Trial · NCT04828993
The Effect Of Tafamidis Meglumine In Transthyretin Amyloid Polyneuropathy Patients
NCT04828993, Phase 4, Completed, Pfizer; results first posted 2024-03-12
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. The Effect Of Tafamidis Meglumine In Transthyretin Amyloid Polyneuropathy Patients. Trials, record NCT04828993. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04828993
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- The Effect Of Tafamidis Meglumine In Transthyretin Amyloid Polyneuropathy Patients
- NCT number
- NCT04828993
- Status
- Completed
- Phases
- Phase 4
- Enrollment
- 15 participants, actual
- Lead sponsor
- Pfizer
- Conditions
- Transthyretin Amyloid Polyneuropathy (ATTR-PN)
- Interventions
- tafamidis meglumine
- First posted
- 2021-04-02
- Last updated
- 2024-03-12
- Results first posted
- 2024-03-12
Source ClinicalTrials.gov
Registered outcomes14
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in Neuropathy Impairment Score-lower Limb (NIS-LL) Total Score at Week 72 | Baseline, Week 72 | NIS-LL: assess muscle weakness, reflexes, 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) scored on scale 0 (normal) to 4 (paralysis), higher score=greater weakness. Components of reflexes (quadriceps femoris, triceps surae); sensation (touch pressure, pin-prick, vibration, joint position) scored 0 = normal, 1 = decreased, or 2 = absent. Total possible NIS-LL score range 0-88, high score = more impairment. Components of muscle weakness are scored to eight levels: 0 = Normal, 1 = 25% Weak, 2 = 50% Weak, 3 = 75% Weak, 3.25 = Move against gravity, 3.5 = Movement, gravity eliminated, 3.75 = Muscle flicker, no movement, 4 = Paralysis. |
| Secondary | Change From Baseline in Neuropathy Impairment Score-lower Limb (NIS-LL) Total Score at Weeks 24, and 48 | Baseline, Week 24, Week 48 | NIS-LL: assess muscle weakness, reflexes, 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) scored on scale 0 (normal) to 4 (paralysis), higher score=greater weakness. Components of reflexes (quadriceps femoris, triceps surae); sensation (touch pressure, pin-prick, vibration, joint position) scored 0 = normal, 1 = decreased, or 2 = absent. Total possible NIS-LL score range 0-88, high score = more impairment. Components of muscle weakness are scored to eight levels: 0 = Normal, 1 = 25% Weak, 2 = 50% Weak, 3 = 75% Weak, 3.25 = Move against gravity, 3.5 = Movement, gravity eliminated, 3.75 = Muscle flicker, no movement, 4 = Paralysis. |
| Secondary | Change From Baseline in Total Quality of Life (TQOL) of Norfolk Quality of Life - Diabetic Neuropathy (Norfolk QOL-DN) at Weeks 24, 48, and 72 | Baseline, Week 24, Week 48, Week 72 | Norfolk QOL-DN: 35-item participant-rated questionnaire assess the impact of neuropathy on the quality of life of participants diagnosed with transthyretin amyloid (ATTR). Scoring is based on 35 questions that yield a TQOL as well as 5 subscale scores: activities of daily living, large fiber neuropathy/physical functioning, small fiber neuropathy, autonomic neuropathy, and symptoms. 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 5 Domains of Norfolk QOL-DN at Weeks 24, 48, and 72 | Baseline, Week 24, Week 48, Week 72 | Norfolk QOL-DN: 35-item participant-rated questionnaire assess the impact of neuropathy on the quality of life of participants diagnosed with transthyretin amyloid (ATTR). It is summarized in 5 domains: (1) Activities of daily living (score ranges from 0 to 20, where higher score=worse quality of life); (2) Large fiber neuropathy/physical functioning (score ranges from -2 to 58, where higher score=worse condition); (3) Small fiber neuropathy (score ranges from 0 to 16, where higher score=worse condition); (4) Autonomic neuropathy (score ranges from 0 to 12, where higher score=worse condition) and (5) Symptoms (score ranges from 0 to 32, where higher score=less symptoms of disease). Total possible score range= -2 to 138, where higher score=worse quality of life. |
