New pill hopes to tame rare ROSAH syndrome
NCT ID NCT06395285
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage trial tests an oral drug called DF-003 in 12 people with ROSAH syndrome, a rare genetic condition causing eye inflammation, enlarged spleen, and headaches. The main goal is to check safety and how the body processes the drug, while also watching for improvements in eye swelling. It is a small, first-in-human study, so results are preliminary.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
DF-003 (an oral drug)
What this could lead to
If successful, this could point toward a treatment to control inflammation and eye damage in ROSAH syndrome.
What could go wrong
This is a very early, small Phase 1 trial with only 12 people, focused on safety. It may not show clear benefit, and side effects are unknown.
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: •••••@•••••
Locations
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Duke Eye Center - Duke University Hospital
RECRUITINGDurham, North Carolina, 27705, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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John A. Moran Eye Center - University of Utah Health
RECRUITINGSalt Lake City, Utah, 84132, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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National Institutes of Health Clinical Center
WITHDRAWNBethesda, Maryland, 20892, United States
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Peking Union Medical College Hospital
RECRUITINGBeijing, 100730, China
Contact Email: •••••@•••••
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Save Sight Institute - University of Sydney Eye Hospital
RECRUITINGSydney, New South Wales, 2000, Australia
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••