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Study on Tildacerfont to Reduce Glucocorticoid Use 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 known as Classic Congenital Adrenal Hyperplasia, a genetic disorder affecting the adrenal glands. The study aims to evaluate the effectiveness and safety of a medication called Tildacerfont (also known by its code name SPR001) in reducing the use of glucocorticoids, which are a type of steroid hormone, in adults with this condition. Participants in the study are currently taking higher-than-normal doses of glucocorticoids to manage their symptoms.

The trial involves taking Tildacerfont in tablet form, and it will be compared to a placebo to assess its potential benefits. Other medications involved in the study include Prednisolone and Hydrocortisone, which are also types of glucocorticoids. The study is designed to be double-blind, meaning neither the participants nor the researchers know who is receiving the actual medication or the placebo, to ensure unbiased results.

Over a period of 24 weeks, the study will monitor changes in the amount of glucocorticoids needed by participants. The goal is to see if Tildacerfont can help reduce the need for these medications, potentially leading to better management of Classic Congenital Adrenal Hyperplasia. Participants will take the medication orally, and their progress will be closely observed throughout the study period.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, you will be asked to provide written consent, confirming your understanding of the study procedures and your willingness to participate.

    You must meet specific criteria, such as being 18 years or older and having a documented diagnosis of classic congenital adrenal hyperplasia.

  2. Step 2

    Initial assessment

    An initial assessment will be conducted to confirm your eligibility. This includes verifying your medical history and current treatment with glucocorticoids like hydrocortisone or prednisolone.

  3. Step 3

    Treatment period

    The treatment period lasts for 24 weeks. During this time, you will receive either the study medication tildacerfont or a placebo. The medication is taken orally in tablet form.

    You will continue your current glucocorticoid therapy, and the study aims to evaluate changes in your glucocorticoid dosage.

  4. Step 4

    Regular check-ups

    Throughout the study, you will have regular check-ups to monitor your health and the effects of the treatment. These check-ups will assess your glucocorticoid levels and any changes in your condition.

  5. Step 5

    End of treatment evaluation

    At the end of the 24-week treatment period, a final evaluation will be conducted to measure the absolute change in your glucocorticoid dose.

    The study will also assess any improvements in cardiovascular risk factors and other health indicators.

Who can join the trial?

7 criteria

  • Participants must be 18 years or older.
  • Must have a documented diagnosis of classic congenital adrenal hyperplasia due to a specific enzyme deficiency. This should be confirmed by genetic testing or specific hormone levels and currently treated with certain medications like hydrocortisone or prednisone.
  • Hormone levels, specifically androstenedione, must be within a certain range at the start of the study, measured before taking morning medication.
  • Must have been on a stable dose of glucocorticoid (a type of steroid hormone) replacement for at least one month before the study begins.
  • If the participant has the salt-wasting form of the condition, they must have been on a stable dose of mineralocorticoid (another type of hormone) replacement for at least one month before the study.
  • Must agree to follow specific contraception guidelines 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.
  • Must be able to understand all study procedures and risks and provide written consent to participate, showing willingness to follow all study requirements.

Who cannot join the trial?

9 criteria

  • Patients who do not have Classic Congenital Adrenal Hyperplasia cannot participate. This is a condition where the body has trouble making certain hormones.
  • Patients who are not within the specified age range cannot participate. The study is for certain age groups only.
  • Patients who are not willing to follow the study procedures cannot participate.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients with other serious health conditions that might interfere with the study cannot participate.
  • Patients who are taking medications that might interfere with the study cannot participate.
  • Patients who have participated in another clinical trial recently cannot participate.
  • Patients who have a history of drug or alcohol abuse cannot participate.
  • Patients who have allergies to the study medication 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. People with this condition often need to take high doses of medications called glucocorticoids to manage their symptoms. Tildacerfont is being tested to see if it can reduce the need for these high doses, which can have side effects. The goal is to find out if Tildacerfont can help patients use lower doses of glucocorticoids while still keeping their condition under control.

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 to help people with Classic Congenital Adrenal Hyperplasia, a condition that affects hormone production. The medication works by targeting specific receptors in the body to reduce the need for high doses of glucocorticoids, which are often used to manage this condition. Tildacerfont is classified as a non-steroidal medication, meaning it does not contain steroids, and it aims to help balance hormone levels more effectively.

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 the disease progresses, individuals may experience symptoms like early puberty, rapid growth during childhood but shorter than average height in adulthood, and ambiguous genitalia in females. The lack of cortisol can also lead to an overproduction of androgens, causing further hormonal imbalances. Over time, the body may struggle to maintain normal blood pressure, blood sugar levels, and respond to stress effectively. The condition is present from birth and requires ongoing management to address hormone imbalances.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2023-503771-13-00Protocol codeSPR001-204Estimated enrolment90 patientsSponsorSpruce Biosciences Inc.

sourced from the EU Clinical Trials Register and site verification

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