Experimental drug aims to curb brain bleeds in rare genetic disease
NCT ID NCT07716865
First seen Jul 21, 2026 · Last updated Jul 22, 2026 · Updated 1 time
Summary
This study tests an experimental drug, NPI-001 (AT-001), in people with hereditary cystatin C amyloid angiopathy (HCCAA), a rare genetic condition that causes repeated brain bleeding and stroke. The trial includes about 15 participants aged 12 and older who carry the specific L68Q gene mutation. Researchers are evaluating the drug's safety and whether it can reduce bleeding events and lower amyloid buildup in the skin.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- an experimental drug called NPI-001 (also known as AT-001 or NAC-amid), taken as tablets twice daily
- What this could lead to
- If successful, this could point toward the first treatment to reduce brain bleeding and slow disease progression in people with HCCAA.
- What could go wrong
- This is a small, early-phase trial with only 15 participants, so results may not apply broadly. The drug's safety and effectiveness are still unproven, and side effects are possible.
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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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Locations
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Landspitali University Hospital
Reykjavik, 108, Iceland
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