Clinical trial NCT01435655: The Effect Of Tafamidis For The Transthyretin Amyloid Polyneuropathy Patients With V30M Or Non-V30M Transthyretin
Official title: The Effect On Transthyretin Stabilization, Safety, Tolerablity, Efficacy And Pharmacokinetics Of Orally Administered Tafamidis In Transthyretin Amyloid Polyneuropathy Patients With V30m Or Non-v30m Transthyretin: A Phase Iii, Open-label Study
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Transthyretin Familial Amyloid Polyneuropathy
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2011-09-16
Enrollment: 10
Sex: ALL
Minimum age (years): 20
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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


Eligibility criteria: 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.

Primary outcomes:
measure: Number of Participants With Transthyretin (TTR) Stabilization at Week 8 Compared With Baseline as Measured by a Validated Immunoturbidimetric Assay
timeFrame: 8 weeks
Secondary outcomes:
measure: 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
timeFrame: Baseline, Week 26, Week 52, Week 78
measure: 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.
timeFrame: Baseline, Week 26, Week 52, Week 78
measure: 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
timeFrame: Baseline, Week 26, Week 52, Week 78
measure: 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
timeFrame: Baseline, Week 26, Week 52, Week 78
measure: Change From Baseline in Modified Body Mass Index (mBMI) at Week 8, Week 26, Week 52 and End of Study
timeFrame: Baseline, Week 8, Week 26, Week 52, End of Study
measure: Change From Baseline in Ambulatory Status at Week 26, Week 52 and Week 78
timeFrame: Baseline, Week 26, Week 52, Week 78
measure: Number of Participants With Transthyretin (TTR) Stabilization at Week 26, Week 52, and Week 78 Compared With Baseline as Measured by a Validated Immunoturbidimetric Assay
timeFrame: Baseline, Week 26, Week 52, Week 78
Other outcomes:
measure: Plasma Concentration of Tafamidis at Week 8, Week 26, Week 52 and Week 78
timeFrame: Week 8, Week 26, Week 52, Week 78
Locations:
city: Kumamoto-shi
country: Japan
facility: Kumamoto University Hospital/Department of Neurology
state: Kumamoto
city: Matsumoto-shi
country: Japan
facility: Shinshu University Hospital/Department of Medicine (Neurology and Reumatology)
state: Nagano
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References:
citation: 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.