ACys amyloidosis

MONDO:0007098

Hereditary cerebral hemorrhage with amyloidosis (HCHWA), Icelandic type is a form of HCHWA characterized by an age of onset of 20-30 years, systemic amyloidosis and recurrent lobar intracerebral hemorrhages.

Also known as: cerebral amyloid angiopathy, CST3-related amyloidosis, HCHWA, Icelandic type, amyloidosis, Cerebroarterial, Icelandic type, cerebral hemorrhage, hereditary, with amyloidosis, cystatin amyloidosis, hereditary cerebral haemorrhage with amyloidosis, hereditary cerebral haemorrhage with amyloidosis, Icelandic type

14 clinical trials for this condition and its sub-types, 1 tagged with ACys amyloidosis itself.

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Where it sits in the disease tree

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Part of

↑ Cerebral amyloid angiopathy (17)

Including sub-types (14) Tagged with ACys amyloidosis (1)

Trials to join now! 7 Not yet recruiting 6 Not yet finished but already full! 1

Experimental drug aims to curb brain bleeds in rare genetic disease

Disease control Ongoing

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 L68…

Phase 2 • Sponsor: Arctic Therapeutics • Aim: Disease control

Last updated Jul 23, 2026 00:00 UTC

ACys amyloidosis

Where it sits in the disease tree

Experimental drug aims to curb brain bleeds in rare genetic disease