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New drug aims to cut steroid use in rare hormone disorder

NCT ID NCT04544410

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 a daily tablet called Tildacerfont in 100 adults with classic congenital adrenal hyperplasia, a condition where the body can't make certain hormones properly. The goal was to see if the drug could safely reduce the high doses of steroids patients need to take. 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), a daily tablet
What this could lead to
If it works, this could help people with congenital adrenal hyperplasia take lower doses of steroids while still controlling their condition.
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 effective or safe in larger groups.

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

100 people

The number who actually took part.

Started

Feb 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

18 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: 1. Male and female subjects ≥18 years old at screening 2. Has a known childhood diagnosis of classic CAH due to 21-hydroxylase deficiency based on genetic mutation in CYP21A2 and/or documented (at any time) elevated 17-hydroxyprogesterone (17-OHP) and currently treated with hydrocortisone (HC), HC acetate, prednisone, prednisolone, methylprednisolone, dexamethasone (or a combination of the aforementioned glucocorticoid \[GCs\]) 3. Has lower limit of detection ≤ androstenedione (A4) ≤ 2.5x upper limit of normal (ULN) at screening measured before a morning GC dose 4. Has been on a stable, supraphysiologic dose of GC replacement (defined as ≥30 mg/day and ≤60 mg/day in HCe) for ≥1 month before screening 5. For subjects with the salt-wasting form of CAH, subject has been on a stable dose of mineralocorticoid replacement for ≥1 month before screening 6. Agrees to follow contraception guidelines. Male subjects must also agree to refrain from donating sperm throughout the Treatment Period and for 90 days after the last dose of study drug 7. Is able to understand all study procedures and risks involved and provides written informed consent indicating willingness to comply with all aspects of the protocol Exclusion Criteria: 1. Has a known or suspected diagnosis of any other known form of classic CAH (not due to 21-hydroxylase deficiency) 2. Has a history that includes bilateral adrenalectomy or hypopituitarism 3. Has a history of allergy or hypersensitivity to tildacerfont, any of its excipients, or any other CRF1 receptor antagonist 4. Shows clinical signs or symptoms of adrenal insufficiency 5. Has had a clinically significant unstable medical condition, medically significant illness, or chronic disease occurring within 30 days of screening, including but not limited to: 1. An ongoing malignancy or \<3 years of remission history from any malignancy, other than successfully treated localized skin cancer 2. eGFR of \<45 mL/min/1.73 m2 3. Current or history of liver disease (with the exception of Gilbert's syndrome) 4. History of alcohol or substance abuse within the last year, or any significant history of alcohol or substance abuse that would likely prevent the subject from reliably participating in the study, based on the opinion of the Investigator 5. Active hepatitis B, hepatitis C, or HIV at screening 6. Subjects who plan to undergo bariatric surgery during the study are excluded 7. Any other condition that would impact subject safety or confound interpretation of study results 6. Psychiatric conditions, including but not limited to bipolar disorder, schizophrenia, or schizoaffective disorders that are not effectively controlled on medication and may have an adverse impact on study compliance. Symptoms including hallucinations, delusions, and psychosis are exclusionary. Additionally: Increased risk of suicide based on the Investigator's judgment or the results of the C-SSRS conducted at screening and baseline (eg, C-SSRS Type 3, 4, or 5 ideation within the past 6 months or any suicidal behavior within the past 12 months) b. Hospital Anxiety and Depression Scale (HADS) score \>12 for either depression or anxiety at screening or baseline 7. Has clinically significant abnormal ECG or clinical laboratory results. Abnormal results that must be reviewed and discussed with the Medical Monitor to determine eligibility for this study include but are not limited to: 1. Any clinically meaningful abnormal ECG results, including QTcF \>450 ms for male participants or \>470 ms for female participants 2. ALT \>2x ULN 3. Total bilirubin \>1.5x ULN 4. Total bile acids \>5x ULN 8. Routinely works overnight shifts 9. Subjects with travel plans/work schedules that result in significant and frequent changes in time zones (\>2 hours) will require Medical Monitor approval for enrollment 10. Females who are pregnant or nursing 11. Use of any other investigational drug from 30 days or 5 half-lives (whichever is longer) before screening to the end of the study 12. Use of the following drugs from 30 days or 5 half-lives (whichever is longer) before the start of the Treatment Period to the end of the study: 1. Rosiglitazone, aromatase inhibitors, testosterone, growth hormones, or any other medication or supplement that could impact subject safety or confound interpretation of study results 2. The drugs which are: i. Moderate to strong inhibitors and/or inducers of CYP3A4 ii. Sensitive substrates or narrow-therapeutic-range substrates of CYP3A4 (except hormonal contraception containing ≤35 μg ethinyl estradiol) iii. Sensitive substrates or narrow-therapeutic-range substrates of BCRP (except those that can be administered QD in the morning, separated by approximately 10 hours from evening administration of study drug) 13. Donation or receipt of blood from 90 days before Screening to the end of the study; donation or receipt of platelets, white blood cells, or plasma from 30 days before Screening to the end of the study

