A Phase 2 Study to Evaluate the Safety, Efficacy and PK of Tildacerfont in Children Aged 2-17 Years With CAH

December 7, 2025 updated by: Spruce Biosciences

A Phase 2 Study to Evaluate the Safety, Efficacy and Pharmacokinetics of SPR001 (Tildacerfont) in Children Aged 2 to 17 Years With Congenital Adrenal Hyperplasia (CAH)

An investigation of the safety and efficacy of tildacerfont in participants with CAH.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Detailed Description

This is a Phase 2 open-label study with up to 10 cohorts that will evaluate the safety, efficacy, and PK of different tildacerfont dosing regimens potentially up to 200mg QD for 12 weeks in children with classic CAH, and up to 400mg BID for 4 weeks in children and adults with classic CAH.

Study Type

Interventional

Enrollment (Actual)

67

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Sacramento, California, United States, 95821
        • Spruce Study Site
      • San Diego, California, United States, 92123
        • Spruce Study Site
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Spruce Study Site
    • Minnesota
      • Minneapolis, Minnesota, United States, 55454
        • Spruce Study Site
    • New York
      • Buffalo, New York, United States, 14203
        • Spruce Study Site
    • Rhode Island
      • Providence, Rhode Island, United States, 02903
        • Spruce Study Site
    • South Carolina
      • Columbia, South Carolina, United States, 29203
        • Spruce Study Site
    • Texas
      • Dallas, Texas, United States, 75231
        • Spruce Study Site
      • Edinburg, Texas, United States, 78539
        • Spruce Study Site
      • Fort Worth, Texas, United States, 76104
        • Spruce Study Site
    • Utah
      • Salt Lake City, Utah, United States, 84113
        • Spruce Study Site
    • Virginia
      • Charlottesville, Virginia, United States, 22903
        • Spruce Study Site
      • Richmond, Virginia, United States, 23284
        • Spruce Study Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 17 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Male and female subjects aged 2+
  • Diagnosis of CAH due to 21-hydroxylase deficiency (OHD) and/or elevated 17- hydroxyprogesterone (OHP) requiring ongoing GC replacement since diagnosis
  • Stable dose of GC replacement for at least 1 month prior to screening

Exclusion Criteria:

  • History of bilateral adrenalectomy or hypopituitarism
  • Clinically significant unstable medical conditions, illness, or chronic diseases
  • History of active bleeding disorders

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Cohort 1: Age 11-17 Treatment with Tildacerfont
50 mg daily for 12 consecutive weeks.
Drug: Tildacerfont
Oral tablet formulation taken once daily in combination with glucocorticoid therapy.
Other Names:
  • SPR001
Experimental: Cohort 2: Age 11-17 Treatment with Tildacerfont
200 mg daily for 12 consecutive weeks.
Drug: Tildacerfont
Oral tablet formulation taken once daily in combination with glucocorticoid therapy.
Other Names:
  • SPR001
Experimental: Cohort 3: Age 2-10 Treatment with Tildacerfont
50, 100, or 200 mg daily for 12 consecutive weeks.
Drug: Tildacerfont
Oral tablet formulation taken once daily in combination with glucocorticoid therapy.
Other Names:
  • SPR001
Experimental: Cohort 4: Age >/= 18 Treatment with Tildacerfont
200 mg twice daily for 4 consecutive weeks.
Drug: Tildacerfont
Oral tablet formulation taken once daily in combination with glucocorticoid therapy.
Other Names:
  • SPR001
Experimental: Cohort 5: Age >/= 18 Treatment with Tildacerfont
300 or 400 mg twice daily for 4 consecutive weeks.
Drug: Tildacerfont
Oral tablet formulation taken once daily in combination with glucocorticoid therapy.
Other Names:
  • SPR001
Experimental: Cohort 6: Age 11-17 Treatment with Tildacerfont
200 mg twice daily for 4 consecutive weeks.
Drug: Tildacerfont
Oral tablet formulation taken once daily in combination with glucocorticoid therapy.
Other Names:
  • SPR001
Experimental: Cohort 7: Age 2-10 Treatment with Tildacerfont
200 mg twice daily for 4 consecutive weeks.
Drug: Tildacerfont
Oral tablet formulation taken once daily in combination with glucocorticoid therapy.
Other Names:
  • SPR001
Experimental: Cohort 8: Age 11-17 Treatment with Tildacerfont
300 or 400 mg twice daily for 4 consecutive weeks.
Drug: Tildacerfont
Oral tablet formulation taken once daily in combination with glucocorticoid therapy.
Other Names:
  • SPR001
Experimental: Cohort 9: Age 2-10 Treatment with Tildacerfont
300 or 400 mg twice daily for 4 consecutive weeks.
Drug: Tildacerfont
Oral tablet formulation taken once daily in combination with glucocorticoid therapy.
Other Names:
  • SPR001

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Participants With Treatment-emergent Adverse Event (TEAE) as Assessed by Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0
Time Frame: 12 weeks
To evaluate safety of tildacerfont in participants with CAH as measured by number of subjects with adverse events following dosing by CTCAE version 5.0
12 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Proportion of Participants Who Achieve a Reduction in Androstenedione (A4) or Reduction in Glucocorticoid (GC) Dosing
Time Frame: 12 weeks
To determine the efficacy of tildacerfont on disease control or reduction of GC use in participants with classic CAH as measured by number of subjects who achieve a reduction in A4 or reduction in GC dosing during treatment period
12 weeks
Proportion of Participants With Elevated Baseline A4 Who Achieve a Reduction in A4
Time Frame: 4 weeks
To determine the efficacy of tildacerfont on disease control in participants with classic CAH measured by the number of participants with elevated baseline A4 who achieve reduction in A4 at week 4
4 weeks
Proportion of Participants With Elevated Baseline A4 Who Achieve a Reduction in A4 Who Achieve A4 Normalization
Time Frame: 4 weeks or 12 weeks
To determine the efficacy of tildacerfont on disease control in participants with classic CAH measured by the number of participants with elevated baseline A4 who achieve reduction in A4 at week 4 or week 12
4 weeks or 12 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Spruce Biosciences

Investigators

  • Study Director: Will Charlton, MD, Spruce Biosciences

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 10, 2021

Primary Completion (Actual)

January 31, 2025

Study Completion (Actual)

January 31, 2025

Study Registration Dates

First Submitted

October 27, 2021

First Submitted That Met QC Criteria

November 9, 2021

First Posted (Actual)

November 22, 2021

Study Record Updates

Last Update Posted (Actual)

December 24, 2025

Last Update Submitted That Met QC Criteria

December 7, 2025

Last Verified

December 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SPR001-205

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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