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; 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

Hide 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

Hide Baseline Characteristics

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

Show All 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 ]

Show Outcome Measure 10

Serious Adverse Events

Show Serious Adverse Events

Other Adverse Events

Show Other Adverse Events

More Information

Hide 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:

	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.

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

To Top