Study of SPR001 in Adults With Classic Congenital Adrenal Hyperplasia

A Phase 2, Multiple-Dose, Dose-Escalation Study to Evaluate the Safety and Efficacy of SPR001 in Adults With Classic Congenital Adrenal Hyperplasia (CAH)

This is a multicenter Phase 2, multiple dose, dose escalation study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of SPR001 in adult patients with classic congenital adrenal hyperplasia (CAH).

Study Overview

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

Detailed Description

This is a 6-week, multiple-dose, dose escalation study of SPR001 for the treatment of adults with classic CAH. After screening, eligible patients will be enrolled into a 6-week treatment period followed by a 4-week washout/safety follow-up period.

It is initially planned that up to approximately 18 patients in 2 dose cohorts will be enrolled. Additional patients or dose groups may be considered based upon specific safety, PK/PD, and/or efficacy findings, or if an active dose has not yet been reached.

SPR001 will be administered as an oral daily dose. Patients will undergo titration of SPR001 through three escalating dosage strengths at 2-week intervals. Patients will have overnight PK/PD assessments performed at baseline, which include an pre-dose overnight assessment and a post-dose overnight assessment for PK/PD following administration of the first dose. At the end of each 2-week dosing period, patients will return for single overnight visits for steady-state PK/PD assessments.

A follow-up outpatient visit will occur 30 days after their last dose.

• Study Type: Interventional
• Enrollment (Actual): 26
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • Orange, California, United States, 92123
      • Spruce Biosciences Clinical Site
    • San Diego, California, United States, 92123
      • Spruce Biosciences Clinical Site
  • Florida
    • Melbourne, Florida, United States, 32935
      • 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
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Male and female patients age 18 or older.
• Documented diagnosis of classic CAH due to 21-hydroxylase deficiency
• Elevated 17-OHP at screening
• On a stable glucocorticoid replacement regimen for a minimum of 30 days

Exclusion Criteria:

• Clinically significant unstable medical condition, illness, or chronic disease
• Clinically significant psychiatric disorder.
• Clinically significant abnormal laboratory finding or assessment
• History of bilateral adrenalectomy or hypopituitarism
• Pregnant or nursing females
• Use of any other investigational drug within 30 days
• Unable to understand and comply with the study procedures, understand the risks, and/or unwilling to provide written informed consent.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NON_RANDOMIZED
• Interventional Model: SEQUENTIAL
• Masking: NONE

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: Cohort A; The first cohort of 9 patients will be administered SPR001 at dose strength of Dose A daily for 2 weeks, and escalating through Dose B per day for 2 weeks and Dose C per day for 2 weeks.	Drug: SPR001; SPR001 Capsules
EXPERIMENTAL: Cohort B; Cohort B will begin enrollment after Cohort A has been fully enrolled. Starting dose selection and the stepwise dosing paradigm for Cohort B will be determined by an interim review of safety and PK/PD data from from Cohort A.	Drug: SPR001; SPR001 Capsules
EXPERIMENTAL: Cohort C; Cohort C will begin enrollment after Cohort B has been fully enrolled. Starting dose selection and the stepwise dosing paradigm for Cohort C will be determined by an interim review of safety and PK/PD data from from Cohort A and B.	Drug: SPR001; SPR001 Capsules

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety of SPR001 in patients with CAH	Incidence of treatment-emergent adverse events; changes from Baseline to End-of-study in clinical laboratory parameters, physical examination findings, vital signs, ECG parameters	6 weeks
Change in 17-hydroxyprogesterone	Change in 17-hydroxyprogesterone from Baseline to End-of-study	6 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes in pharmacodynamic (PD) markers	Changes in ACTH and androgens from Baseline to End-of-study	6 weeks
Maximum plasma concentration (Cmax)	To evaluate the pharmacokinetic (PK) parameter of maximum plasma concentration (Cmax) of SPR001 in patients with CAH	6 weeks
Area under the concentration-time curve (AUC)	To evaluate the PK parameter of area under the concentration-time curve (AUC) of SPR001 in patients with CAH	6 weeks
PK/PD relationships	To explore the potential relationships between pharmacokinetics and pharmacodynamics	6 weeks

Collaborators and Investigators

• Sponsor: Spruce Biosciences
• Investigators: Study Director: Michael Huang, MD, Spruce Biosciences; Principal Investigator: Richard Auchus, MD, PhD, University of Michigan

Publications and helpful links

Helpful Links

• On-line clinical study-related information for patients, including pre-screening questionnaire.

Study record dates

Study Major Dates

• Study Start (ACTUAL): July 26, 2017
• Primary Completion (ACTUAL): April 15, 2019
• Study Completion (ACTUAL): May 28, 2019

Study Registration Dates

• First Submitted: August 15, 2017
• First Submitted That Met QC Criteria: August 21, 2017
• First Posted (ACTUAL): August 22, 2017

Study Record Updates

• Last Update Posted (ACTUAL): June 26, 2019
• Last Update Submitted That Met QC Criteria: June 24, 2019
• Last Verified: June 1, 2018

More Information

Terms related to this study

• Keywords: 17-hydroxyprogesterone
• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Endocrine System Diseases; Gonadal Disorders; Disorders of Sex Development; Urogenital Abnormalities; Congenital Abnormalities; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Adrenal Gland Diseases; Steroid Metabolism, Inborn Errors; Hyperplasia; Adrenal Hyperplasia, Congenital; Adrenogenital Syndrome; Adrenocortical Hyperfunction
• Other Study ID Numbers: SPR001-201

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