Trial · NCT01435655
The Effect Of Tafamidis For The Transthyretin Amyloid Polyneuropathy Patients With V30M Or Non-V30M Transthyretin
NCT01435655, Phase 3, Completed, Pfizer; results first posted 2015-09-09
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. The Effect Of Tafamidis For The Transthyretin Amyloid Polyneuropathy Patients With V30M Or Non-V30M Transthyretin. Trials, record NCT01435655. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01435655
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- The Effect Of Tafamidis For The Transthyretin Amyloid Polyneuropathy Patients With V30M Or Non-V30M Transthyretin
- NCT number
- NCT01435655
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 10 participants, actual
- Lead sponsor
- Pfizer
- Conditions
- Transthyretin Familial Amyloid Polyneuropathy
- Interventions
- tafamidis
- First posted
- 2011-09-16
- Last updated
- 2015-09-09
- Results first posted
- 2015-09-09
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Transthyretin (TTR) Stabilization at Week 8 Compared With Baseline as Measured by a Validated Immunoturbidimetric Assay | 8 weeks | TTR tetramer level for each plasma sample was assessed using a validated immunoturbidimetric assay before and after urea denaturation. The Fraction of Initial (FOI) tetramer concentration is the ratio of the measured TTR tetramer concentration after denaturation to the measured TTR tetramer average 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. A patient who has the "TTR stabilization" is defined as the patient whose percent stabilization is equal to or more than 32%. |
| Secondary | Change From Baseline in Neuropathy Impairment Score (NIS); NIS (Total), NIS-LL (Lower Limb) and NIS-UL (Upper Limb) at Week 26, Week 52 and Week 78 | Baseline, Week 26, Week 52, Week 78 | The NIS provides a total body single score of neuropathic deficits (score range: 0-122, higher score = more deficit), comprising subset scores for cranial nerves, muscle weakness, reflexes, and sensation (based on mean of 2 scores in 1 week period; each item scored separately for left and right). The NIS-LL is a subscale that provides a score for the lower limbs functions (muscle weakness, reflexes and sensation in great toe) and has a score range of 0-44 (higher score = more deficit). The NIS-UL is a subscale that provides a score for the upper body functions (muscle weakness \[including cranial nerves\], reflexes and sensation in finger) and has a score range of 0-78 (higher score = more deficit). The components for cranial nerves and muscle weakness are scored from 0 (Normal) to 4 (Paralysis), and those for reflexes and sensation from 0 (Normal) to 2 (Absent). For all items, higher scores indicate greater impairment. |
| Secondary | Change From Baseline in Scores of the Total Quality of Life (TQOL) and 5 Domains as Measured by the Norfolk QOL - Diabetic Neuropathy (Norfolk QOL-DN) at Week 26, Week 52 and Week 78. | Baseline, Week 26, Week 52, Week 78 | Norfolk QOL-DN is a 35-item participant-rated questionnaire. It consists of 5 domains: Physical Functioning/Large Fiber \[score range: -4 - 56\] , Activities of Daily Living (ADL) \[0 - 20\], Symptoms \[0 - 32\], Small Fiber \[0 - 16\] and Autonomic \[0 - 12\]. Total of quality of life (TQOL) score is the sum of all five domains with a range of -4 to 136 (Pfizer Data Standards). Higher scores on each item of the Norfolk QOL-DN TQOL indicate worse quality of life. |
| Secondary | Change From Baseline in Summated 7 Nerve Tests Normal Deviate Score (∑ 7 NTs Nds) as Measured by Nerve Conduction Studies (NCS), Vibration Detection Threshold (VDT) and Heart Rate Response to Deep Breathing (HRDB) at Week 26, Week 52, and Week 78 | Baseline, Week 26, Week 52, Week 78 | The Σ7 NTs nds measures primarily large-fiber function. It is a composite score derived from five 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 VDT obtained in great toes by Quantitative Sensory Testing (QST), and HRDB value. It is defined as 7 times the mean of non-missing values of, the five normal deviates of NCS, HRDB, and average normal deviate for VDT of toes. Score was determined through reference to normal values for age, sex, height and abnormalities scored. Total score range is approximately -26 to 26, where higher score=worse nerve function. |
| Secondary | Change From Baseline in Summated 3 Nerve Tests Small Fiber Normal Deviate Score (∑ 3 NTSF Nds) as Measured by Cooling and Heat Pain Thresholds by QST and HRDB at Week 26, Week 52 and Week 78 | Baseline, Week 26, Week 52, Week 78 | The Σ3 NTSF nds measures small-fiber function. It is a composite score defined as 3 times the mean of non-missing values of normal deviates of cooling threshold for lower limbs, heat pain intermediate response for lower limbs, and HRDB. The total score range is approximately -11.2 to 11.2, with a higher score demonstrating worse nerve function. |
