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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT01435655
• Date of registration: 13/09/2011
• Prospective Registration: Yes
• Primary sponsor: Pfizer
• Public title: The Effect Of Tafamidis For The Transthyretin Amyloid Polyneuropathy Patients With V30M Or Non-V30M Transthyretin
• Scientific 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
• Date of first enrolment: November 2011
• Target sample size: 10
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01435655
• Study type: Interventional
• Phase: Phase 3

Countries of recruitment

Japan

Contacts

• Name: Pfizer CT.gov Call Center
• Affiliation: Pfizer

Key inclusion & exclusion 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.



Age minimum: 20 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Transthyretin Familial Amyloid Polyneuropathy

Intervention(s)

Drug: tafamidis

Primary Outcome(s)

Number of Participants With Transthyretin (TTR) Stabilization at Week 8 Compared With Baseline as Measured by a Validated Immunoturbidimetric Assay [Time Frame: 8 weeks]

Secondary Outcome(s)

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. [Time Frame: Baseline, Week 26, Week 52, Week 78]

Change From Baseline in Ambulatory Status at Week 26, Week 52 and Week 78 [Time Frame: Baseline, Week 26, Week 52, Week 78]

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 [Time Frame: 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 [Time Frame: Baseline, Week 8, Week 26, Week 52, End of Study]

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 [Time Frame: 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 [Time Frame: Baseline, Week 26, Week 52, Week 78]

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 [Time Frame: Baseline, Week 26, Week 52, Week 78]

Secondary ID(s)

B3461010

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 09/09/2015
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01435655