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Study of the effect of metyrapone in patients with mild Cushing syndrome caused by primary bilateral macronodular adrenal hyperplasia

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What is this trial about?

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

This study examines the effects of metyrapone on patients living with Primary Bilateral Macronodular Adrenal Hyperplasia. This condition occurs when the two adrenal glands, which are small organs located on top of the kidneys, develop many large lumps that cause them to produce too much cortisol. Too much of this hormone can lead to Cushing Syndrome, a condition that often results in hypertension (high blood pressure) and diabetes (high blood sugar levels).

The purpose of this study is to see if inhibiting the production of cortisol can help improve blood pressure and blood sugar control. Participants will be assigned to receive either metyrapone or a placebo. During the study, various health measurements will be taken to track changes in hormone levels, weight, and blood sugar levels over several months.

The research process

The trial runs in 2 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    <b>medication administration</b>

    Participation involves taking either the test medication, known as metyrapone, or a placebo, which is an inactive substance that looks like the medicine.

    If assigned to the test medication, a daily dose of 2000 mg of metyrapone is taken in the form of soft capsules by mouth.

    This treatment period continues for a total of 6 months.

  2. Step 2

    <b>medical monitoring and assessments</b>

    Assessments are conducted at the beginning of the study, at 3 months, and at 6 months to track changes in health.

    Tests include measuring cortisol, a hormone, through urine, saliva, and blood levels.

    Measurements are taken for blood pressure, blood sugar (including continuous glucose monitoring), body mass index (a measure of weight relative to height), and cholesterol levels.

    Evaluations of insulin sensitivity (how the body uses sugar) and blood sugar control (HbA1c) are performed.

    Questionnaires are completed at these intervals to assess quality of life and mood.

    Information regarding any side effects or adverse events is collected throughout the process.

Who can join the trial?

10 criteria

  • You must be at least 18 years old.
  • You must be able to speak French.
  • You must have Primary Bilateral Macronodular Adrenal Hyperplasia, which is a condition where both adrenal glands (small organs located above the kidneys) have multiple large growths or nodules. This must be confirmed by medical scans like a CT-scan or MRI.
  • You must have mild autonomous cortisol secretion, meaning your adrenal glands are producing a small, uncontrolled amount of cortisol (a hormone produced by the body). This is confirmed by specific laboratory tests involving dexamethasone (a steroid used in testing) and measuring urinary free cortisol (the amount of cortisol passed in urine over 24 hours).
  • The excess cortisol must come from the adrenal glands, which is confirmed by checking your levels of ACTH (a hormone that signals the adrenal glands to produce cortisol) through blood tests.
  • You must have hypertension (high blood pressure, defined as a top number higher than 140 or a bottom number higher than 90) or diabetes (a condition affecting blood sugar levels) that can be managed with medication.
  • Women who are able to become pregnant must have a negative bHCG test (a blood test to confirm pregnancy is not present) and must use a highly effective form of contraception (birth control).
  • Men must use an effective method of contraception.
  • You must provide a signed written informed consent, which is a document stating that you understand the study and agree to participate.
  • You must be covered by social security or a similar health insurance system.

Who cannot join the trial?

15 criteria

  • You cannot join if your Cushing syndrome (a condition caused by too much cortisol in the body) is caused by something other than PBMAH (a specific condition where both adrenal glands grow small lumps).
  • You cannot participate if you are pregnant or breastfeeding.
  • You cannot participate if you have taken steroidogenesis inhibitors (medicines that stop the body from making certain hormones) less than 6 weeks before starting the study.
  • You cannot join if you have a medical condition that is life-threatening (could cause death) within the next year.
  • You cannot participate if you have major psychiatric disorders (serious mental health conditions) that might make it difficult to follow the study rules.
  • You cannot join if you are receiving AME (state medical aid or government-funded healthcare).
  • You cannot participate if you are already part of another clinical trial using medicines for humans.
  • You cannot join if you have primary adrenal insufficiency (a condition where the adrenal glands do not produce enough essential hormones).
  • You cannot participate if you are currently using glucocorticoids (a type of steroid hormone medicine, either taken throughout the body or in high doses locally).
  • You cannot join if you have a medical reason why you should not take steroidogenesis inhibitors.
  • You cannot participate if you have a hypersensitivity (an allergy or extreme sensitivity) to the active medicine or any of the other ingredients used to make it.
  • You cannot join if your diabetes is not well-controlled, specifically if your HBA1c (a blood test that shows your average blood sugar levels over the past few months) is higher than 9%.
  • You cannot participate if you are taking loop diuretics or thiazide/thiazide-like diuretics (water pills used to reduce fluid) that could cause hypokalemia (low levels of potassium in the blood).
  • You cannot join if you are at a high risk of cardiac rhythm disorders (irregular heartbeats) based on your QT/QTc interval (a specific measurement of the time it takes for your heart muscle to recharge between beats), which must be greater than 470ms for women or 450ms for men.
  • You cannot participate if you are taking medicines known to cause QT prolongation (a condition where the heart's electrical recharging process takes too long).
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Investigated drugs

Metyrapone is a medication used to lower the amount of cortisol produced by the body. In this study, it is being tested to see if it can help improve blood pressure and blood sugar levels in people with specific conditions involving the adrenal glands.

What is already known about the treatment

Metyrapone - This medication is taken by mouth in the form of a soft capsule. It is a known drug used to manage conditions where the body produces too much cortisol, such as Cushing syndrome. It works by blocking a specific enzyme in the adrenal glands that is responsible for making cortisol, thereby lowering the hormone levels in the blood. This drug is classified as a steroidogenesis inhibitor, which means it stops the production of steroid hormones.

Investigated diseases

Primary Bilateral Macronodular Adrenal Hyperplasia - This condition occurs when both adrenal glands develop multiple large nodules. These nodules cause the glands to produce excess amounts of cortisol independently of the body's normal regulatory signals. As the disease progresses, the continuous hormone production can lead to various metabolic imbalances. The condition is often characterized by a gradual increase in hormone levels over time.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-507010-27-00Protocol codeAPHP211003Estimated enrolment70 patientsSponsorAssistance Publique Hopitaux De Paris

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