The Effect Of Tafamidis For The Transthyretin Amyloid Polyneuropathy Patients With V30M Or Non-V30M Transthyretin

Pfizer

Status and phase: Completed; Phase 3

Conditions: Transthyretin Familial Amyloid Polyneuropathy

Treatments: Drug: tafamidis

Study type: Interventional

Funder types: Industry

Identifiers

NCT01435655

B3461010

Details and patient eligibility

About

Tafamidis has been developed as an oral specific stabilizer of transthyretin tetramer.

Enrollment: 10 patients

Sex: All

Ages: 20 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Transthyretin amyloid polyneuropathy with V30M or non-V30M transthyretin mutation.
• Subject had amyloid documented by biopsy in accordance with institutional site standard of care.

Exclusion criteria

• Primary amyloidosis and secondary amyloidosis.
• History of liver transplant.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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10 participants in 1 patient group

open

Experimental group

Description:

tafamidis

Treatment:

Drug: tafamidis