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

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What is this study about?

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.

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.

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.

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.

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.

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 Study?

  • 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.

Who Cannot Join the Study?

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Hopital Necker Enfants Malades Paris France

Other Sites

Site Name City Country Status
Robert Debre University Hospital Paris France
Vrije Universiteit Brussel Jette Belgium
Ospedale Pediatrico Bambino Gesu’ Rome Italy
Bicetre Hospital Le Kremlin-Bicetre France
Azienda Ospedaliero Universitaria Pisana Pisa Italy

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Belgium Belgium
Not recruiting
01.03.2019
France France
Not recruiting
01.03.2019
Italy Italy
Not recruiting
01.03.2019

Trial locations

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.

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.

Trial ID:
2024-516825-30-00
Protocol code:
CLCI699C2203
NCT ID:
NCT03708900
Trial Phase:
Therapeutic exploratory (Phase II)

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