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Trial record 3 of 7 for:    "Amyloid neuropathy"
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The Effect Of Tafamidis For The Transthyretin Amyloid Polyneuropathy Patients With V30M Or Non-V30M Transthyretin

This study is currently recruiting participants.
Verified October 2012 by Pfizer
Sponsor:
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT01435655
First received: September 13, 2011
Last updated: May 1, 2013
Last verified: October 2012
History of Changes
  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:

Drug Information available for: Meglumine
U.S. FDA Resources

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 ]

Estimated Enrollment: 10
Study Start Date: November 2011
Estimated Study Completion Date: September 2013
Estimated Primary Completion Date: September 2013 (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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01435655

Contacts
Contact: Pfizer CT.gov Call Center 1-800-718-1021

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

Additional Information:
To obtain contact information for a study center near you, click here.  This link exits the ClinicalTrials.gov site

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: May 1, 2013
Health Authority: Japan: Pharmaceuticals and Medical Devices Agency

Additional relevant MeSH terms:
Amyloid Neuropathies, Familial
Amyloid Neuropathies
Polyneuropathies
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

ClinicalTrials.gov processed this record on May 19, 2013