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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 ]
  Hide Outcome Measure 3

Measure Type Secondary
Measure Title Change From Baseline in Neuropathy Impairment Score- Lower Limb (NIS-LL) Score at Month 6, 12 and 18
Measure Description NIS-LL: assessed muscle weakness, reflexes and sensation; scored separately for left and right limbs. Components of muscle weakness (hip and knee flexion, hip and knee extension, ankle dorsiflexors, ankle plantar flexors, toe extensors, toe flexors) are scored on 0(normal) to 4(paralysis) scale, higher score=greater weakness. Components of reflexes (quadriceps femoris, triceps surae) and sensation (touch pressure, pin-prick, vibration, joint position) were scored 0 = normal, 1= decreased, or 2 = absent. Total possible NIS-LL score range 0-88, higher score=greater impairment.
Time Frame Baseline, 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 included all randomized participants who received at least 1 dose of study medication and had at least 1 post-baseline efficacy assessment for NIS-LL and Norfolk QOL-DN or discontinued study due to death/LT. ‘n’ = those participants who were evaluable for this measure at given time point 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   64   61 
Change From Baseline in Neuropathy Impairment Score- Lower Limb (NIS-LL) Score at Month 6, 12 and 18 
[Units: Units on a scale]
Mean (Standard Deviation) 		   
Baseline (n=64, 61)   8.359  (11.399)   11.445  (13.544) 
Change at Month 6 (n=60, 57)   1.260  (3.007)   2.075  (6.407) 
Change at Month 12 (n=49, 50)   1.005  (3.964)   4.835  (7.697) 
Change at Month 18 (n=48, 47)   2.193  (4.372)   5.402  (8.661) 

No statistical analysis provided for Change From Baseline in Neuropathy Impairment Score- Lower Limb (NIS-LL) Score at Month 6, 12 and 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 ]
  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 ]
  Show Outcome Measure 10


  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