Study of SPR001 in Adults With Classic Congenital Adrenal Hyperplasia

NCT ID: NCT03257462

Last Updated: 2025-10-22

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

24 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-07-12

Study Completion Date

2019-03-29

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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).

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Congenital Adrenal Hyperplasia CAH - Congenital Adrenal Hyperplasia

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

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.

Group Type EXPERIMENTAL

SPR001

Intervention Type DRUG

SPR001 Capsules

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.

Group Type EXPERIMENTAL

SPR001

Intervention Type DRUG

SPR001 Capsules

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.

Group Type EXPERIMENTAL

SPR001

Intervention Type DRUG

SPR001 Capsules

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

SPR001

SPR001 Capsules

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Spruce Biosciences

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Spruce Chief Medical Officer, MD

Role: STUDY_DIRECTOR

Spruce Biosciences

Richard Auchus, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

University of Michigan

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Spruce Biosciences Clinical Site

Orange, California, United States

Site Status

Spruce Biosciences Clinical Site

San Diego, California, United States

Site Status

Spruce Biosciences Clinical Site

Melbourne, Florida, United States

Site Status

Spruce Biosciences Clinical Site

Atlanta, Georgia, United States

Site Status

Spruce Biosciences Clinical Site

Indianapolis, Indiana, United States

Site Status

Spruce Biosciences Clinical Site

Ann Arbor, Michigan, United States

Site Status

Spruce Biosciences Clinical Site

Minneapolis, Minnesota, United States

Site Status

Spruce Biosciences Clinical Site

Las Vegas, Nevada, United States

Site Status

Spruce Biosciences Clinical Site

Philadelphia, Pennsylvania, United States

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States

References

Explore related publications, articles, or registry entries linked to this study.

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.

Reference Type RESULT
PMID: 34146101 (View on PubMed)

Provided Documents

Download supplemental materials such as informed consent forms, study protocols, or participant manuals.

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

Access external resources that provide additional context or updates about the study.

http://cahclinicaltrial.com/

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

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

SPR001-201

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.