| Secondary | Change From Baseline in Modified Body Mass Index (mBMI) at Week 8, Week 26, Week 52 and End of Study | Baseline, Week 8, Week 26, Week 52, End of Study | The mBMI was calculated by multiplying the BMI (the weight in kilograms divided by the square of the height in meters) by serum albumin level (gram/liter). Change in mBMI was calculated as the mBMI at the given week minus the Baseline mBMI. |
| Secondary | Change From Baseline in Ambulatory Status at Week 26, Week 52 and Week 78 | Baseline, Week 26, Week 52, Week 78 | Ambulatory status was evaluated using walking ability scale in polyneuropathy disability score. The ambulatory status was evaluated as: 0=Good, 1=Sensory disturbances in the feet but able to walk without difficulty, 2=Some difficulties with walking but can walk without aid, 3a=Able to walk with 1 stick or crutch, 3b=Able to walk with 2 sticks or crutches, 4=Not ambulatory, confined to a wheelchair or bedridden. |
| Secondary | Number of Participants With Transthyretin (TTR) Stabilization at Week 26, Week 52, and Week 78 Compared With Baseline as Measured by a Validated Immunoturbidimetric Assay | Baseline, Week 26, Week 52, Week 78 | TTR tetramer was assessed using a validated immunoturbidimetric assay. The TTR tetramer level for each plasma sample was measured before and after urea denaturation. The Fraction of Initial (FOI) tetramer concentration 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. A patient who has the "TTR stabilization" is defined as the patient whose percent stabilization is equal to or more than 32%. |
| Other | Plasma Concentration of Tafamidis at Week 8, Week 26, Week 52 and Week 78 | Week 8, Week 26, Week 52, Week 78 | Mean plasma concentration of tafamidis at 3 hours after administration |
Source ClinicalTrials.gov
Outcomes9
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Transthyretin (TTR) Stabilization at Week 8 Compared With Baseline as Measured by a Validated Immunoturbidimetric Assay | 8 weeks | Full Analysis Set (FAS) included all participants who received at least one dose of the study drug. | Participants | NUMBER |
| SECONDARY | Change From Baseline in Neuropathy Impairment Score (NIS); NIS (Total), NIS-LL (Lower Limb) and NIS-UL (Upper Limb) at Week 26, Week 52 and Week 78 | Baseline, Week 26, Week 52, Week 78 | Full Analysis Set (FAS) included all participants who received at least one dose of the study drug. | Units on a scale | MEAN |
| SECONDARY | Change From Baseline in Scores of the Total Quality of Life (TQOL) and 5 Domains as Measured by the Norfolk QOL - Diabetic Neuropathy (Norfolk QOL-DN) at Week 26, Week 52 and Week 78. | Baseline, Week 26, Week 52, Week 78 | Full Analysis Set (FAS) included all participants who received at least one dose of the study drug. | Units on a scale | MEAN |
| SECONDARY | Change From Baseline in Summated 7 Nerve Tests Normal Deviate Score (∑ 7 NTs Nds) as Measured by Nerve Conduction Studies (NCS), Vibration Detection Threshold (VDT) and Heart Rate Response to Deep Breathing (HRDB) at Week 26, Week 52, and Week 78 | Baseline, Week 26, Week 52, Week 78 | Full Analysis Set (FAS) included all participants who received at least one dose of the study drug. | Units on a scale | MEAN |
| SECONDARY | Change From Baseline in Summated 3 Nerve Tests Small Fiber Normal Deviate Score (∑ 3 NTSF Nds) as Measured by Cooling and Heat Pain Thresholds by QST and HRDB at Week 26, Week 52 and Week 78 | Baseline, Week 26, Week 52, Week 78 | Full Analysis Set (FAS) included all participants who received at least one dose of the study drug. | Units on a scale | MEAN |
| SECONDARY | Change From Baseline in Modified Body Mass Index (mBMI) at Week 8, Week 26, Week 52 and End of Study | Baseline, Week 8, Week 26, Week 52, End of Study | Full Analysis Set (FAS) included all participants who received at least one dose of the study drug. | (kilogram/square meter)*(gram/liter) | MEAN |
| SECONDARY | Change From Baseline in Ambulatory Status at Week 26, Week 52 and Week 78 | Baseline, Week 26, Week 52, Week 78 | Full Analysis Set (FAS) included all participants who received at least one dose of the study drug. | Participants | NUMBER |
| SECONDARY | Number of Participants With Transthyretin (TTR) Stabilization at Week 26, Week 52, and Week 78 Compared With Baseline as Measured by a Validated Immunoturbidimetric Assay | Baseline, Week 26, Week 52, Week 78 | Full Analysis Set (FAS) included all participants who received at least one dose of the study drug. | Participants | NUMBER |
