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

Study Overview
=================
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.

Official Title
-----------------
A 3-Month Phase 2 Study to Evaluate the Safety and Efficacy of SPR001 in Subjects With Classic Congenital Adrenal Hyperplasia

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

Intervention / Treatment
-----------------
* Drug: SPR001


Participation Criteria
=================
Eligibility Criteria
-----------------
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.

Ages Eligible for Study
-----------------
Minimum Age: 18 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: SPR001<br>SPR001 at Dose A | Drug: SPR001<br>* Open label SPR001<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 the course of 12 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline in 17-hydroxyprogesterone (17-OHP) | Change from Baseline to Week 12 in 17-OHP following dosing of SPR001 in subjects with CAH | Over the course of 12 weeks | 
| Change from baseline in androstenedione | Change from Baseline to Week 12 in androstenedione following dosing of SPR001 in subjects with CAH | Over the course of 12 weeks | 
| Change from baseline in adrenocorticotropic hormone (ACTH) | Change from Baseline to Week 12 in ACTH following dosing of SPR001 in subjects with CAH | Over the course of 12 weeks |