New hope for kids with Cushing's: drug trial launches

NCT ID NCT03708900

Recruiting now Disease control Sponsor: RECORDATI GROUP Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a drug called osilodrostat in children aged 2 to 17 with Cushing's syndrome, a condition where the body makes too much cortisol. The goal is to see how the drug is processed, how well it works, and if it is safe. Only 12 children will take part, and they must weigh over 10 kg and have not been helped by surgery or are waiting for it.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: Osilodrostat (a drug that lowers cortisol levels)
What this could lead to: If it works, this could provide a new treatment option for children with Cushing's syndrome who cannot have surgery or need a temporary solution.
What could go wrong: This is a small, early-phase trial with only 12 participants, so results may not apply to all children. The drug may cause side effects or not control cortisol levels effectively.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

Cushing syndrome pituitary gland disorder

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

CUSHING SYNDROME

Contacts and locations

Locations

ABMED Clinical Research Corp
RECRUITING

Cape Coral, Florida, 33914, United States
Alder Hey Childrens NHS Foundation Trust
WITHDRAWN

Liverpool, L12 2AP, United Kingdom
Ann & Robert H. Lurie Children's Hospital
RECRUITING

Chicago, Illinois, 60611, United States
Aziendal Ospedaliero Universitaria Pisana Presidio Ospedale di Cisanello
COMPLETED

Pisa, PI, 56124, Italy
CHU Bicetre APHP Paris Saclay
COMPLETED

Paris, 94270, France
Hospital Necker Enfants Malades
COMPLETED

Paris, 75015, France
Multiprofile Hospital for Active Treatment Sveta Marina EAD
WITHDRAWN

Varna, 9010, Bulgaria
National Institute of Child Health and Human Development
COMPLETED

Bethesda, Maryland, 20892, United States
Ospedale Bambino Gesu
COMPLETED

Roma, 00165, Italy
Robert Debre Hospital
COMPLETED

Paris, 75019, France
Texas Valley Clinical Research
RECRUITING

Weslaco, Texas, 78596, United States
The Royal London Childrens Hospital
WITHDRAWN

London, E11BB, United Kingdom
UZ Brussel
COMPLETED

Jette, Brussels Capital, 1090, Belgium
University Clinical Center Ljubljana
WITHDRAWN

Ljubljana, 1525, Slovenia
University of California San Francisco UCSF
RECRUITING

San Francisco, California, 94143, United States

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