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New hope for kids with rare hormone disorder: drug may cut steroid doses

NCT ID NCT05128942

Disease control Sponsor: Spruce Biosciences Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested an experimental drug called tildacerfont in 67 children aged 2 to 17 with congenital adrenal hyperplasia (CAH), a genetic condition that disrupts hormone production. The goal was to see if adding this once-daily pill to standard steroid treatment could improve disease control and lower steroid doses. The trial was terminated early, so the full results are not available.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Tildacerfont (also called SPR001) – an oral tablet taken once daily alongside standard steroid therapy.
What this could lead to
If it works, this could help children with CAH better control their condition and reduce the amount of steroids they need to take.
What could go wrong
This trial was terminated early, so results are limited. It is a small, phase 2 study, and the drug may not prove safe or effective enough for wider use.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

67 people

The number who actually took part.

Started

Dec 2021

Finished

Jan 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

2 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

adrenal gland disorder classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency congenital adrenal hyperplasia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

21-ohd Congenital adrenal hyperplasia

Contacts and locations

Locations

  • Spruce Study Site

    Sacramento, California, 95821, United States

  • Spruce Study Site

    San Diego, California, 92123, United States

  • Spruce Study Site

    Chicago, Illinois, 60611, United States

  • Spruce Study Site

    Minneapolis, Minnesota, 55454, United States

  • Spruce Study Site

    Buffalo, New York, 14203, United States

  • Spruce Study Site

    Providence, Rhode Island, 02903, United States

  • Spruce Study Site

    Columbia, South Carolina, 29203, United States

  • Spruce Study Site

    Dallas, Texas, 75231, United States

  • Spruce Study Site

    Edinburg, Texas, 78539, United States

  • Spruce Study Site

    Fort Worth, Texas, 76104, United States

  • Spruce Study Site

    Salt Lake City, Utah, 84113, United States

  • Spruce Study Site

    Charlottesville, Virginia, 22903, United States

  • Spruce Study Site

    Richmond, Virginia, 23284, United States

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