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

History of Changes

Results First Received: November 16, 2012

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

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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
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]

125

Age, Customized ^[1]
[Units: Participants]

Less than or equal to 65 years

117

Greater than 65 years

^[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

Male

^[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 ]

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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 ]

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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 ]

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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 ]

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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 ]

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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 ]

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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 ]

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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 ]

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9. Secondary:

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

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10. Secondary:

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

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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

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Other Adverse Events

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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.

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:

	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.
	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.
	Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed. Restriction Description: Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.

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):

Waddington Cruz M, Amass L, Keohane D, Schwartz J, Li H, Gundapaneni B. Early intervention with tafamidis provides long-term (5.5-year) delay of neurologic progression in transthyretin hereditary amyloid polyneuropathy. Amyloid. 2016 Sep;23(3):178-183. Epub 2016 Aug 5.

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

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