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.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Duke Eye Center - Duke University Hospital
RECRUITINGDurham, North Carolina, 27705, United States
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John A. Moran Eye Center - University of Utah Health
RECRUITINGSalt Lake City, Utah, 84132, United States
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National Institutes of Health Clinical Center
WITHDRAWNBethesda, Maryland, 20892, United States
-
Peking Union Medical College Hospital
RECRUITINGBeijing, 100730, China
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Save Sight Institute - University of Sydney Eye Hospital
RECRUITINGSydney, New South Wales, 2000, Australia
More trials for these conditions
Other studies related to the condition(s) this trial covers.