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Study on the Effects of Tildacerfont and Hydrocortisone in Adults with Classic Congenital Adrenal Hyperplasia

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition called (CAH), which is a genetic disorder affecting the adrenal glands. The trial will evaluate a new treatment using a medication called , also known by its code name . The purpose of the study is to assess how effective and safe Tildacerfont is for adults with CAH who have high levels of certain hormones in their blood.

Participants in the study will receive treatment over a period of up to 70 weeks. The study is designed to be "double-blind," meaning neither the participants nor the researchers will know who is receiving Tildacerfont and who is receiving a placebo. This helps ensure the results are unbiased. The study will be conducted in three parts, with the main goal being to see if Tildacerfont can reduce levels of a hormone called androstenedione over 12 weeks.

In addition to Tildacerfont, some participants may continue their current treatment with , a medication commonly used to manage CAH. The trial will monitor changes in hormone levels and any side effects experienced by participants. The results will help determine if Tildacerfont is a beneficial treatment option for people with CAH.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    Upon joining the study, an initial assessment will be conducted to confirm eligibility. This includes reviewing your medical history and current treatment for classic congenital adrenal hyperplasia.

    You will need to provide information about your current medications, including hydrocortisone or other glucocorticoids, and ensure that your dosage has been stable for at least one month.

  2. Step 2

    Baseline evaluation

    A baseline evaluation will be performed to measure your hormone levels, specifically androstenedione and 17-hydroxyprogesterone. These levels will be used to assess changes during the trial.

    If you have the salt-wasting form of the condition, your mineralocorticoid replacement dose must also be stable for at least one month before this evaluation.

  3. Step 3

    Treatment period part 1

    The first part of the treatment period involves taking the study medication, tildacerfont, in tablet form. This medication is taken orally.

    The dosage and frequency will be determined by the study protocol, and you will be required to follow the instructions carefully for the duration of this part.

  4. Step 4

    Treatment period part 2

    The second part of the treatment period continues with the administration of tildacerfont. Your response to the medication will be monitored through regular check-ups and hormone level assessments.

    This part of the treatment aims to evaluate the effect of the medication on reducing androstenedione levels over a 12-week period.

  5. Step 5

    Treatment period part 3

    The final part of the treatment period involves continued monitoring and assessment of your hormone levels and overall health.

    The study will evaluate the proportion of participants who achieve normal hormone levels and any changes in testicular adrenal rest tumors, if applicable.

  6. Step 6

    End of treatment evaluation

    At the end of the treatment period, a comprehensive evaluation will be conducted to assess the overall impact of the study medication on your condition.

    This includes measuring the percent change in androstenedione levels from the baseline and other secondary outcomes.

Who can join the trial?

7 criteria

  • Participants must be 18 years or older.
  • Participants must have a known childhood diagnosis of classic congenital adrenal hyperplasia due to a specific enzyme deficiency. This should be confirmed by a genetic test or past medical records showing high levels of a hormone called 17-hydroxyprogesterone. They should currently be treated with certain medications like hydrocortisone or similar drugs.
  • Participants must have been on a stable dose of their medication for at least 1 month before joining the study.
  • Participants must have levels of a hormone called androstenedione that are higher than normal at both the start of the study and at Week 4, depending on their medication dose.
  • If participants have a form of the condition that affects salt balance, they must be on a stable dose of a specific medication for at least 1 month before joining the study.
  • Participants must agree to follow specific guidelines for contraception during the study. Male participants must also agree not to donate sperm during the study and for 90 days after the last dose of the study drug.
  • Participants must be able to understand all study procedures and risks and provide written consent to show they agree to follow all study requirements.

Who cannot join the trial?

5 criteria

  • Patients who do not have **Classic Congenital Adrenal Hyperplasia** cannot participate.
  • Patients who are not within the specified age range cannot participate.
  • Patients who are not part of the specified clinical trial groups cannot participate.
  • Patients who are not female or male cannot participate.
  • Patients who are not considered part of a vulnerable population cannot participate.
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Investigated drugs

Tildacerfont is a medication being studied for its potential to help people with a condition called congenital adrenal hyperplasia. This condition affects the adrenal glands, which are small organs on top of the kidneys that produce important hormones. In people with this condition, the adrenal glands produce too much of a hormone called androstenedione. Tildacerfont is being tested to see if it can help reduce the levels of this hormone in the body, which might help manage the symptoms of the condition. The study aims to see how well this medication works and if it is safe for people to use over a period of 12 weeks.

What is already known about the treatment

Tildacerfont – Tildacerfont is administered orally in the form of a tablet. It is currently being studied in clinical trials for its potential use in treating Classic Congenital Adrenal Hyperplasia, a condition affecting hormone production in the adrenal glands. The medication works by reducing levels of androstenedione, a hormone that can be elevated in this condition, over a period of 12 weeks. Tildacerfont belongs to a class of drugs known as steroidogenesis inhibitors, which help manage hormone levels in the body.

Investigated diseases

Classic Congenital Adrenal Hyperplasia – Classic Congenital Adrenal Hyperplasia is a genetic disorder affecting the adrenal glands, which are responsible for producing certain hormones. This condition leads to a deficiency in enzymes needed for hormone production, resulting in an imbalance of hormones such as cortisol and aldosterone. As a result, the body produces excess androgens, which are male sex hormones. The disease often manifests in early childhood and can cause symptoms such as early puberty, rapid growth during childhood but shorter than average final height, and ambiguous genitalia in females. Over time, individuals may experience issues with salt balance, blood pressure, and energy levels. The progression of the disease can vary, with some individuals experiencing more severe symptoms than others.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2023-503770-21-00Protocol codeSPR001-203Estimated enrolment75 patientsSponsorSpruce Biosciences Inc.

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