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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT01435655
First received: September 13, 2011
Last updated: March 28, 2014
Last verified: March 2014
  Purpose

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


Condition Intervention Phase
Transthyretin Familial Amyloid Polyneuropathy
Drug: tafamidis
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Pharmacodynamics Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: The Effect Of Transthyretin Stabilization, Safety, Tolerability, Efficacy And Pharmacokinetics Of Orally Administered Tafamidis In Transthyretin Amyloid Polyneuropathy Patients With V30M Or Non-V30M Transthyretin: A Phase 3, Open-Label

Resource links provided by NLM:


Further study details as provided by Pfizer:

Primary Outcome Measures:
  • TTR stabilization at Week 8 compared with Baseline, as measured by a validated immunoturbidimetric assay. [ Time Frame: 8 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Change from baseline on the following scales: Neuropathy Impairment Score;QOL;"Σ7 NTs NDS" as measured by nerve conduction studies (NCS), vibration detection threshold (VDT) and heart rate response to deep breathing (HRDB); [ Time Frame: 3 years ] [ Designated as safety issue: No ]
  • Change from baseline on the following scales: "Σ3 NTSF NDS" as measured by cooling and heat pain thresholds utilizing CASE IV and heart rate response to deep breathing (HRDB); mBMI; Ambulatory status. [ Time Frame: 3 years ] [ Designated as safety issue: No ]
  • Transthyretin (TTR) stabilization at each follow up visit after Week 8. [ Time Frame: 3 years ] [ Designated as safety issue: No ]
  • Blood samples for determination of plasma levels of tafamidis will be collected at Baseline and during the visits at Week 2. [ Time Frame: 2 weeks ] [ Designated as safety issue: No ]
  • Blood samples for determination of plasma levels of tafamidis will be collected at Week 4. [ Time Frame: 4 weeks ] [ Designated as safety issue: No ]
  • Blood samples for determination of plasma levels of tafamidis will be collected Week 8. [ Time Frame: 8 weeks ] [ Designated as safety issue: No ]
  • Blood samples for determination of plasma levels of tafamidis will be collected at Week 26. [ Time Frame: 26 weeks ] [ Designated as safety issue: No ]
  • Blood samples for determination of plasma levels of tafamidis will be collected at Week 52. [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
  • Blood samples for determination of plasma levels of tafamidis will be collected at Week 78. [ Time Frame: 78 weeks ] [ Designated as safety issue: No ]
  • Blood samples for determination of plasma levels of tafamidis will be collected at End of study. [ Time Frame: 3 years ] [ Designated as safety issue: No ]

Enrollment: 10
Study Start Date: November 2011
Study Completion Date: February 2014
Primary Completion Date: February 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: open
tafamidis
Drug: tafamidis
tafamidis meglumine 20 mg QD
Other Name: tafamidis meglumine

  Eligibility

Ages Eligible for Study:   20 Years to 75 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Transthyretin amyloid polyneuropathy with V30M or non-V30M transthyretin mutation.

Exclusion Criteria:

  • Primary amyloidosis and secondary amyloidosis.
  • History of liver transplant.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01435655

Locations
Japan
Pfizer Investigational Site
Kumamoto-shi, Kumamoto, Japan
Pfizer Investigational Site
Matsumoto-shi, Nagano, Japan
Sponsors and Collaborators
Pfizer
Investigators
Study Director: Pfizer CT.gov Call Center Pfizer
  More Information

Additional Information:
No publications provided

Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT01435655     History of Changes
Other Study ID Numbers: B3461010
Study First Received: September 13, 2011
Last Updated: March 28, 2014
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Polyneuropathies
Amyloid Neuropathies, Familial
Amyloid Neuropathies
Peripheral Nervous System Diseases
Neuromuscular Diseases
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Amyloidosis, Familial
Metabolism, Inborn Errors
Metabolic Diseases
Amyloidosis
Proteostasis Deficiencies
Meglumine
Contrast Media
Diagnostic Uses of Chemicals
Pharmacologic Actions

ClinicalTrials.gov processed this record on July 31, 2014