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
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
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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.
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  
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
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Time Frame No text entered.
Additional Description The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in one subject and as nonserious in another subject, or one subject may have experienced both a serious and nonserious event during the study.

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.

Serious Adverse Events
    Tafamidis     Placebo  
Total, serious adverse events      
# participants affected / at risk     6/65 (9.23%)     5/63 (7.94%)  
Blood and lymphatic system disorders      
Anaemia * 1    
# participants affected / at risk     0/65 (0.00%)     1/63 (1.59%)  
Cardiac disorders      
Conduction disorder * 1    
# participants affected / at risk     1/65 (1.54%)     0/63 (0.00%)  
Cardiac amyloidosis * 1    
# participants affected / at risk     0/65 (0.00%)     1/63 (1.59%)  
Gastrointestinal disorders      
Nausea * 1    
# participants affected / at risk     0/65 (0.00%)     1/63 (1.59%)  
Vomiting * 1    
# participants affected / at risk     0/65 (0.00%)     1/63 (1.59%)  
General disorders      
Catheter site phlebitis * 1    
# participants affected / at risk     0/65 (0.00%)     1/63 (1.59%)  
Oedema peripheral * 1    
# participants affected / at risk     0/65 (0.00%)     1/63 (1.59%)  
Infections and infestations      
Urinary tract infection * 1    
# participants affected / at risk     2/65 (3.08%)     0/63 (0.00%)  
Localised infection * 1    
# participants affected / at risk     1/65 (1.54%)     0/63 (0.00%)  
Pneumonia * 1    
# participants affected / at risk     1/65 (1.54%)     0/63 (0.00%)  
Viral infection * 1    
# participants affected / at risk     1/65 (1.54%)     0/63 (0.00%)  
Cellulitis * 1    
# participants affected / at risk     0/65 (0.00%)     1/63 (1.59%)  
Lymphangitis * 1    
# participants affected / at risk     0/65 (0.00%)     1/63 (1.59%)  
Staphylococcal infection * 1    
# participants affected / at risk     0/65 (0.00%)     1/63 (1.59%)  
Injury, poisoning and procedural complications      
Burns third degree * 1    
# participants affected / at risk     0/65 (0.00%)     1/63 (1.59%)  
Nervous system disorders      
Syncope * 1    
# participants affected / at risk     0/65 (0.00%)     1/63 (1.59%)  
Respiratory, thoracic and mediastinal disorders      
Pneumothorax * 1    
# participants affected / at risk     0/65 (0.00%)     1/63 (1.59%)  
Skin and subcutaneous tissue disorders      
Urticaria * 1    
# participants affected / at risk     1/65 (1.54%)     0/63 (0.00%)  
Skin ulcer * 1    
# participants affected / at risk     0/65 (0.00%)     1/63 (1.59%)  
Vascular disorders      
Hypertensive emergency * 1    
# participants affected / at risk     0/65 (0.00%)     1/63 (1.59%)  
* Events were collected by non-systematic assessment
1 Term from vocabulary, MedDRA 10.0




  Other Adverse Events


  More Information
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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.


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.  


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


No publications provided


Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT00409175     History of Changes
Other Study ID Numbers: FX-005, B3461020
Study First Received: December 6, 2006
Results First Received: November 16, 2012
Last Updated: November 16, 2012
Health Authority: United States: Food and Drug Administration