Not recruitingRare disease

Study of Osilodrostat for Children and Adolescents with Cushing's Disease

Verified siteRegistered drugNo placebo

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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 Cushing's disease, a condition where the body produces too much of a hormone called cortisol. The study involves a medication called osilodrostat, also known by its code name LCI699. Osilodrostat is available in the form of film-coated tablets, with doses of 1 mg, 5 mg, and 10 mg. The purpose of the study is to understand how osilodrostat is processed in the body and how well it is tolerated by children and adolescents aged 6 to under 18 years who have Cushing's disease.

Participants in the study will take osilodrostat orally, meaning they will swallow the tablets. The study will last for a period of up to 48 weeks. During this time, researchers will monitor how the medication affects the levels of cortisol in the body and assess any side effects. The study aims to gather information on the safety and effectiveness of osilodrostat in managing Cushing's disease in young patients.

Throughout the study, participants will have regular check-ups to track their progress and ensure their well-being. The study will help determine if osilodrostat can be a suitable treatment option for children and adolescents with Cushing's disease, especially for those who have not had success with surgery or are waiting for surgery. The findings from this study could provide valuable insights into managing this condition in younger patients.

The research process

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

Step 1
Initial assessment
Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes verifying age, body weight, and a confirmed diagnosis of Cushing's disease.
The ability to swallow the study drug tablets whole is also assessed.
Step 2
Medication administration
The study involves taking osilodrostat, which is available in film-coated tablets of 1 mg, 5 mg, and 10 mg.
The medication is taken orally. The specific dosage and frequency are determined by the study team based on individual needs.
Step 3
Monitoring and evaluation
Regular monitoring is conducted to evaluate the pharmacokinetics, which is how the body absorbs, distributes, metabolizes, and excretes the drug.
The study also assesses the pharmacodynamics, which is the effect of the drug on the body, and its tolerability.
Step 4
Safety assessments
Safety assessments are performed throughout the study to monitor any side effects or adverse reactions to the medication.
The goal is to ensure the treatment is safe and well-tolerated.
Step 5
End of study
The study is estimated to conclude by November 30, 2025.
Final evaluations are conducted to assess the overall outcomes and effectiveness of the treatment.

Who can join the trial?

6 criteria

Children and teenagers aged 6 to less than 18 years old with **Cushing's disease**. This means the condition is caused by the body itself, not by external factors.
They must have tried surgery that didn't work, are waiting for surgery, or surgery isn't an option right now.
They must weigh more than 30 kilograms (about 66 pounds).
They need to have a confirmed diagnosis of **Cushing's disease**.
They must be able to swallow the study medicine in tablet form without crushing or splitting it.
Their parents or legal guardians must be able to give permission for them to join the study.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Azienda Ospedaliero Universitaria Pisana
Verified
Pisa, Italy
Bicetre Hospital
Verified
Le Kremlin-Bicetre, France
Hopital Necker Enfants Malades
Verified
Paris, France

Trial locations

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Investigated drugs

Osilodrostat is a medication being studied for its effects in children and adolescents with Cushing's disease. It works by reducing the production of cortisol, a hormone that is often present in excess in patients with this condition. The trial aims to understand how the body processes and responds to this medication, as well as to assess its safety and tolerability in younger patients.

Osilodrostat

What is already known about the treatment

Osilodrostat – Osilodrostat is administered orally in tablet form. It is currently being studied in clinical trials, particularly in a phase II study focusing on its use in children and adolescents with Cushing's disease. The main therapeutic indication for osilodrostat is the treatment of Cushing's disease, a condition characterized by excessive cortisol production. At the molecular level, osilodrostat works by inhibiting an enzyme called 11β-hydroxylase, which is involved in cortisol synthesis, thereby reducing cortisol levels in the body. It is classified as an adrenal steroidogenesis inhibitor.

Investigated diseases

Cushing's Disease – Cushing's Disease is a condition caused by an excess of the hormone cortisol in the body, often due to a pituitary gland tumor that produces too much adrenocorticotropic hormone (ACTH). This leads to symptoms such as weight gain, particularly around the abdomen and face, thinning skin, easy bruising, and high blood pressure. Over time, individuals may also experience muscle weakness, fatigue, and mood changes. The disease can affect bone health, leading to osteoporosis, and may cause irregular menstrual cycles in women. It is a rare condition that requires careful management to control hormone levels and alleviate symptoms.