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

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

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.

Study Overview

• Status: Completed
• Conditions: Congenital Adrenal Hyperplasia; CAH - Congenital Adrenal Hyperplasia; CAH - 21-Hydroxylase Deficiency
• Intervention / Treatment: Drug: SPR001

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.

• Study Type: Interventional
• Enrollment (Actual): 11
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • Orange, California, United States, 92868
      • Spruce Biosciences Clinical Site
    • San Diego, California, United States, 92123
      • Spruce Biosciences Clinical Site
  • Georgia
    • Atlanta, Georgia, United States, 30046
      • Spruce Biosciences Clinical Site
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Spruce Biosciences Clinical Site
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • Spruce Biosciences Clinical Site
  • Minnesota
    • Minneapolis, Minnesota, United States, 55414
      • Spruce Biosciences Clinical Site
  • Nevada
    • Las Vegas, Nevada, United States, 89148
      • Spruce Biosciences Clinical Site
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Spruce Biosciences Clinical Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: SPR001; SPR001 at Dose A	Drug: SPR001; Open label SPR001

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 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. 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.	Week 12
Change From Baseline in Androstenedione (A4)	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.	Week 12
Change From Baseline in Adrenocorticotropic Hormone (ACTH)	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.	Week 12

Collaborators and Investigators

• Sponsor: Spruce Biosciences
• Investigators: Study Director: Chief Medical Officer, Spruce Biosciences

Publications and helpful links

General Publications

• 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. Erratum In: J Clin Endocrinol Metab. 2022 Jul 14;107(8):e3546. doi: 10.1210/clinem/dgac224.

Study record dates

Study Major Dates

• Study Start (Actual): September 6, 2018
• Primary Completion (Actual): July 8, 2019
• Study Completion (Actual): August 9, 2019

Study Registration Dates

• First Submitted: September 11, 2018
• First Submitted That Met QC Criteria: September 25, 2018
• First Posted (Actual): September 27, 2018

Study Record Updates

• Last Update Posted (Actual): April 1, 2025
• Last Update Submitted That Met QC Criteria: March 28, 2025
• Last Verified: March 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Urogenital Diseases; Endocrine System Diseases; Pathologic Processes; Male Urogenital Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases; Gonadal Disorders; Congenital Abnormalities; Adrenal Gland Diseases; Disorders of Sex Development; Urogenital Abnormalities; Steroid Metabolism, Inborn Errors; Hyperplasia; Adrenal Hyperplasia, Congenital; Adrenogenital Syndrome; Adrenocortical Hyperfunction
• Other Study ID Numbers: SPR001-202

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No