Trial · NCT00409175
Safety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis
NCT00409175, 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. Safety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis. Trials, record NCT00409175. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00409175
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis
- NCT number
- NCT00409175
- Status
- Completed
- Phases
- Phase 2, Phase 3
- Enrollment
- 128 participants, actual
- Lead sponsor
- Pfizer
- Conditions
- Familial Amyloid Polyneuropathy
- Interventions
- Fx-1006A, Placebo
- First posted
- 2006-12-08
- Last updated
- 2012-12-17
- Results first posted
- 2012-12-17
- Limitations and caveats
- Instead of the intended endpoint 'heat pain and cooling threshold', results of 'summated 3 score for small nerve fiber function' were reported.
Source ClinicalTrials.gov
Registered outcomes10
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 18 | Month 18 | Response to treatment was 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 scored on 0(normal) to 4(paralysis) scale, higher score=greater weakness. Components of reflexes, sensation scored 0=normal, 1=decreased, or 2=absent. Total NIS-LL score range 0-88, higher score=greater impairment. |
| Primary | Change From Baseline in Norfolk Quality of Life- Diabetic Neuropathy (QOL-DN) Total Quality of Life (TQOL) Score at Month 18 | Baseline, Month 18 | 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 symptom 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, 12 and 18 | Baseline, Month 6, 12, 18 | 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 0(normal) to 4(paralysis) scale, 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 | Percentage of Participants With Response to Treatment as Measured by Neuropathy Impairment Score - Lower Limb (NIS-LL) at Month 6 and 12 | Month 6, 12 | Response to treatment was indicated by either improvement (decrease from baseline) or stabilization (change from baseline of 0 to \<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 scored on 0 (normal) to 4 (paralysis) scale, higher score=greater weakness. Components of reflexes, sensation scored 0=normal, 1=decreased, or 2=absent. Total NIS-LL score range 0-88, higher score=greater impairment. |
| Secondary | Change From Baseline in Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Total Quality of Life (TQOL) Score at Month 6 and 12 | Baseline, Month 6, 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 symptom 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 Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Domain Scores at Month 6, 12 and 18 | Baseline, Month 6, 12, 18 | Norfolk QOL-DN:35-item participant-rated questionnaire to assess impact of DN on QOL; Item 1-7:scored as 1=symptom present, 0=symptom 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, 12 and 18 | Baseline, Month 6, 12, 18 | 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, 12 and 18 | Baseline, Month 6, 12, 18 | 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, 12 and 18 | Baseline, Month 6, 12, 18 | BMI was calculated by weight divided by height squared. mBMI was calculated by multiplying BMI by serum albumin levels to compensate for edema formation associated with malnutrition. A progressive decline in mBMI indicated worsening of disease severity. |
| Secondary | Percentage of Participants With Stabilized Transthyretin (TTR) Tetramer | Week 8, Month 6, 12, 18 | 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. |
Source ClinicalTrials.gov
Outcomes10
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 18 | Month 18 | ITTset:randomized participants received atleast(\>=)1 dose of study drug, had \>=1 post-baseline efficacy assessment for NIS-LL,Norfolk Quality of Life-Diabetic Neuropathy(QOL-DN) or discontinued study due to death/liver transplant(LT).Last-observation-carried-forward(LOCF) used;participant who discontinued due to death/LT was set non-responder. | 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 18 | Baseline, Month 18 | ITT population included all randomized participants who received at least 1 dose of study medication and had at least 1 post-baseline efficacy assessment for NIS-LL and Norfolk QOL-DN or discontinued study due to death or LT. LOCF method was used. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in Neuropathy Impairment Score- Lower Limb (NIS-LL) Score at Month 6, 12 and 18 | Baseline, Month 6, 12, 18 | ITT population included all randomized participants who received at least 1 dose of study medication and had at least 1 post-baseline efficacy assessment for NIS-LL and Norfolk QOL-DN or discontinued study due to death/LT. 'n' = those participants who were evaluable for this measure at given time point for each group respectively. | units on a scale | MEAN |
| SECONDARY | Percentage of Participants With Response to Treatment as Measured by Neuropathy Impairment Score - Lower Limb (NIS-LL) at Month 6 and 12 | Month 6, 12 | ITT population included all randomized participants who received at least 1 dose of study medication and had at least 1 post-baseline efficacy assessment for NIS-LL and Norfolk QOL-DN or discontinued study due to death/LT. LOCF method was used; participant who discontinued due to death/LT was set non-responder. | percentage of participants | NUMBER |