| Secondary | Change From Baseline in Modified Body Mass Index (mBMI) at Weeks 4, 8, 12, 24, 36, 48, and 72 | Baseline, Weeks 4, 8, 12, 24, 36, 48, and 72 | BMI is calculated by weight divided by height squared and measured as kilogram per square meter (kg/m\^2). mBMI is calculated by multiplying BMI by serum albumin levels \[gram/liter (g/L)\]. mBMI is measured as kg/m\^2\*g/L. A progressive decline in mBMI indicates worsening of disease severity. |
| Secondary | Change From Baseline in Physical Component Summary and Mental Component Summary of 36-Item Short Form Survey (SF-36) at Weeks 24, 48, and 72 | Baseline, Weeks 24, 48, and 72 | The SF-36 is a participant administered scale assessing general quality of life. It consists of self-administered 36-item questionnaire that measured 8 health domains: physical function, role-physical, bodily pain, general health, vitality, social function, role-emotional, and mental health. These 8 domains are also summarized as physical and mental component scores. The score for each domain and component score is the mean of the individual question scores, which are scaled from 0 (minimum) to 100 (maximum), where high scores in each dimension and high overall scores indicate a better quality of life. |
| Secondary | Change From Baseline in EuroQoL 5 Dimensions 5 Levels (EQ-5D-5L) Index Score at Weeks 24, 48, and 72 | Baseline, Weeks 24, 48, and 72 | EQ-5D-5L: standardized participant (aged \>17 years) completed questionnaire consists of 2 components: a health state profile and an optional VAS. EQ-5D health state profile has 5 dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Each dimension has 5 levels: 1= no problems, 2= slight problems, 3= moderate problems, 4= severe problems, and 5= extreme problems. Responses to 5 dimensions comprise a health state/a single utility index value. E.g. if a participant responds "no problems" for each 5 dimensions, then health state was coded as "11111" with a predefined index value to it. Every health state (coded as combination of responses on each of 5 dimensions) has a unique predefined utility index value assigned to it, by EuroQol. Chinese value sets (with all possible health states) is used for adults in the study, range from -0.391 to 1. Higher (positive) scores = better health state. |
| Secondary | Number of Participants Reporting Treatment-Emergent Adverse Events (TEAEs) | Baseline up to Week 77 | An adverse event is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. Treatment emergent adverse event is defined as any adverse event started after the first dose. The TEAEs were collected until Week 77. |
| Secondary | Number of Participants With Categorical Vital Signs Data | Baseline up to Week 72 | Vital signs categorical criteria: 1) pulse rate \<40 beats per minute (bpm), or \>120 bpm; 2) standing diastolic blood pressure (BP) \<50 mmHg, or increase ≥20 mmHg, or decrease ≥20 mmHg; 3) standing systolic BP \<90 mmHg, or increase ≥30 mmHg, or decrease ≥30 mmHg; 4) supine diastolic BP \<50 mmHg, or increase ≥20 mmHg, or decrease ≥20 mmHg; 5) supine systolic BP \<90 mmHg, or increase ≥30 mmHg, or decrease ≥30 mmHg. |
| Secondary | Number of Participants With Categorical Electrocardiogram (ECG) Data | Baseline up to Week 72 | ECG categorical criteria: 1) ECG mean heart rate \<40 beats/minute, or \>120 beats/minute; 2) PR interval not otherwise specified ≥300 milliseconds (msec), or baseline \>200 msec and %increase ≥25%/ baseline ≤200 msec and %increase ≥50% (% change ≥25/50%); 3) QRS interval not otherwise specified ≥140 msec, or %change ≥50%; 4) QT interval not otherwise specified ≥500 msec; 5) corrected QT (QTc) interval not otherwise specified ≥450 and \<480 msec, or ≥480 and \<500 msec, or ≥500 msec; or change ≥30 and \<60 msec, or change ≥60 msec. |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 registered outcomesOutcomes14