| OTHER_PRE_SPECIFIED | Plasma Concentration of Tafamidis at Week 8, Week 26, Week 52 and Week 78 | Week 8, Week 26, Week 52, Week 78 | The PK Analysis Set included all participants treated who had at least 1 quantifiable plasma tafamidis concentration. | ng/mL | MEAN |
Source ClinicalTrials.gov
Results by group81
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Transthyretin (TTR) Stabilization at Week 8 Compared With Baseline as Measured by a Validated Immunoturbidimetric Assay | Tafamidis 20 mg | 10 | Participants | ||
| Change From Baseline in Neuropathy Impairment Score (NIS); NIS (Total), NIS-LL (Lower Limb) and NIS-UL (Upper Limb) at Week 26, Week 52 and Week 78 | Tafamidis 20 mg | NIS-Total: Baseline (n=10) | 31.03 | Standard Deviation 26.260 | Units on a scale |
| Change From Baseline in Neuropathy Impairment Score (NIS); NIS (Total), NIS-LL (Lower Limb) and NIS-UL (Upper Limb) at Week 26, Week 52 and Week 78 | Tafamidis 20 mg | NIS-Total: Change at Week 26 (n=10) | 4.37 | Standard Deviation 9.408 | Units on a scale |
| Change From Baseline in Neuropathy Impairment Score (NIS); NIS (Total), NIS-LL (Lower Limb) and NIS-UL (Upper Limb) at Week 26, Week 52 and Week 78 | Tafamidis 20 mg | NIS-Total: Change at Week 52 (n=10) | 8.30 | Standard Deviation 8.326 | Units on a scale |
| Change From Baseline in Neuropathy Impairment Score (NIS); NIS (Total), NIS-LL (Lower Limb) and NIS-UL (Upper Limb) at Week 26, Week 52 and Week 78 | Tafamidis 20 mg | NIS-Total: Change at Week 78 (n=8) | 9.90 | Standard Deviation 12.584 | Units on a scale |
| Change From Baseline in Neuropathy Impairment Score (NIS); NIS (Total), NIS-LL (Lower Limb) and NIS-UL (Upper Limb) at Week 26, Week 52 and Week 78 | Tafamidis 20 mg | NIS-LL: Baseline (n=10) | 16.99 | Standard Deviation 13.143 | Units on a scale |
| Change From Baseline in Neuropathy Impairment Score (NIS); NIS (Total), NIS-LL (Lower Limb) and NIS-UL (Upper Limb) at Week 26, Week 52 and Week 78 | Tafamidis 20 mg | NIS-LL: Change at Week 26 (n=10) | 2.06 | Standard Deviation 5.565 | Units on a scale |
| Change From Baseline in Neuropathy Impairment Score (NIS); NIS (Total), NIS-LL (Lower Limb) and NIS-UL (Upper Limb) at Week 26, Week 52 and Week 78 | Tafamidis 20 mg | NIS-LL: Change at Week 52 (n=10) | 3.62 | Standard Deviation 4.374 | Units on a scale |
| Change From Baseline in Neuropathy Impairment Score (NIS); NIS (Total), NIS-LL (Lower Limb) and NIS-UL (Upper Limb) at Week 26, Week 52 and Week 78 | Tafamidis 20 mg | NIS-LL: Change at Week 78 (n=8) | 3.30 | Standard Deviation 4.739 | Units on a scale |
| Change From Baseline in Neuropathy Impairment Score (NIS); NIS (Total), NIS-LL (Lower Limb) and NIS-UL (Upper Limb) at Week 26, Week 52 and Week 78 | Tafamidis 20 mg | NIS-UL: Baseline (n=10) | 14.03 | Standard Deviation 13.716 | Units on a scale |
1 to 10 of 81Source ClinicalTrials.gov
See all 81 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Tafamidis 20 mg | 10 | |
| Overall Study | COMPLETED | Tafamidis 20 mg | 7 | |
| Overall Study | NOT COMPLETED | Tafamidis 20 mg | 3 |
Source ClinicalTrials.gov
Adverse events66
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 | Iron deficiency anaemia | Blood and lymphatic system disorders | Tafamidis 20 mg | 1 | 10 | |
| other | Atrioventricular block second degree | Cardiac disorders | Tafamidis 20 mg | 1 | 10 | |
| other | Palpitations | Cardiac disorders | Tafamidis 20 mg | 1 | 10 | |
| other | Ear discomfort | Ear and labyrinth disorders | Tafamidis 20 mg | 1 | 10 | |
| other | Meniere's disease | Ear and labyrinth disorders | Tafamidis 20 mg | 1 | 10 | |
| other | Cataract | Eye disorders | Tafamidis 20 mg | 2 | 10 | |
| other | Conjunctivitis | Eye disorders | Tafamidis 20 mg | 1 | 10 | |
| other | Dry eye | Eye disorders | Tafamidis 20 mg | 1 | 10 | |
| other | Eye discharge | Eye disorders | Tafamidis 20 mg | 1 | 10 | |
| other | Vitreous opacities | Eye disorders | Tafamidis 20 mg | 2 | 10 |
1 to 10 of 66Source ClinicalTrials.gov
See all 66 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Tafamidis 20 mg | ||
| Total, other adverse events | Tafamidis 20 mg | 10 | 10 |
| Total, serious adverse events | Tafamidis 20 mg | 7 | 10 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 31353964 | DERIVED |
Source PubMed
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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/NCT01435655
GET https://conductscience.org/api/trials/v1/NCT01435655/results?page=2