Clinical study of tolcapone for transthyretin (TTR)-type cerebral amyloid angiopathy in patients with familial amyloid polyneuropahty (FAP)

Clinical study of tolcapone for transthyretin (TTR)-type cerebral amyloid angiopathy in patients with familial amyloid polyneuropahty (FAP) - Clinical study of tolcapone for ATTR-type CAA

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

JPRN

Registry ID

JPRN-UMIN000030341

Enrollment

Registered

2018-01-01

Start date

2018-01-01

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Familial amyloid polyneuropahty

Interventions

Administer tolcapone at 300 mg/day orally for 7 days. Administer tolcapone at 600 mg/day orally for 7 days.

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Biopsy proven amyloid deposition Genotyping of variant TTR Age > 20

Exclusion criteria

Exclusion criteria: Asymptomatic mutation carriers Contraindication for tolcapone An ALT or AST measurement > 2 times the ULN (Upper Limit of Normal) Treatment with NSAID or a known TTR binder within the last 4 weeks Stage 3 disease

Design outcomes

Primary

Measure	Time frame
stability ratio of CSF TTR tetramer	—

Countries

Japan

Contacts

Public ContactYoshiki Sekijima

Shinshu University School of Medicine Department of Medicine (Neurology & Rheumatology)

sekijima@shinshu-u.ac.jp0263372673

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026