Who is sponsoring NCT04828993?

NCT04828993 is sponsored by Pfizer.

What drugs are being tested in NCT04828993?

Interventions in NCT04828993: tafamidis meglumine.

What condition does NCT04828993 study?

NCT04828993 studies Transthyretin Amyloid Polyneuropathy (ATTR-PN).

Is NCT04828993 still recruiting?

NCT04828993 is currently completed. Last updated 12 March 2024.

Are results published for NCT04828993?

Yes — primary outcome results have been posted to ClinicalTrials.gov. See the outcomes section above for details.

Last reviewed 2024-03-12 · How we verify

NCT04828993

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

The Effect Of Tafamidis Meglumine In Transthyretin Amyloid Polyneuropathy Patients

Completed Phase 4 Results posted Last updated 12 March 2024

What this trial tests

Phase 4 trial testing tafamidis meglumine in Transthyretin Amyloid Polyneuropathy (ATTR-PN) in 15 participants. Completed in 12 February 2023.

Timeline

28 April 2021

Primary endpoint
12 February 2023

12 February 2023

Quick facts

Lead sponsor	Pfizer
Phase	Phase 4
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	15
Start date	28 April 2021
Primary completion	12 February 2023
Estimated completion	12 February 2023
Sites	9 locations across China

Drugs / interventions tested

tafamidis meglumine (Tafamidis Meglumine) — full drug profile →

Conditions studied

Transthyretin Amyloid Polyneuropathy (ATTR-PN) — all drugs for Transthyretin Amyloid Polyneuropathy (ATTR-PN) →

Sponsor

Pfizer — full company profile →

Who can join

Adults 18 to 80, any sex, with Transthyretin Amyloid Polyneuropathy (ATTR-PN). Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Change From Baseline in Neuropathy Impairment Score-lower Limb (NIS-LL) Total Score at Week 72 Primary · 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 s

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	2.3	-3.0 – 46.0

Change From Baseline in Neuropathy Impairment Score-lower Limb (NIS-LL) Total Score at Weeks 24, and 48 Secondary · Baseline, Week 24, Week 48

Change From Baseline in Neuropathy Impairment Score-lower limb (NIS-LL) Total Score at Week 24

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	3.0	-20.0 – 20.0

Change From Baseline in Neuropathy Impairment Score-lower limb (NIS-LL) Total Score at Week 48

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	8.0	-13.5 – 18.0

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 Secondary · 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.

Change From Baseline in TQOL of Norfolk QOL-DN at Week 24

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	-1.00	-26.00 – 54.00

Change From Baseline in TQOL of Norfolk QOL-DN at Week 48

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	2.00	-26.00 – 63.00

Change From Baseline in TQOL of Norfolk QOL-DN at Week 72

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	8.00	-11.00 – 74.00

Change From Baseline in 5 Domains of Norfolk QOL-DN at Weeks 24, 48, and 72 Secondary · 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)

Change From Baseline in Physical Functioning/Large Fiber Domain of Norfolk QOL-DN at Week 24

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	-3.00	-22.00 – 28.00

Change From Baseline in Physical Functioning/Large Fiber Domain of Norfolk QOL-DN at Week 48

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	2.00	-13.00 – 38.00

Change From Baseline in Physical Functioning/Large Fiber Domain of Norfolk QOL-DN at Week 72

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	4.00	-10.00 – 39.00

Change From Baseline in Activities of Daily Living Domain of Norfolk QOL-DN at Week 24

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	0.00	-4.00 – 12.00

Change From Baseline in Activities of Daily Living Domain of Norfolk QOL-DN at Week 48

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	1.00	-4.00 – 15.00

Change From Baseline in Activities of Daily Living Domain of Norfolk QOL-DN at Week 72

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	3.00	-2.00 – 15.00

Change From Baseline in Symptoms Domain of Norfolk QOL-DN at Week 24

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	1.00	-13.00 – 9.00

Change From Baseline in Symptoms Domain of Norfolk QOL-DN at Week 48

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	2.00	-6.00 – 9.00

Change From Baseline in Modified Body Mass Index (mBMI) at Weeks 4, 8, 12, 24, 36, 48, and 72 Secondary · 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.