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in Neuropathy Impairment Score-lower Limb (NIS-LL) Total Score at Week 72 | Baseline, Week 72 | All participants who took at least 1 dose of tafamidis meglumine soft gelatin capsule 20 mg. | Units on a scale | MEDIAN |
| SECONDARY | Change From Baseline in Neuropathy Impairment Score-lower Limb (NIS-LL) Total Score at Weeks 24, and 48 | Baseline, Week 24, Week 48 | All participants who took at least 1 dose of tafamidis meglumine soft gelatin capsule 20 mg. | Units on a scale | MEDIAN |
| SECONDARY | Change From Baseline in Total Quality of Life (TQOL) of Norfolk Quality of Life - Diabetic Neuropathy (Norfolk QOL-DN) at Weeks 24, 48, and 72 | Baseline, Week 24, Week 48, Week 72 | All participants who took at least 1 dose of tafamidis meglumine soft gelatin capsule 20 mg. | Units on a scale | MEDIAN |
| SECONDARY | Change From Baseline in 5 Domains of Norfolk QOL-DN at Weeks 24, 48, and 72 | Baseline, Week 24, Week 48, Week 72 | All participants who took at least 1 dose of tafamidis meglumine soft gelatin capsule 20 mg. | Units on a scale | MEDIAN |
| SECONDARY | Change From Baseline in Modified Body Mass Index (mBMI) at Weeks 4, 8, 12, 24, 36, 48, and 72 | Baseline, Weeks 4, 8, 12, 24, 36, 48, and 72 | All participants who took at least 1 dose of tafamidis meglumine soft gelatin capsule 20 mg. | kg/m^2*g/L | MEDIAN |
| SECONDARY | Change From Baseline in Physical Component Summary and Mental Component Summary of 36-Item Short Form Survey (SF-36) at Weeks 24, 48, and 72 | Baseline, Weeks 24, 48, and 72 | All participants who took at least 1 dose of tafamidis meglumine soft gelatin capsule 20 mg. | Units on a scale | MEDIAN |
| SECONDARY | Change From Baseline in EuroQoL 5 Dimensions 5 Levels (EQ-5D-5L) Index Score at Weeks 24, 48, and 72 | Baseline, Weeks 24, 48, and 72 | All participants who took at least 1 dose of tafamidis meglumine soft gelatin capsule 20 mg. | Units on a scale | MEDIAN |
| SECONDARY | Number of Participants Reporting Treatment-Emergent Adverse Events (TEAEs) | Baseline up to Week 77 | All participants who took at least 1 dose of tafamidis meglumine soft gelatin capsule 20 mg. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Categorical Vital Signs Data | Baseline up to Week 72 | All participants who took at least 1 dose of tafamidis meglumine soft gelatin capsule 20 mg. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Categorical Electrocardiogram (ECG) Data | Baseline up to Week 72 | All participants who took at least 1 dose of tafamidis meglumine soft gelatin capsule 20 mg. | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 outcomesResults by group80
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in Neuropathy Impairment Score-lower Limb (NIS-LL) Total Score at Week 72 | Tafamidis Meglumine 20 mg Once Daily (QD) | 2.3 | Full Range -3.0 to 46.0 | Units on a scale | |
| Change From Baseline in Neuropathy Impairment Score-lower Limb (NIS-LL) Total Score at Weeks 24, and 48 | Tafamidis Meglumine 20 mg Once Daily (QD) | Change From Baseline in Neuropathy Impairment Score-lower limb (NIS-LL) Total Score at Week 24 | 3.0 | Full Range -20.0 to 20.0 | Units on a scale |
| Change From Baseline in Neuropathy Impairment Score-lower Limb (NIS-LL) Total Score at Weeks 24, and 48 | Tafamidis Meglumine 20 mg Once Daily (QD) | Change From Baseline in Neuropathy Impairment Score-lower limb (NIS-LL) Total Score at Week 48 | 8.0 | Full Range -13.5 to 18.0 | Units on a scale |
| Change From Baseline in Total Quality of Life (TQOL) of Norfolk Quality of Life - Diabetic Neuropathy (Norfolk QOL-DN) at Weeks 24, 48, and 72 | Tafamidis Meglumine 20 mg Once Daily (QD) | Change From Baseline in TQOL of Norfolk QOL-DN at Week 24 | -1.00 | Full Range -26.00 to 54.00 | Units on a scale |
