Safety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT00409175
First received: December 6, 2006
Last updated: November 16, 2012
Last verified: November 2012
Results First Received: November 16, 2012  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition: Familial Amyloid Polyneuropathy
Interventions: Drug: Fx-1006A
Drug: Placebo

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups
  Description
Tafamidis Tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily for 18 months.
Placebo Placebo, matched to tafamidis (Fx-1006A) 20 mg capsule, orally once daily for 18 months.

Participant Flow:   Overall Study
    Tafamidis     Placebo  
STARTED     65     63  
COMPLETED     47     44  
NOT COMPLETED     18     19  
Adverse Event                 4                 3  
Withdrawal by Subject                 1                 2  
Negative genotype                 0                 1  
Liver transplantation                 13                 13  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
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Reporting Groups
  Description
Tafamidis Tafamidis (Fx-1006A) 20 milligram (mg) capsule orally once daily for 18 months.
Placebo Placebo, matched to tafamidis (Fx-1006A) 20 mg capsule, orally once daily for 18 months.
Total Total of all reporting groups

Baseline Measures
    Tafamidis     Placebo     Total  
Number of Participants  
[units: participants]
  64     61     125  
Age, Customized [1]
[units: participants]
     
Less than or equal to 65 years     59     58     117  
Greater than 65 years     5     3     8  
Gender [2]
[units: participants]
     
Female     32     35     67  
Male     32     26     58  
[1] Out of a total of 128 participants, baseline characteristic (Age) was available for only 125 participants who were included in intent-to-treat (ITT) population.
[2] Out of a total of 128 participants, baseline characteristic (Gender) was available for only 125 participants who were included in ITT population.



  Outcome Measures
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1.  Primary:   Percentage of Participants With Response to Treatment as Measured by Neuropathy Impairment Score - Lower Limb (NIS-LL) at Month 18   [ Time Frame: Month 18 ]

2.  Primary:   Change From Baseline in Norfolk Quality of Life- Diabetic Neuropathy (QOL-DN) Total Quality of Life (TQOL) Score at Month 18   [ Time Frame: Baseline, Month 18 ]

3.  Secondary:   Change From Baseline in Neuropathy Impairment Score- Lower Limb (NIS-LL) Score at Month 6, 12 and 18   [ Time Frame: Baseline, Month 6, 12, 18 ]

4.  Secondary:   Percentage of Participants With Response to Treatment as Measured by Neuropathy Impairment Score - Lower Limb (NIS-LL) at Month 6 and 12   [ Time Frame: Month 6, 12 ]

5.  Secondary:   Change From Baseline in Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Total Quality of Life (TQOL) Score at Month 6 and 12   [ Time Frame: Baseline, Month 6, 12 ]

6.  Secondary:   Change From Baseline in Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Domain Scores at Month 6, 12 and 18   [ Time Frame: Baseline, Month 6, 12, 18 ]

7.  Secondary:   Change From Baseline in Summated 7 Score for Large Nerve Fiber Function at Month 6, 12 and 18   [ Time Frame: Baseline, Month 6, 12, 18 ]

8.  Secondary:   Change From Baseline in Summated 3 Score for Small Nerve Fiber Function at Month 6, 12 and 18   [ Time Frame: Baseline, Month 6, 12, 18 ]

9.  Secondary:   Change From Baseline in Modified Body Mass Index (mBMI) at Month 6, 12 and 18   [ Time Frame: Baseline, Month 6, 12, 18 ]

10.  Secondary:   Percentage of Participants With Stabilized Transthyretin (TTR) Tetramer   [ Time Frame: Week 8, Month 6, 12, 18 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats


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