Change From Baseline in mBMI at Week 4

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	-5.9	-87.2 – 95.5

Change From Baseline in Modified Body Mass Index (mBMI) at Week 8

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	22.3	-101.2 – 219.7

Change From Baseline in Modified Body Mass Index (mBMI) at Week 12

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	9.2	-39.0 – 199.3

Change From Baseline in Modified Body Mass Index (mBMI) at Week 24

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	21.8	-132.8 – 179.1

Change From Baseline in Modified Body Mass Index (mBMI) at Week 36

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	37.0	-79.3 – 207.7

Change From Baseline in Modified Body Mass Index (mBMI) at Week 48

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	41.4	-130.2 – 152.6

Change From Baseline in Modified Body Mass Index (mBMI) at Week 72

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	9.4	-161.8 – 179.5

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 Secondary · 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

Change from Baseline in Physical Component Summary at Week 24

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	-2.15	-9.29 – 11.84

Change from Baseline in Physical Component Summary at Week 48

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	-0.12	-12.13 – 12.08

Change from Baseline in Physical Component Summary at Week 72

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	-1.60	-17.61 – 8.89

Change from Baseline in Mental Component Summary at Week 24

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	1.21	-9.56 – 19.30

Change from Baseline in Mental Component Summary at Week 48

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	-2.56	-31.69 – 9.36

Change from Baseline in Mental Component Summary at Week 72

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	-6.10	-31.85 – 8.17

Change From Baseline in EuroQoL 5 Dimensions 5 Levels (EQ-5D-5L) Index Score at Weeks 24, 48, and 72 Secondary · 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 pr

Change from Baseline in EQ-5D-5L Index Score at Week 24

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	0.00	-0.50 – 0.22

Change from Baseline in EQ-5D-5L Index Score at Week 48

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	-0.05	-0.51 – 0.11

Change from Baseline in EQ-5D-5L Index Score at Week 72

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	-0.16	-0.75 – 0.11

Number of Participants Reporting Treatment-Emergent Adverse Events (TEAEs) Secondary · 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.

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	13

Number of Participants With Categorical Vital Signs Data Secondary · 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.

Pulse rate <40 bpm

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	0

Pulse rate >120 bpm

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	0

Standing diastolic BP <50 mmHg

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	4

Standing diastolic BP increase ≥20 mmHg

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	3

Standing diastolic BP decrease ≥20 mmHg

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	0

Standing systolic BP <90 mmHg

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	6

Standing systolic BP increase ≥30 mmHg

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	5

Standing systolic BP decrease ≥30 mmHg

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	3

Number of Participants With Categorical Electrocardiogram (ECG) Data Secondary · 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.

ECG mean heart rate <40 beats/minute

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	0

ECG mean heart rate >120 beats/minute

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	0

PR interval not otherwise specified ≥300 milliseconds (msec)

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	0

PR interval not otherwise specified % change ≥25/50%

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	0

QRS interval not otherwise specified ≥140 msec

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	1

QRS interval not otherwise specified %change ≥50%

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	1

QT interval not otherwise specified ≥500 msec

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	0

corrected QT (QTc) interval not otherwise specified ≥450 and <480 msec

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	9

Number of Participants With Clinically Significant Echocardiography (ECHO) Value Related to Primary Diagnosis (Transthyretin Amyloidosis [ATTR]) at Baseline, Weeks 24, 48, and 72 Secondary · Baseline, Weeks 24, 48, and 72

Clinically significant ECHO findings include: left ventricular (LV) posterior wall thickness greater than or equal to (\>=)13 mm, LV septal thickness \>= 13 mm, right ventricular thickness \>= 7 mm, ratio of peak mitral early diastolic and atrial contraction velocity (E/A ratio) \>= 2, prime septal (E/E) \>15, ejection fraction \< 50 percent (%), E deceleration time \<= 150 millisecond (ms), isovolumic relaxation time (IVRT) \<= 70 ms, any valve thickening (\> trace regurgitation in mitral, aortic, pulmonary, or tricuspid valves), abnormal respiratory variation of inferior vena cava, pericardi

Number of Participants at Baseline

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	9

Number of Participants at Week 24

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	11

Number of Participants at Week 48

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	9

Number of Participants at Week 72

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	10

Number of Participants With Clinical Laboratory Abnormalities Secondary · Baseline up to Week 72

