Phase 2 N=10 Treatment

Study of SPR001 in Adults With Classic Congenital Adrenal Hyperplasia

Congenital Adrenal Hyperplasia · CAH - Congenital Adrenal Hyperplasia

Bottom Line

View on ClinicalTrials.gov: NCT03257462 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

Oct 2025

Primary outcome: Primary: Safety of SPR001 in Patients With CAH — 6; 5; 4 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: SPR001 (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Spruce Biosciences
Primary completion: Mar 2019

Outcome Measures

Outcome	Result	p-value
PRIMARY Safety of SPR001 in Patients With CAH	6; 5; 4	—
PRIMARY Change in 17-hydroxyprogesterone	-47.79; -33.19; 22.16; -23.71; -13.79	—
SECONDARY Changes in Pharmacodynamic (PD) Markers	-26.05; 16.66; -28.53; -67.60; -36.83; -1.58	—
SECONDARY Pharmacokinetic Parameter - Maximum Plasma Concentration (Cmax)	411.25; 200.96; 283.69; 719.30; 892.56; 96.35	—
SECONDARY Pharmacokinetic Parameter - Area Under the Concentration-time Curve (AUC)	2975.968; 1425.582; 1474.162; 4449.263; 6212.983; 421.539	—

Summary

This is a multicenter Phase 2, multiple dose, dose escalation study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of SPR001 in adult patients with classic congenital adrenal hyperplasia (CAH).

Eligibility Criteria

Inclusion Criteria

Male and female patients age 18 or older.
Documented diagnosis of classic CAH due to 21-hydroxylase deficiency
Elevated 17-OHP at screening
On a stable glucocorticoid replacement regimen for a minimum of 30 days

Exclusion Criteria

Clinically significant unstable medical condition, illness, or chronic disease
Clinically significant psychiatric disorder.
Clinically significant abnormal laboratory finding or assessment
History of bilateral adrenalectomy or hypopituitarism
Pregnant or nursing females
Use of any other investigational drug within 30 days
Unable to understand and comply with the study procedures, understand the risks, and/or unwilling to provide written informed consent.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03257462). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.