Trial · NCT00791492
An Extension of Study Fx-005 Evaluating Long-Term Safety And Clinical Outcomes Of Fx-1006A In Patients With Transthyretin Amyloid Polyneuropathy
NCT00791492, Phase 2, Phase 3, Completed, Pfizer; results first posted 2012-12-17
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. An Extension of Study Fx-005 Evaluating Long-Term Safety And Clinical Outcomes Of Fx-1006A In Patients With Transthyretin Amyloid Polyneuropathy. Trials, record NCT00791492. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00791492
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- An Extension of Study Fx-005 Evaluating Long-Term Safety And Clinical Outcomes Of Fx-1006A In Patients With Transthyretin Amyloid Polyneuropathy
- NCT number
- NCT00791492
- Status
- Completed
- Phases
- Phase 2, Phase 3
- Enrollment
- 86 participants, actual
- Lead sponsor
- Pfizer
- Conditions
- Familial Amyloid Polyneuropathy, ATTR-PN
- Interventions
- Fx-1006A
- First posted
- 2008-11-14
- Last updated
- 2012-12-17
- Results first posted
- 2012-12-17
- Limitations and caveats
Instead of intended endpoint 'heat pain and cooling threshold', results of 'summated 3 score for small nerve fiber function' were reported. Designation of outcomes as primary and secondary was based on study team input as study did not specify this.
Source ClinicalTrials.gov
Registered outcomes19
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants With Response to Treatment as Measured by Neuropathy Impairment Score - Lower Limb (NIS-LL) at Month 6 | Month 6 | Response to treatment indicated by either improvement(decrease from baseline) or stabilization(change from baseline of 0 to less than\[\<\] 2) in NIS-LL score,based on mean of 2 scores in 1 week period.NIS-LL assessed muscle weakness,reflexes,sensation.Each item scored separately for left,right limbs.Components of muscle weakness:0(normal)-4(paralysis),higher score=more weakness;reflexes,sensation:0=normal,1=decreased,or 2=absent.Total NIS-LL score range 0-88,higher score=more impairment. For tafamidis-tafamidis group, NIS-LL baseline value of previous study FX-005(NCT00409175) used as reference. |
| Primary | Percentage of Participants With Response to Treatment as Measured by Neuropathy Impairment Score - Lower Limb (NIS-LL) at Month 12 | Month 12 | Response to treatment indicated by either improvement(decrease from baseline) or stabilization(change from baseline of 0 to less than\[\<\] 2) in NIS-LL score,based on mean of 2 scores in 1 week period.NIS-LL assessed muscle weakness,reflexes,sensation.Each item scored separately for left,right limbs.Components of muscle weakness:0(normal)-4(paralysis),higher score=more weakness;reflexes,sensation:0=normal,1=decreased,or 2=absent.Total NIS-LL score range 0-88,higher score=more impairment. For tafamidis-tafamidis group, NIS-LL baseline value of previous study FX-005(NCT00409175) used as reference. |
| Primary | Change From Baseline in Norfolk Quality of Life- Diabetic Neuropathy (QOL-DN) Total Quality of Life (TQOL) Score at Month 6 | Baseline, Month 6 | Norfolk QOL-DN: 35-item participant-rated questionnaire used to assess impact of diabetic neuropathy on the quality of life of participants with diabetic neuropathy; Item 1 to 7: related to symptoms and presence of symptoms was assessed as 1 and absence was assessed as 0. Item 8-35: related to activities of daily living and scored on a 5-point Likert scale, where 0= no problem and 4= severe problem (except item 32, where -2= much better, 0=about the same, 2=much worse). TQOL= sum of all the items, total possible score range= -2 to 138, where higher score=worse quality of life. |
| Primary | Change From Baseline in Norfolk Quality of Life- Diabetic Neuropathy (QOL-DN) Total Quality of Life (TQOL) Score at Month 12 | Baseline, Month 12 | Norfolk QOL-DN: 35-item participant-rated questionnaire used to assess impact of diabetic neuropathy on the quality of life of participants with diabetic neuropathy; Item 1 to 7: related to symptoms and presence of symptoms was assessed as 1 and absence was assessed as 0. Item 8-35: related to activities of daily living and scored on a 5-point Likert scale, where 0= no problem and 4= severe problem (except item 32, where -2= much better, 0=about the same, 2=much worse). TQOL= sum of all the items, total possible score range= -2 to 138, where higher score=worse quality of life. |