Protocol-required safety laboratory assessments include: Lymphocytes \<0.8 × LLN; Neutrophils \<0.8 × LLN, or \>1.2 × ULN; Basophils \>1.2 × ULN; Activated Partial Thromboplastin Time \>1.1 × ULN; Prothrombin Time \>1.1 × ULN; Prothrombin International Normalized Ratio \>1.1 × ULN; Bilirubin \>1.5 × ULN; Urate \> 1.2 × ULN; Cholesterol \>1.3 × ULN; Potassium \<0.9 × LLN; Phosphate \>1.2 × ULN; Bicarbonate \>1.1 × ULN; Thyroid Stimulating Hormone \>1.2 × ULN; URINE Protein ≥1; URINE Hemoglobin ≥1; Nitrite ≥1; URINE Erythrocytes ≥20; Epithelial Cells ≥6; and Casts \>1.

Group	Value	95% CI
Tafamidis Meglumine 20 mg Once Daily (QD)	12

Adverse events — posted to ClinicalTrials.gov

Time frame: Baseline up to Week 77. Reporting threshold: 1%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Tafamidis Meglumine 20 mg Once Daily (QD)

Serious: 3/15 (20%)

Deaths: 0/15

Serious adverse events (3 terms)

Reaction	System	Tafamidis Meglumine 20 mg …
Amyloidosis	Immune system disorders	—
Femur fracture	Injury, poisoning and procedural complications	—
Orthostatic hypotension	Vascular disorders	—

Other adverse events (51 terms — click to expand)

Reaction	System	Tafamidis Meglumine 20 mg …
Upper respiratory tract infection	Infections and infestations	—
Urinary tract infection	Infections and infestations	—
Fall	Injury, poisoning and procedural complications	—
Anaemia	Blood and lymphatic system disorders	—
Diarrhoea	Gastrointestinal disorders	—
Blood thyroid stimulating hormone decreased	Investigations	—
Urinary sediment present	Investigations	—
White blood cells urine positive	Investigations	—
Hypertriglyceridaemia	Metabolism and nutrition disorders	—
Hyperuricaemia	Metabolism and nutrition disorders	—
Coagulopathy	Blood and lymphatic system disorders	—
Leukopenia	Blood and lymphatic system disorders	—
Thrombocytopenia	Blood and lymphatic system disorders	—
Palpitations	Cardiac disorders	—
Supraventricular extrasystoles	Cardiac disorders	—
Supraventricular tachycardia	Cardiac disorders	—
Cataract	Eye disorders	—
Abdominal discomfort	Gastrointestinal disorders	—
Abdominal pain	Gastrointestinal disorders	—
Constipation	Gastrointestinal disorders	—
Nausea	Gastrointestinal disorders	—
Vomiting	Gastrointestinal disorders	—
Oedema peripheral	General disorders	—
Hyperbilirubinaemia	Hepatobiliary disorders	—
Hypersensitivity	Immune system disorders	—
Seasonal allergy	Immune system disorders	—
COVID-19	Infections and infestations	—
Compression fracture	Injury, poisoning and procedural complications	—
Ligament sprain	Injury, poisoning and procedural complications	—
Limb injury	Injury, poisoning and procedural complications	—
Blood creatine phosphokinase increased	Investigations	—
Blood lactate dehydrogenase increased	Investigations	—
Blood pressure increased	Investigations	—
Electrocardiogram ST segment elevation	Investigations	—
Electrocardiogram abnormal	Investigations	—
Myoglobin blood increased	Investigations	—
N-terminal prohormone brain natriuretic peptide increased	Investigations	—
Protein urine present	Investigations	—
QRS axis abnormal	Investigations	—
Total bile acids increased	Investigations	—

Most-reported serious reactions: Amyloidosis, Femur fracture, Orthostatic hypotension.

Data from ClinicalTrials.gov NCT04828993 adverse events section.

Sponsor's own description

This is a single-arm, open-label, multicenter study designed to evaluate the efficacy, safety, tolerability as well as pharmacodynamics of tafamidis meglumine in ATTR-PN participants in China. Approximately 10-15 participants are planned to be enrolled. All enrolled participants will receive oral tafamidis meglumine 20 mg soft capsules once daily for 72 weeks (18 months).

Publications & conference data

No peer-reviewed publications indexed yet for this trial. Completed trials usually publish results within 12-18 months.

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Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT04828993 (US National Library of Medicine, public domain)
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Pfizer
Last refreshed: 12 March 2024

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