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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.

Congenital adrenal hyperplasia

Contacts and locations

Locations

  • Spruce Biosciences Clinical Site

    Ann Arbor, Michigan, 48109, United States

  • Spruce Study Site

    Birmingham, Alabama, 35294, United States

  • Spruce Study Site

    Los Angeles, California, 90027, United States

  • Spruce Study Site

    San Diego, California, 92123, United States

  • Spruce Study Site

    Indianapolis, Indiana, 46202, United States

  • Spruce Study Site

    Baltimore, Maryland, 21287, United States

  • Spruce Study Site

    Minneapolis, Minnesota, 55454, United States

  • Spruce Study Site

    New Brunswick, New Jersey, 08901, United States

  • Spruce Study Site

    Canton, Ohio, 44718, United States

  • Spruce Study Site

    Cincinnati, Ohio, 45219, United States

  • Spruce Study Site

    Cleveland, Ohio, 44195, United States

  • Spruce Study Site

    Columbus, Ohio, 43210, United States

  • Spruce Study Site

    Philadelphia, Pennsylvania, 19104, United States

  • Spruce Study Site

    Philadelphia, Pennsylvania, 19107, United States

  • Spruce Study Site

    Philadelphia, Pennsylvania, 19140, United States

  • Spruce Study Site

    Providence, Rhode Island, 02903, United States

  • Spruce Study Site

    Columbia, South Carolina, 29203, United States

  • Spruce Study Site

    Fort Worth, Texas, 76104, United States

  • Spruce Study Site

    Blacktown, Australia

  • Spruce Study Site

    Brisbane, 4029, Australia

  • Spruce Study Site

    Elizabeth Vale, Australia

  • Spruce Study Site

    Parkville, Australia

  • Spruce Study Site

    Curitiba, Brazil

  • Spruce Study Site

    São Paulo, Brazil

  • Spruce Study Site

    Ottawa, Ontario, Canada

  • Spruce Study Site

    Sherbrooke, Quebec, J1H 5N4, Canada

  • Spruce Study Site

    Tallinn, Estonia

  • Spruce Study Site

    Tartu, Estonia

  • Spruce Study Site

    Munich, Germany

  • Spruce Study Site

    Roma, Italy

  • Spruce Study Site

    Riga, Latvia

  • Spruce Study Site

    Kaunas, Lithuania

  • Spruce Study Site

    Krakow, Poland

  • Spruce Study Site

    Warsaw, Poland

  • Spruce Study Site

    Bucharest, Romania

  • Spruce Study Site

    Seoul, South Korea

  • Spruce Study Site

    Barcelona, Spain

  • Spruce Study Site

    Madrid, Spain

  • Spruce Study Site

    Seville, Spain

  • Spruce Study Site

    Tarragona, Spain

  • Spruce Study Site

    Stockholm, Sweden

  • Spruce Study Site

    Istanbul, Turkey (Türkiye)

  • Spruce Study Site

    Birmingham, B15 2GW, United Kingdom

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