NCT01435655

Brief Summary

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

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
10

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Nov 2011

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

September 13, 2011

Completed
3 days until next milestone

First Posted

Study publicly available on registry

September 16, 2011

Completed
2 months until next milestone

Study Start

First participant enrolled

November 1, 2011

Completed
2.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2014

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2014

Completed
1.6 years until next milestone

Results Posted

Study results publicly available

September 9, 2015

Completed
Last Updated

September 9, 2015

Status Verified

August 1, 2015

Enrollment Period

2.3 years

First QC Date

September 13, 2011

Results QC Date

February 24, 2015

Last Update Submit

August 10, 2015

Conditions

Transthyretin Familial Amyloid Polyneuropathy

Outcome Measures

Primary Outcomes (1)

  • Number of Participants With Transthyretin (TTR) Stabilization at Week 8 Compared With Baseline as Measured by a Validated Immunoturbidimetric Assay

    TTR tetramer level for each plasma sample was assessed using a validated immunoturbidimetric assay before and after urea denaturation. The Fraction of Initial (FOI) tetramer concentration is the ratio of the measured TTR tetramer concentration after denaturation to the measured TTR tetramer average concentration before denaturation. TTR tetramer stabilization is based on the difference between the on-treatment FOI and the baseline FOI expressed as a percentage of the baseline FOI. A patient who has the "TTR stabilization" is defined as the patient whose percent stabilization is equal to or more than 32%.

    8 weeks

Secondary Outcomes (7)

  • Change From Baseline in Neuropathy Impairment Score (NIS); NIS (Total), NIS-LL (Lower Limb) and NIS-UL (Upper Limb) at Week 26, Week 52 and Week 78

    Baseline, Week 26, Week 52, Week 78

  • Change From Baseline in Scores of the Total Quality of Life (TQOL) and 5 Domains as Measured by the Norfolk QOL - Diabetic Neuropathy (Norfolk QOL-DN) at Week 26, Week 52 and Week 78.

    Baseline, Week 26, Week 52, Week 78

  • Change From Baseline in Summated 7 Nerve Tests Normal Deviate Score (∑ 7 NTs Nds) as Measured by Nerve Conduction Studies (NCS), Vibration Detection Threshold (VDT) and Heart Rate Response to Deep Breathing (HRDB) at Week 26, Week 52, and Week 78

    Baseline, Week 26, Week 52, Week 78

  • Change From Baseline in Summated 3 Nerve Tests Small Fiber Normal Deviate Score (∑ 3 NTSF Nds) as Measured by Cooling and Heat Pain Thresholds by QST and HRDB at Week 26, Week 52 and Week 78

    Baseline, Week 26, Week 52, Week 78

  • Change From Baseline in Modified Body Mass Index (mBMI) at Week 8, Week 26, Week 52 and End of Study

    Baseline, Week 8, Week 26, Week 52, End of Study

  • +2 more secondary outcomes

Other Outcomes (1)

  • Plasma Concentration of Tafamidis at Week 8, Week 26, Week 52 and Week 78

    Week 8, Week 26, Week 52, Week 78

Study Arms (1)

open

EXPERIMENTAL

tafamidis

Drug: tafamidis

Interventions

tafamidisDRUG

tafamidis meglumine 20 mg QD

Also known as: tafamidis meglumine
open

Eligibility Criteria

Age20 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Kumamoto University Hospital/Department of Neurology

Kumamoto, Kumamoto, 860-8556, Japan

Location

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

Matsumoto-shi, Nagano, 390-8621, Japan

Location

Related Publications (1)

  • 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.

    PMID: 31353964DERIVED

Related Links

MeSH Terms

Interventions

tafamidis

Results Point of Contact

Title
Pfizer ClinicalTrials.gov Call Center
Organization
Pfizer, Inc.
ClinicalTrials.gov_Inquiries@pfizer.com
1-800-718-1021

Study Officials

  • Pfizer CT.gov Call Center

    Pfizer

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 13, 2011

First Posted

September 16, 2011

Study Start

November 1, 2011

Primary Completion

February 1, 2014

Study Completion

February 1, 2014

Last Updated

September 9, 2015

Results First Posted

September 9, 2015

Record last verified: 2015-08

Locations

JP(2)