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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 
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
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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
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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.

Frequency Threshold
Threshold above which other adverse events are reported   5  

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.

Other Adverse Events
    Tafamidis   Placebo
Total, other (not including serious) adverse events     
# participants affected / at risk   57/65 (87.69%)   56/63 (88.89%) 
Blood and lymphatic system disorders     
Anaemia * 1     
# participants affected / at risk   0/65 (0.00%)   4/63 (6.35%) 
Cardiac disorders     
Atrioventricular block first degree * 1     
# participants affected / at risk   2/65 (3.08%)   6/63 (9.52%) 
Ear and labyrinth disorders     
Vertigo * 1     
# participants affected / at risk   3/65 (4.62%)   4/63 (6.35%) 
Eye disorders     
Lacrimation decreased * 1     
# participants affected / at risk   6/65 (9.23%)   7/63 (11.11%) 
Punctate keratitis * 1     
# participants affected / at risk   5/65 (7.69%)   3/63 (4.76%) 
Gastrointestinal disorders     
Diarrhoea * 1     
# participants affected / at risk   17/65 (26.15%)   11/63 (17.46%) 
Nausea * 1     
# participants affected / at risk   8/65 (12.31%)   8/63 (12.70%) 
Vomiting * 1     
# participants affected / at risk   7/65 (10.77%)   7/63 (11.11%) 
Constipation * 1     
# participants affected / at risk   4/65 (6.15%)   7/63 (11.11%) 
Abdominal pain upper * 1     
# participants affected / at risk   8/65 (12.31%)   2/63 (3.17%) 
Abdominal pain * 1     
# participants affected / at risk   3/65 (4.62%)   5/63 (7.94%) 
General disorders     
Oedema peripheral * 1     
# participants affected / at risk   4/65 (6.15%)   8/63 (12.70%) 
Fatigue * 1     
# participants affected / at risk   0/65 (0.00%)   6/63 (9.52%) 
Infections and infestations     
Urinary tract infection * 1     
# participants affected / at risk   14/65 (21.54%)   8/63 (12.70%) 
Influenza * 1     
# participants affected / at risk   10/65 (15.38%)   9/63 (14.29%) 
Nasopharyngitis * 1     
# participants affected / at risk   9/65 (13.85%)   8/63 (12.70%) 
Pharyngitis * 1     
# participants affected / at risk   4/65 (6.15%)   5/63 (7.94%) 
Upper respiratory tract infection * 1     
# participants affected / at risk   4/65 (6.15%)   3/63 (4.76%) 
Vaginal infection * 1     
# participants affected / at risk   4/65 (6.15%)   1/63 (1.59%) 
Injury, poisoning and procedural complications     
Thermal burn * 1     
# participants affected / at risk   4/65 (6.15%)   5/63 (7.94%) 
Investigations     
Weight decreased * 1     
# participants affected / at risk   3/65 (4.62%)   5/63 (7.94%) 
Musculoskeletal and connective tissue disorders     
Pain in extremity * 1     
# participants affected / at risk   11/65 (16.92%)   6/63 (9.52%) 
Back pain * 1     
# participants affected / at risk   5/65 (7.69%)   4/63 (6.35%) 
Muscle spasms * 1     
# participants affected / at risk   2/65 (3.08%)   7/63 (11.11%) 
Myalgia * 1     
# participants affected / at risk   5/65 (7.69%)   2/63 (3.17%) 
Nervous system disorders     
Headache * 1     
# participants affected / at risk   10/65 (15.38%)   12/63 (19.05%) 
Neuralgia * 1     
# participants affected / at risk   2/65 (3.08%)   12/63 (19.05%) 
Paraesthesia * 1     
# participants affected / at risk   3/65 (4.62%)   10/63 (15.87%) 
Dizziness * 1     
# participants affected / at risk   2/65 (3.08%)   4/63 (6.35%) 
Hypoaesthesia * 1     
# participants affected / at risk   1/65 (1.54%)   4/63 (6.35%) 
Psychiatric disorders     
Anxiety * 1     
# participants affected / at risk   4/65 (6.15%)   3/63 (4.76%) 
Depression * 1     
# participants affected / at risk   4/65 (6.15%)   3/63 (4.76%) 
Reproductive system and breast disorders     
Erectile dysfunction * 1     
# participants affected / at risk   4/65 (6.15%)   4/63 (6.35%) 
Respiratory, thoracic and mediastinal disorders     
Pharyngolaryngeal pain * 1     
# participants affected / at risk   2/65 (3.08%)   7/63 (11.11%) 
* Events were collected by non-systematic assessment
1 Term from vocabulary, MedDRA 10.0



  Limitations and Caveats
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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.


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