Brief 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).

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

participants targeted

Target at below P25 for phase_2

Timeline

Completed

Started Jul 2017

Geographic Reach

1 country

9 active sites

Status

completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

1.7 years study duration

Study Start

First participant enrolled

July 12, 2017

Completed

1 month until next milestone

First Submitted

Initial submission to the registry

August 15, 2017

Completed

7 days until next milestone

First Posted

Study publicly available on registry

August 22, 2017

Completed

1.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 2, 2019

Completed

27 days until next milestone

Study Completion

Last participant's last visit for all outcomes

March 29, 2019

Completed

6.6 years until next milestone

Results Posted

Study results publicly available

October 22, 2025

Completed

Last Updated

October 22, 2025

Status Verified

October 1, 2025

Enrollment Period

1.6 years

First QC Date

August 15, 2017

Results QC Date

February 26, 2025

Last Update Submit

October 7, 2025

Conditions

Congenital Adrenal Hyperplasia CAH - Congenital Adrenal Hyperplasia

Keywords

17-hydroxyprogesterone

Outcome Measures

Primary Outcomes (2)

Safety of SPR001 in Patients With CAH
Incidence of treatment-emergent adverse events, changes from Baseline to End-of-study in clinical laboratory parameters, physical examination findings, vital signs, ECG parameters
6 weeks
Change in 17-hydroxyprogesterone
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.
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)

Secondary Outcomes (3)

Changes in Pharmacodynamic (PD) Markers
Cohort A: Visit 2a (Day -1 to 0), Visit 2b (Days 0-1), Visit 3 (Day 13-14), Visit 4 (Day 27-28), Visit 5 (Day 41 to 42). Cohorts B+C: Visit 2 (Day 0-1), Visit 3 (Day 8), Visit 4 (Day 27-28), Visit 5 (+30 days after last dose)
Pharmacokinetic Parameter - Maximum Plasma Concentration (Cmax)
Serial PK sampling was performed at the end of the 2 weeks for all cohorts and dose levels
Pharmacokinetic Parameter - Area Under the Concentration-time Curve (AUC)
For Cohort A, serial blood collections were made for PK measurements on Day 1 for 200 mg SD and at Week 2 for all QD dose levels. In Cohorts B and C, serial blood samples were drawn for PK measurements at the end of the 2-week treatment period.

Study Arms (3)

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.

Drug: SPR001

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.

Drug: SPR001

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.

Drug: SPR001

Interventions

SPR001DRUG

SPR001 Capsules

Cohort ACohort BCohort C

Eligibility Criteria

Age18 Years+

Sexall

Healthy VolunteersNo

Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

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.

Contact the study team to confirm eligibility.