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

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ClinicalTrials.gov Identifier: NCT00409175
Recruitment Status : Completed
First Posted : December 8, 2006
Results First Posted : December 17, 2012
Last Update Posted : December 17, 2012
Sponsor:
Information provided by (Responsible Party):
Pfizer

Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition: Familial Amyloid Polyneuropathy
Interventions: Drug: Fx-1006A
Drug: Placebo

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

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

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.
No text entered.

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 
Overall Participants Analyzed 
[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 
[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.
Gender [1] 
[Units: Participants]
 		     
Female   32   35   67 
Male   32   26   58 
[1] Out of a total of 128 participants, baseline characteristic (Gender) was available for only 125 participants who were included in ITT population.


  Outcome Measures

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 ]
  Show Outcome Measure 1

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 ]
  Show Outcome Measure 2

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 ]
  Show Outcome Measure 3

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 ]
  Show Outcome Measure 4

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 ]
  Show Outcome Measure 5

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 ]
  Show Outcome Measure 6

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 ]
  Show Outcome Measure 7

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 ]
  Show Outcome Measure 8

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

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

Measure Type Secondary
Measure Title Percentage of Participants With Stabilized Transthyretin (TTR) Tetramer
Measure Description TTR tetramer was assessed using a validated immunoturbidimetric assay. The Fraction of Initial (FOI) is the ratio of the measured TTR tetramer concentration after denaturation to the measured TTR tetramer concentration before denaturation. TTR tetramer stabilization is based on the difference between the on-treatment FOI and the baseline FOI expressed as a percentage of the baseline FOI.
Time Frame Week 8, Month 6, 12, 18  

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.
ITT population. 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. ‘n’ = those participants who were evaluable for this measure at given time points for each group respectively.

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.

Measured Values
   Tafamidis   Placebo 
Participants Analyzed 
[Units: Participants]
 		 63   60 
Percentage of Participants With Stabilized Transthyretin (TTR) Tetramer 
[Units: Percentage of participants]
Number (95% Confidence Interval) 		   
Week 8 (n=63, 60)   98.4 
 (95.3 to 100.0)  		 6.7 
 (0.4 to 13.0) 
Month 6 (n= 59, 58)   100.0 
 (100.0 to 100.0)  		 5.2 
 (0.0 to 10.9) 
Month 12 (n=48, 50)   97.9 
 (93.9 to 100.0)  		 2.0 
 (0.0 to 5.9) 
Month 18 (n=48, 44)   97.9 
 (93.9 to 100.0)  		 0.0 
 (0.0 to 0.0) 

No statistical analysis provided for Percentage of Participants With Stabilized Transthyretin (TTR) Tetramer




  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
Instead of the intended endpoint 'heat pain and cooling threshold', results of 'summated 3 score for small nerve fiber function' were reported.


  More Information

Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Pfizer ClinicalTrials.gov Call Center
Organization: Pfizer, Inc.
phone: 1-800-718-1021
e-mail: ClinicalTrials.gov_Inquiries@pfizer.com


Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):


Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT00409175     History of Changes
Other Study ID Numbers: FX-005
B3461020
First Submitted: December 6, 2006
First Posted: December 8, 2006
Results First Submitted: November 16, 2012
Results First Posted: December 17, 2012
Last Update Posted: December 17, 2012