Clinical trial NCT03687242: Study to Evaluate the Safety and Efficacy of SPR001 in Subjects With Classic Congenital Adrenal Hyperplasia
Official title: A 3-Month Phase 2 Study to Evaluate the Safety and Efficacy of SPR001 in Subjects With Classic Congenital Adrenal Hyperplasia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Congenital Adrenal Hyperplasia
CAH - Congenital Adrenal Hyperplasia
CAH - 21-Hydroxylase Deficiency
Lead sponsor: Spruce Biosciences
Sponsor class: INDUSTRY
First posted: 2018-09-27
Enrollment: 11
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: The Incidence of Treatment-emergent Adverse Events (Safety and Tolerability) in Subjects With CAH
timeFrame: Over 12 weeks
Secondary outcomes:
measure: Change From Baseline in 17-hydroxyprogesterone (17-OHP)
timeFrame: Week 12
measure: Change From Baseline in Androstenedione (A4)
timeFrame: Week 12
measure: Change From Baseline in Adrenocorticotropic Hormone (ACTH)
timeFrame: Week 12
Other outcomes:
Locations:
city: Orange
country: United States
facility: Spruce Biosciences Clinical Site
state: California
city: San Diego
country: United States
facility: Spruce Biosciences Clinical Site
state: California
city: Atlanta
country: United States
facility: Spruce Biosciences Clinical Site
state: Georgia
city: Indianapolis
country: United States
facility: Spruce Biosciences Clinical Site
state: Indiana
city: Ann Arbor
country: United States
facility: Spruce Biosciences Clinical Site
state: Michigan
city: Minneapolis
country: United States
facility: Spruce Biosciences Clinical Site
state: Minnesota
city: Las Vegas
country: United States
facility: Spruce Biosciences Clinical Site
state: Nevada
city: Philadelphia
country: United States
facility: Spruce Biosciences Clinical Site
state: Pennsylvania
Study officials:
affiliation: Spruce Biosciences
name: Chief Medical Officer
role: STUDY_DIRECTOR
References:
citation: Sarafoglou K, Barnes CN, Huang M, Imel EA, Madu IJ, Merke DP, Moriarty D, Nakhle S, Newfield RS, Vogiatzi MG, Auchus RJ. Tildacerfont in Adults With Classic Congenital Adrenal Hyperplasia: Results from Two Phase 2 Studies. J Clin Endocrinol Metab. 2021 Oct 21;106(11):e4666-e4679. doi: 10.1210/clinem/dgab438.