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

NCT ID: NCT01435655

Last Updated: 2015-09-09

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

10 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-11-30

Study Completion Date

2014-02-28

Brief Summary

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

Detailed Description

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Conditions

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Transthyretin Familial Amyloid Polyneuropathy

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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open

tafamidis

Group Type EXPERIMENTAL

tafamidis

Intervention Type DRUG

tafamidis meglumine 20 mg QD

Interventions

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tafamidis

tafamidis meglumine 20 mg QD

Intervention Type DRUG

Other Intervention Names

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tafamidis meglumine

Eligibility Criteria

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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.
Minimum Eligible Age

20 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Pfizer

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Pfizer CT.gov Call Center

Role: STUDY_DIRECTOR

Pfizer

Locations

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Kumamoto University Hospital/Department of Neurology

Kumamoto, Kumamoto, Japan

Site Status

Shinshu University Hospital/Department of Medicine (Neurology and Reumatology)

Matsumoto-shi, Nagano, Japan

Site Status

Countries

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Japan

References

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Huber P, Flynn A, Sultan MB, Li H, Rill D, Ebede B, Gundapaneni B, Schwartz JH. A comprehensive safety profile of tafamidis in patients with transthyretin amyloid polyneuropathy. Amyloid. 2019 Dec;26(4):203-209. doi: 10.1080/13506129.2019.1643714. Epub 2019 Jul 27.

Reference Type DERIVED
PMID: 31353964 (View on PubMed)

Related Links

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Other Identifiers

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B3461010

Identifier Type: -

Identifier Source: org_study_id

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