| Change From Baseline in Total Quality of Life (TQOL) of Norfolk Quality of Life - Diabetic Neuropathy (Norfolk QOL-DN) at Weeks 24, 48, and 72 | Tafamidis Meglumine 20 mg Once Daily (QD) | Change From Baseline in TQOL of Norfolk QOL-DN at Week 48 | 2.00 | Full Range -26.00 to 63.00 | Units on a scale |
| Change From Baseline in Total Quality of Life (TQOL) of Norfolk Quality of Life - Diabetic Neuropathy (Norfolk QOL-DN) at Weeks 24, 48, and 72 | Tafamidis Meglumine 20 mg Once Daily (QD) | Change From Baseline in TQOL of Norfolk QOL-DN at Week 72 | 8.00 | Full Range -11.00 to 74.00 | Units on a scale |
| Change From Baseline in 5 Domains of Norfolk QOL-DN at Weeks 24, 48, and 72 | Tafamidis Meglumine 20 mg Once Daily (QD) | Change From Baseline in Physical Functioning/Large Fiber Domain of Norfolk QOL-DN at Week 24 | -3.00 | Full Range -22.00 to 28.00 | Units on a scale |
| Change From Baseline in 5 Domains of Norfolk QOL-DN at Weeks 24, 48, and 72 | Tafamidis Meglumine 20 mg Once Daily (QD) | Change From Baseline in Physical Functioning/Large Fiber Domain of Norfolk QOL-DN at Week 48 | 2.00 | Full Range -13.00 to 38.00 | Units on a scale |
| Change From Baseline in 5 Domains of Norfolk QOL-DN at Weeks 24, 48, and 72 | Tafamidis Meglumine 20 mg Once Daily (QD) | Change From Baseline in Physical Functioning/Large Fiber Domain of Norfolk QOL-DN at Week 72 | 4.00 | Full Range -10.00 to 39.00 | Units on a scale |
| Change From Baseline in 5 Domains of Norfolk QOL-DN at Weeks 24, 48, and 72 | Tafamidis Meglumine 20 mg Once Daily (QD) | Change From Baseline in Activities of Daily Living Domain of Norfolk QOL-DN at Week 24 | 0.00 | Full Range -4.00 to 12.00 | Units on a scale |
1 to 10 of 80Source ClinicalTrials.gov
See all 80 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Disposition Phase: TREATMENT | STARTED | Tafamidis Meglumine 20 mg Once Daily (QD) | 15 | |
| Disposition Phase: TREATMENT | COMPLETED | Tafamidis Meglumine 20 mg Once Daily (QD) | 15 | |
| Disposition Phase: TREATMENT | NOT COMPLETED | Tafamidis Meglumine 20 mg Once Daily (QD) | 0 | |
| Disposition Phase: FOLLOW-UP | STARTED | Tafamidis Meglumine 20 mg Once Daily (QD) | 15 | |
| Disposition Phase: FOLLOW-UP | COMPLETED | Tafamidis Meglumine 20 mg Once Daily (QD) | 15 | |
| Disposition Phase: FOLLOW-UP | NOT COMPLETED | Tafamidis Meglumine 20 mg Once Daily (QD) | 0 |
Source ClinicalTrials.gov
Adverse events54
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 Meglumine 20 mg Once Daily (QD) | 2 | 15 | |
| other | Coagulopathy | Blood and lymphatic system disorders | Tafamidis Meglumine 20 mg Once Daily (QD) | 1 | 15 | |
| other | Leukopenia | Blood and lymphatic system disorders | Tafamidis Meglumine 20 mg Once Daily (QD) | 1 | 15 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Tafamidis Meglumine 20 mg Once Daily (QD) | 1 | 15 | |
| other | Palpitations | Cardiac disorders | Tafamidis Meglumine 20 mg Once Daily (QD) | 1 | 15 | |
| other | Supraventricular extrasystoles | Cardiac disorders | Tafamidis Meglumine 20 mg Once Daily (QD) | 1 | 15 | |
| other | Supraventricular tachycardia | Cardiac disorders | Tafamidis Meglumine 20 mg Once Daily (QD) | 1 | 15 | |
| other | Cataract | Eye disorders | Tafamidis Meglumine 20 mg Once Daily (QD) | 1 | 15 | |
| other | Abdominal discomfort | Gastrointestinal disorders | Tafamidis Meglumine 20 mg Once Daily (QD) | 1 | 15 | |
| other | Abdominal pain | Gastrointestinal disorders | Tafamidis Meglumine 20 mg Once Daily (QD) | 1 | 15 |
1 to 10 of 54Source ClinicalTrials.gov
See all 54 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Tafamidis Meglumine 20 mg Once Daily (QD) | 0 | 15 |
| Total, other adverse events | Tafamidis Meglumine 20 mg Once Daily (QD) | 13 | 15 |
| Total, serious adverse events | Tafamidis Meglumine 20 mg Once Daily (QD) | 3 | 15 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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
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