| Secondary | Change From Baseline in Neuropathy Impairment Score - Lower Limb (NIS-LL) Score at Month 6 and 12 | Baseline, Month 6, 12 | 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 scale 0 (normal) to 4 (paralysis), 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. |
| Secondary | Change From Baseline in Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Domain Scores at Month 6 and 12 | Baseline, Month 6, 12 | Norfolk QOL-DN: 35-item participant-rated questionnaire to assess impact of DN on QOL; Item 1-7:scored as 1=symptoms present, 0=symptoms absent. Item 8-35:scored on 5-point Likert scale: 0=no problem,4=severe problem (except item 32: -2=much better, 0=about same, 2=much worse). Norfolk QOL-DN summarized in 5 domains(score range):physical functioning/large fiber neuropathy(-2 to 58), activities of daily living(ADLs) (0 to 20), symptoms(0 to 32), small fiber neuropathy(0 to 16), autonomic neuropathy(0 to 12); higher score=greater impairment,for each. Total score=-2 to138(higher score=worse QOL). |
| Secondary | Change From Baseline in Summated 7 Score for Large Nerve Fiber Function at Month 6 and 12 | Baseline, Month 6, 12 | Summated 7 score: composite score included five Nerve Conduction Studies (NCS) attributes (peroneal nerve distal motor latency, peroneal nerve compound muscle action potential, peroneal nerve motor conduction velocity, tibial nerve distal motor latency, and sural nerve sensory nerve action potential amplitude) along with Vibration Detection Threshold (VDT) obtained in great toes, and Heart Rate Response to Deep Breathing (HRDB) value. Score was determined through reference to normal values for age, sex and height. Total score range= -26 to 26, where higher score=worse nerve function. |
| Secondary | Change From Baseline in Summated 3 Score for Small Nerve Fiber Function at Month 6 and 12 | Baseline, Month 6, 12 | Summated 3 Nerve Tests Small Fiber Normal Deviates Score (NTSFnds) included cooling threshold for the lower limbs, heat pain threshold for the lower limbs and HRDB. Total score range= -11.2 to 11.2, where higher score=worse nerve function. |
| Secondary | Change From Baseline in Modified Body Mass Index (mBMI) at Month 6 and 12 | Baseline, Month 6, 12 | BMI was calculated by weight divided by height squared. mBMI was calculated by multiplying BMI by serum albumin levels to compensate for edema formation. A progressive decline in mBMI indicated worsening of disease severity. |
| Secondary | Change From Baseline in Troponin I Concentration at Week 6, Month 3, 6 and 12 | Baseline, Week 6, Month 3, 6, 12 | Troponin I was biomarker of cardiac stress (myocardial necrosis and increased filling pressures/ left ventricular \[LV\] wall stress). |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 registered outcomesOutcomes19
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants With Response to Treatment as Measured by Neuropathy Impairment Score - Lower Limb (NIS-LL) at Month 6 | Month 6 | Intent-to-treat (ITT) population included all participants who received at least one dose of study medication and had no more than 2 months interruption between studies FX-005 and FX-006. 'N' (number of participants analyzed) signifies participants evaluable for this measure. | percentage of participants | NUMBER |
| PRIMARY | Percentage of Participants With Response to Treatment as Measured by Neuropathy Impairment Score - Lower Limb (NIS-LL) at Month 12 | Month 12 | ITT population included all participants who received at least one dose of study medication and had no more than 2 months interruption between studies FX-005 and FX-006. 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure. | percentage of participants | NUMBER |
| PRIMARY | Change From Baseline in Norfolk Quality of Life- Diabetic Neuropathy (QOL-DN) Total Quality of Life (TQOL) Score at Month 6 | Baseline, Month 6 | ITT population included all participants who received at least one dose of study medication and had no more than 2 months interruption between studies FX-005 and FX-006. 'N' (number of participants analyzed) signifies participants evaluable for this measure. | units on a scale | MEAN |
