Phase 3 N=10 Treatment

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

Transthyretin Familial Amyloid Polyneuropathy

Bottom Line

View on ClinicalTrials.gov: NCT01435655 ↗

Enrolled (actual)

Serious AEs

70.0%

Results posted

Sep 2015

Primary outcome: Primary: Number of Participants With Transthyretin (TTR) Stabilization at Week 8 Compared With Baseline as Measured by a Validated Immunoturbidimetric Assay — 10 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: tafamidis (Drug)
Age: Adult, Older Adult · 20+ yrs
Sex: All
Sponsor: Pfizer
Primary completion: Feb 2014

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants With Transthyretin (TTR) Stabilization at Week 8 Compared With Baseline as Measured by a Validated Immunoturbidimetric Assay	10	—
SECONDARY 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	31.03; 4.37; 8.30; 9.90; 16.99; 2.06	—
SECONDARY 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.	52.9; 11.8; 9.1; 10.8; 25.6; 4.8	—
SECONDARY 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	8.33; 0.55; 1.04; 1.92	—
SECONDARY 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	6.39; 0.16; -0.13; 0.40	—
SECONDARY Change From Baseline in Modified Body Mass Index (mBMI) at Week 8, Week 26, Week 52 and End of Study	805.70; 74.86; 26.61; 64.86; 53.65	—
SECONDARY Change From Baseline in Ambulatory Status at Week 26, Week 52 and Week 78	0; 3; 4; 2; 1; 0	—
SECONDARY 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	10; 9; 8	—

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.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01435655). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.