Brief Summary

This is a Phase 2 study of SPR001 for the treatment of classic CAH that will provide 12 weeks of open-label treatment to eligible subjects.

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 Sep 2018

Shorter than P25 for phase_2

Geographic Reach

1 country

8 active sites

Status

completed

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

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

11 months study duration

Study Start

First participant enrolled

September 6, 2018

Completed

5 days until next milestone

First Submitted

Initial submission to the registry

September 11, 2018

Completed

16 days until next milestone

First Posted

Study publicly available on registry

September 27, 2018

Completed

9 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 8, 2019

Completed

1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

August 9, 2019

Completed

5.6 years until next milestone

Results Posted

Study results publicly available

April 1, 2025

Completed

Last Updated

April 1, 2025

Status Verified

March 1, 2025

Enrollment Period

10 months

First QC Date

September 11, 2018

Results QC Date

February 26, 2025

Last Update Submit

March 28, 2025

Conditions

Congenital Adrenal Hyperplasia CAH - Congenital Adrenal Hyperplasia CAH - 21-Hydroxylase Deficiency

Outcome Measures

Primary Outcomes (1)

The Incidence of Treatment-emergent Adverse Events (Safety and Tolerability) in Subjects With CAH
Incidence of treatment-emergent adverse events including any serious adverse events, dose-limiting toxicities, and adverse events leading to discontinuation of study drug.
Over 12 weeks

Secondary Outcomes (3)

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

Study Arms (1)

SPR001

EXPERIMENTAL

SPR001 at Dose A

Drug: SPR001

Interventions

SPR001DRUG

Open label SPR001

SPR001

Eligibility Criteria

Age18 Years+

Sexall

Healthy VolunteersNo

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

You may qualify if:

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 Study SPR001-201, the subject will have serum 17-OHP measured at screening.
Agrees to follow contraception guidelines
Is able to understand all study procedures and risks involved and provides written informed consent indicating willingness to comply with all aspects of the protocol

You may not qualify if:

Experienced a clinically significant AE considered at least possibly related to SPR001 in Study SPR001-201
If screening for this study occurs \>3 months after the subject's final follow-up visit in Study SPR001-201, the subject will be screened for any clinically significant unstable medical condition, medically significant illness, or chronic disease occurring within 30 days of screening
Is at increased risk of suicide
Clinically significant depression or anxiety at screening or baseline
Clinically significant abnormal clinical or laboratory assessments must be discussed with the Medical Monitor to determine eligibility for this study.
Subjects who routinely work overnight shifts require Medical Monitor approval for enrollment
Females who are pregnant or lactating
Use of any other investigational drug within 30 days or 5 half-lives before screening
Use of prohibited concomitant medications (including rosiglitazone, testosterone, and strong inhibitors and/or inducers of CYP3A4) within 30 days or 5 half-lives of baseline. Medications metabolized by CYP3A4, 2C8, 2C9, or 2C19, especially those that are sensitive substrates or substrates with narrow therapeutic ranges should be discussed on a case-by-case basis with the Medical Monitor.

Contact the study team to confirm eligibility.