| PRIMARY | Change From Baseline in Norfolk Quality of Life- Diabetic Neuropathy (QOL-DN) Total Quality of Life (TQOL) Score at Month 12 | Baseline, Month 12 | ITT population included all participants who received at least one dose of study medication and had no more than 2 months interruption between studies FX-005 and FX-006. 'N' (number of participants analyzed) signifies participants evaluable for this measure. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in Neuropathy Impairment Score - Lower Limb (NIS-LL) Score at Month 6 and 12 | Baseline, Month 6, 12 | ITT population included all participants who received at least one dose of study medication and had no more than 2 months interruption between studies FX-005 and FX-006. 'N' (number of participants analyzed) signifies participants evaluable for this measure. 'n' signifies participants for this measure at specified time point for each arm group. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Domain Scores at Month 6 and 12 | Baseline, Month 6, 12 | ITT population included all participants who received at least one dose of study medication and had no more than 2 months interruption between studies FX-005 and FX-006. 'N' (number of participants analyzed) signifies participants evaluable for this measure. 'n' signifies participants for this measure at specified time point for each arm group. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in Summated 7 Score for Large Nerve Fiber Function at Month 6 and 12 | Baseline, Month 6, 12 | ITT population included all participants who received at least one dose of study medication and had no more than 2 months interruption between studies FX-005 and FX-006. 'N' (number of participants analyzed) signifies participants evaluable for this measure. 'n' signifies participants for this measure at specified time point for each arm group. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in Summated 3 Score for Small Nerve Fiber Function at Month 6 and 12 | Baseline, Month 6, 12 | ITT population included all participants who received at least one dose of study medication and had no more than 2 months interruption between studies FX-005 and FX-006. 'N' (number of participants analyzed) signifies participants evaluable for this measure. 'n' signifies participants for this measure at specified time point for each arm group. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in Modified Body Mass Index (mBMI) at Month 6 and 12 | Baseline, Month 6, 12 | ITT population included all participants who received at least one dose of study medication and had no more than 2 months interruption between studies FX-005 and FX-006. 'N' (number of participants analyzed) signifies participants evaluable for this measure. 'n' signifies participants for this measure at specified time point for each arm group. | (kilogram/square meter)*(gram/liter) | MEAN |
| SECONDARY | Change From Baseline in Troponin I Concentration at Week 6, Month 3, 6 and 12 | Baseline, Week 6, Month 3, 6, 12 | Safety population included participants who received at least one dose of study medication. 'N' (number of participants analyzed) signifies participants evaluable for this measure. 'n' signifies participants evaluable for this measure at the specified time point for each arm group. | nanogram/milliliter (ng/mL) | MEDIAN |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 outcomesResults by group110
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants With Response to Treatment as Measured by Neuropathy Impairment Score - Lower Limb (NIS-LL) at Month 6 | Tafamidis-Tafamidis | 62.2 | 95% Confidence Interval 44.8 to 77.5 | percentage of participants | |
| Percentage of Participants With Response to Treatment as Measured by Neuropathy Impairment Score - Lower Limb (NIS-LL) at Month 6 | Placebo-Tafamidis | 68.8 | 95% Confidence Interval 50.0 to 83.9 | percentage of participants | |
| Percentage of Participants With Response to Treatment as Measured by Neuropathy Impairment Score - Lower Limb (NIS-LL) at Month 12 | Tafamidis-Tafamidis | 54.5 | 95% Confidence Interval 36.4 to 71.9 | percentage of participants | |
