Study to Evaluate the Safety and Efficacy of SPR001 in Subjects With Classic Congenital Adrenal Hyperplasia

Phase 2

Completed

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

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.

Detailed Description: 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. To be eligible for this study, an individual must either have completed Study SPR001-201 or meet eligibility criteria for SPR001-naïve subjects. The expected duration of study participation for each subject is up to approximately 5 months. This includes a screening period of ≤30 days, a treatment period of 12 weeks, and a safety follow-up period of 30 days.

Recruitment & Eligibility

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 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

Exclusion Criteria

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.

Arm && Interventions

Group	Intervention	Description
SPR001	SPR001	SPR001 at Dose A

Primary Outcome Measures

Name	Time	Method
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.

Secondary Outcome Measures

Name	Time	Method
Change From Baseline in 17-hydroxyprogesterone (17-OHP)	Week 12	Change from Baseline to Week 12 in 17-OHP following dosing of SPR001 in subjects with CAH. 17-OHP is measured in patient serum sample. Results are expressed as mean percent change from baseline. Reductions in 17-OHP are indicators of better disease control.
Change From Baseline in Androstenedione (A4)	Week 12	Change from Baseline to Week 12 in androstenedione (A4) following dosing of SPR001 in subjects with CAH. A4 is measured in patient serum. Results are expressed as mean percent change from baseline. Reductions in A4 are indicators of better disease control.
Change From Baseline in Adrenocorticotropic Hormone (ACTH)	Week 12	Change from Baseline to Week 12 in adrenocorticotropic hormone (ACTH) following dosing of SPR001 in subjects with CAH. ACTH is measured in patient serum. Results are expressed as mean percent change from baseline. Reductions in ACTH are indicators of better disease control.