| SECONDARY | Change From Baseline in Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Total Quality of Life (TQOL) Score at Month 6 and 12 | Baseline, Month 6, 12 | ITT population included all randomized participants who received at least 1 dose of study medication and had at least 1 post-baseline efficacy assessment for NIS-LL and Norfolk QOL-DN or discontinued study due to death/LT. 'n' = those participants who were evaluable for this measure at given time point for each group respectively. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Domain Scores at Month 6, 12 and 18 | Baseline, Month 6, 12, 18 | ITT population included all randomized participants who received at least 1 dose of study medication and had at least 1 post-baseline efficacy assessment for NIS-LL and Norfolk QOL-DN or discontinued study due to death or LT. 'n' = those participants who were evaluable for this measure at given time points for each group respectively. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in Summated 7 Score for Large Nerve Fiber Function at Month 6, 12 and 18 | Baseline, Month 6, 12, 18 | ITT population included all randomized participants who received at least 1 dose of study medication and had at least 1 post-baseline efficacy assessment for NIS-LL and Norfolk QOL-DN or discontinued study due to death/LT. 'n' = those participants who were evaluable for this measure at given time points for each group respectively. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in Summated 3 Score for Small Nerve Fiber Function at Month 6, 12 and 18 | Baseline, Month 6, 12, 18 | ITT population included all randomized participants who received at least 1 dose of study medication and had at least 1 post-baseline efficacy assessment for NIS-LL and Norfolk QOL-DN or discontinued study due to death/LT. 'n' = those participants who were evaluable for this measure at given time points for each group respectively. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in Modified Body Mass Index (mBMI) at Month 6, 12 and 18 | Baseline, Month 6, 12, 18 | ITT population included all randomized participants who received at least 1 dose of study medication and had at least 1 post-baseline efficacy assessment for NIS-LL and Norfolk QOL-DN or discontinued study due to death/LT. 'n' = those participants who were evaluable for this measure at given time points for each group respectively. | (kilogram/square meter)*(gram/liter) | MEAN |
| SECONDARY | Percentage of Participants With Stabilized Transthyretin (TTR) Tetramer | Week 8, Month 6, 12, 18 | 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. | percentage of participants | NUMBER |
Source ClinicalTrials.gov
Results by group96
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 18 | Tafamidis | 45.3 | 95% Confidence Interval 33.1 to 57.5 | percentage of participants | |
| Percentage of Participants With Response to Treatment as Measured by Neuropathy Impairment Score - Lower Limb (NIS-LL) at Month 18 | Placebo | 29.5 | 95% Confidence Interval 18.1 to 41.0 | percentage of participants | |
| Change From Baseline in Norfolk Quality of Life- Diabetic Neuropathy (QOL-DN) Total Quality of Life (TQOL) Score at Month 18 | Tafamidis | Baseline | 27.3 | Standard Deviation 24.2 | units on a scale |
| Change From Baseline in Norfolk Quality of Life- Diabetic Neuropathy (QOL-DN) Total Quality of Life (TQOL) Score at Month 18 | Placebo | Baseline | 30.8 | Standard Deviation 26.7 | units on a scale |
| Change From Baseline in Norfolk Quality of Life- Diabetic Neuropathy (QOL-DN) Total Quality of Life (TQOL) Score at Month 18 | Tafamidis | Change at Month 18 | 2.4 | Standard Deviation 14.6 | units on a scale |
| Change From Baseline in Norfolk Quality of Life- Diabetic Neuropathy (QOL-DN) Total Quality of Life (TQOL) Score at Month 18 | Placebo | Change at Month 18 | 6.9 | Standard Deviation 22.9 | units on a scale |
| Change From Baseline in Neuropathy Impairment Score- Lower Limb (NIS-LL) Score at Month 6, 12 and 18 | Tafamidis | Baseline (n=64, 61) | 8.359 | Standard Deviation 11.399 | units on a scale |
| Change From Baseline in Neuropathy Impairment Score- Lower Limb (NIS-LL) Score at Month 6, 12 and 18 | Placebo | Baseline (n=64, 61) | 11.445 | Standard Deviation 13.544 | units on a scale |
| Change From Baseline in Neuropathy Impairment Score- Lower Limb (NIS-LL) Score at Month 6, 12 and 18 | Tafamidis | Change at Month 6 (n=60, 57) | 1.260 | Standard Deviation 3.007 | units on a scale |
| Change From Baseline in Neuropathy Impairment Score- Lower Limb (NIS-LL) Score at Month 6, 12 and 18 | Placebo | Change at Month 6 (n=60, 57) | 2.075 | Standard Deviation 6.407 | units on a scale |
1 to 10 of 96Source ClinicalTrials.gov
See all 96 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Tafamidis | 65 | |
| Overall Study | STARTED | Placebo | 63 | |
| Overall Study | COMPLETED | Tafamidis | 47 | |
| Overall Study | COMPLETED | Placebo | 44 | |
| Overall Study | NOT COMPLETED | Tafamidis | 18 | |
| Overall Study | NOT COMPLETED | Placebo | 19 |
Source ClinicalTrials.gov
Adverse events108
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 | Anaemia | Blood and lymphatic system disorders | Tafamidis | 0 | 65 | |
| other | Anaemia | Blood and lymphatic system disorders | Placebo | 4 | 63 | |
| other | Atrioventricular block first degree | Cardiac disorders | Tafamidis | 2 | 65 | |
| other | Atrioventricular block first degree | Cardiac disorders | Placebo | 6 | 63 | |
| other | Vertigo | Ear and labyrinth disorders | Tafamidis | 3 | 65 | |
| other | Vertigo | Ear and labyrinth disorders | Placebo | 4 | 63 | |
| other | Lacrimation decreased | Eye disorders | Tafamidis | 6 | 65 | |
| other | Lacrimation decreased | Eye disorders | Placebo | 7 | 63 | |
| other | Punctate keratitis | Eye disorders | Tafamidis | 5 | 65 | |
| other | Punctate keratitis | Eye disorders | Placebo | 3 | 63 |
1 to 10 of 108Source ClinicalTrials.gov
See all 108 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Tafamidis | ||
| Total, all-cause mortality | Placebo | ||
| Total, other adverse events | Tafamidis | 57 | 65 |
| Total, other adverse events | Placebo | 56 | 63 |
| Total, serious adverse events | Tafamidis | 6 | 65 |
| Total, serious adverse events | Placebo | 5 | 63 |
Source ClinicalTrials.gov
Papers6
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 comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| Tafamidis vs Placebo: serious adverse events |
Source 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.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT00409175
GET https://conductscience.org/api/trials/v1/NCT00409175/results?page=2