| Percentage of Participants With Response to Treatment as Measured by Neuropathy Impairment Score - Lower Limb (NIS-LL) at Month 12 | Placebo-Tafamidis | 60.0 | 95% Confidence Interval 40.6 to 77.3 | percentage of participants | |
| Change From Baseline in Norfolk Quality of Life- Diabetic Neuropathy (QOL-DN) Total Quality of Life (TQOL) Score at Month 6 | Tafamidis-Tafamidis | Baseline | 21.05 | Standard Deviation 21.88 | units on a scale |
| Change From Baseline in Norfolk Quality of Life- Diabetic Neuropathy (QOL-DN) Total Quality of Life (TQOL) Score at Month 6 | Placebo-Tafamidis | Baseline | 38.09 | Standard Deviation 31.89 | units on a scale |
| Change From Baseline in Norfolk Quality of Life- Diabetic Neuropathy (QOL-DN) Total Quality of Life (TQOL) Score at Month 6 | Tafamidis-Tafamidis | Change at Month 6 | 0.03 | Standard Deviation 8.83 | units on a scale |
| Change From Baseline in Norfolk Quality of Life- Diabetic Neuropathy (QOL-DN) Total Quality of Life (TQOL) Score at Month 6 | Placebo-Tafamidis | Change at Month 6 | -4.88 | Standard Deviation 15.29 | units on a scale |
| Change From Baseline in Norfolk Quality of Life- Diabetic Neuropathy (QOL-DN) Total Quality of Life (TQOL) Score at Month 12 | Tafamidis-Tafamidis | 2.25 | Standard Deviation 8.91 | units on a scale | |
| Change From Baseline in Norfolk Quality of Life- Diabetic Neuropathy (QOL-DN) Total Quality of Life (TQOL) Score at Month 12 | Placebo-Tafamidis | -2.33 | Standard Deviation 15.66 | units on a scale |
1 to 10 of 110Source ClinicalTrials.gov
See all 110 results by groupParticipant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Tafamidis-Tafamidis | 45 | |
| Overall Study | STARTED | Placebo-Tafamidis | 41 | |
| Overall Study | TREATED | Tafamidis-Tafamidis | 44 | |
| Overall Study | TREATED | Placebo-Tafamidis | 41 | |
| Overall Study | COMPLETED | Tafamidis-Tafamidis | 39 | |
| Overall Study | COMPLETED | Placebo-Tafamidis | 38 | |
| Overall Study | NOT COMPLETED | Tafamidis-Tafamidis | 6 | |
| Overall Study | NOT COMPLETED | Placebo-Tafamidis | 3 |
Source ClinicalTrials.gov
Adverse events50
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Punctate keratitis | Eye disorders | Tafamidis-Tafamidis | 3 | 44 | |
| other | Punctate keratitis | Eye disorders | Placebo-Tafamidis | 3 | 41 | |
| other | Dry eye | Eye disorders | Tafamidis-Tafamidis | 2 | 44 | |
| other | Dry eye | Eye disorders | Placebo-Tafamidis | 3 | 41 | |
| other | Diarrhoea | Gastrointestinal disorders | Tafamidis-Tafamidis | 4 | 44 | |
| other | Diarrhoea | Gastrointestinal disorders | Placebo-Tafamidis | 3 | 41 | |
| other | Vomiting | Gastrointestinal disorders | Tafamidis-Tafamidis | 2 | 44 | |
| other | Vomiting | Gastrointestinal disorders | Placebo-Tafamidis | 4 | 41 | |
| other | Urinary tract infection | Infections and infestations | Tafamidis-Tafamidis | 5 | 44 | |
| other | Urinary tract infection | Infections and infestations | Placebo-Tafamidis | 6 | 41 |
1 to 10 of 50Source ClinicalTrials.gov
See all 50 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Tafamidis-Tafamidis | ||
| Total, all-cause mortality | Placebo-Tafamidis | ||
| Total, other adverse events | Tafamidis-Tafamidis | 24 | 44 |
| Total, other adverse events | Placebo-Tafamidis | 24 | 41 |
| Total, serious adverse events | Tafamidis-Tafamidis | 5 | 44 |
| Total, serious adverse events | Placebo-Tafamidis | 4 | 41 |
Source ClinicalTrials.gov
Papers5
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons4
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Tafamidis-Tafamidis vs Placebo-Tafamidis: Number of Participants With Treatment-emergent Adverse Events Greater Than or Equal to Grade 3 | Trial comparisons |
| Tafamidis-Tafamidis vs Placebo-Tafamidis: Number of Participants With Treatment-emergent Serious Adverse Events (SAEs) | Trial comparisons |
| Tafamidis-Tafamidis vs Placebo-Tafamidis: Number of Participants Who Discontinued Due to Clinical or Laboratory Adverse Events | Trial comparisons |
| Tafamidis-Tafamidis vs Placebo-Tafamidis: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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