New hope for kids with Cushing's: drug trial launches
NCT ID NCT03708900
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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-••••
Locations
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ABMED Clinical Research Corp
RECRUITINGCape Coral, Florida, 33914, United States
Contact Email: •••••@•••••
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Alder Hey Childrens NHS Foundation Trust
WITHDRAWNLiverpool, L12 2AP, United Kingdom
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Ann & Robert H. Lurie Children's Hospital
RECRUITINGChicago, Illinois, 60611, United States
Contact Email: •••••@•••••
Contact
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Aziendal Ospedaliero Universitaria Pisana Presidio Ospedale di Cisanello
COMPLETEDPisa, PI, 56124, Italy
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CHU Bicetre APHP Paris Saclay
COMPLETEDParis, 94270, France
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Hospital Necker Enfants Malades
COMPLETEDParis, 75015, France
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Multiprofile Hospital for Active Treatment Sveta Marina EAD
WITHDRAWNVarna, 9010, Bulgaria
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National Institute of Child Health and Human Development
COMPLETEDBethesda, Maryland, 20892, United States
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Ospedale Bambino Gesu
COMPLETEDRoma, 00165, Italy
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Robert Debre Hospital
COMPLETEDParis, 75019, France
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Texas Valley Clinical Research
RECRUITINGWeslaco, Texas, 78596, United States
Contact Email: •••••@•••••
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The Royal London Childrens Hospital
WITHDRAWNLondon, E11BB, United Kingdom
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UZ Brussel
COMPLETEDJette, Brussels Capital, 1090, Belgium
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University Clinical Center Ljubljana
WITHDRAWNLjubljana, 1525, Slovenia
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University of California San Francisco UCSF
RECRUITINGSan Francisco, California, 94143, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••