Hyperplasia?

SPR001 is an investigational small molecule being developed for the treatment of Congenital Adrenal Hyperplasia (CAH), a group of inherited endocrine disorders. It is currently in Phase 2 clinical development and has been studied in adult patients with classic CAH, including those with 21-hydroxylase deficiency.

Who makes SPR001?

SPR001 is being developed by Spruce Biosciences, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol SPRB. The company is conducting clinical trials to evaluate the safety and efficacy of SPR001 in patients with Congenital Adrenal Hyperplasia.

What phase is SPR001 in?

SPR001 is in Phase 2 clinical development. Two Phase 2 clinical trials have been completed, with a total of 35 adult patients enrolled. The drug is investigational and has not been approved by regulatory authorities, as it is still undergoing clinical evaluation.

What clinical trials is SPR001 in?

SPR001 has been studied in two completed Phase 2 clinical trials. The first trial, NCT03257462, enrolled 24 adults with classic Congenital Adrenal Hyperplasia in the United States. The second trial, NCT03687242, enrolled 11 adults with classic CAH, including those with 21-hydroxylase deficiency, also in the United States.

Is SPR001 the same as any other drug?

SPR001 is the investigational name used by Spruce Biosciences for its small molecule candidate. No alternative names for SPR001 have been disclosed in the clinical trial records, and it is distinct from other marketed or investigational therapies for Congenital Adrenal Hyperplasia.