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

NCT ID NCT05128942

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

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.

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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

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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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