What side effects are associated with this type of intervention?

Tafamidis, a transthyretin stabilizer used to treat Transthyretin Amyloid Cardiomyopathy, has been shown to have a similar incidence of adverse events compared to placebo. Common side effects include mild gastrointestinal symptoms, such as diarrhea and nausea, as well as urinary tract infections. Overall, tafamidis is well-tolerated, with no significant increase in serious adverse events compared to placebo.

What prior FDA approvals exist?

Tafamidis, marketed as Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis), is an FDA-approved drug for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM). Tafamidis works by stabilizing the transthyretin protein, preventing its misfolding and subsequent deposition in the heart. The FDA approved an 80 mg daily dose of tafamidis meglumine and a 61 mg daily dose of tafamidis for this condition. These approvals were based on clinical trials demonstrating that tafamidis significantly reduces mortality, cardiovascular-related hospitalizations, and declines in functional capacity and quality of life in patients with ATTR-CM.

What other types of research exist?

Promising alternatives to tafamidis for treating transthyretin amyloid cardiomyopathy include transthyretin silencers such as patisiran and inotersen. These therapies have shown effectiveness in clinical trials and represent novel approaches to managing the disease.

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Transthyretin Stabilizer

Tafamidis for Cardiomyopathy

Phase 3

Waitlist Available

Research Sponsored by Pfizer

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline to month 60

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial is testing the safety of tafamidis, a medication for patients with a heart condition, over a long period. Tafamidis helps stabilize a protein to prevent it from forming harmful deposits in the heart. The study aims to provide additional safety data and continue treatment for several years. Tafamidis was initially approved for commercial use in Europe in 2011 and has since been approved for use in Japan, Mexico, and Argentina.

Who is the study for?

This trial is for two groups: Cohort A, who finished 30 months in a previous Pfizer study, and Cohort B, patients from certain countries with transthyretin cardiomyopathy (ATTR-CM) but not in the prior study. Those with heart or liver transplants or mechanical heart devices can't join.

What is being tested?

The trial is testing Tafamidis's long-term safety for treating ATTR-CM. It's an open-label study, meaning everyone knows they're getting Tafamidis and there are no placebos or comparison drugs involved.

What are the potential side effects?

While specific side effects aren't listed here, generally such medications could cause issues like gastrointestinal symptoms, potential liver problems, and possibly nerve-related symptoms.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline to month 60

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline to month 60

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to month 60 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

All-cause mortality and incidence of treatment emergent adverse events

Other study objectives

All-cause hospitalization

Body Mass Index/modified Body Mass Index

Cardiac biomarkers

+4 more

Side effects data

From 2020 Phase 3 trial • 93 Patients • NCT00925002

67%

Fall

28%

Neuropathy Peripheral

22%

Diarrhoea

22%

Hypoaesthesia

22%

Cough

22%

Orthostatic Hypotension

22%

Oedema Peripheral

17%

Dysphagia

17%

Contusion

17%

Muscular Weakness

17%

Dizziness

17%

Dysphonia

17%

Upper Respiratory Tract Infection

11%

Nausea

11%

Fatigue

11%

Cardiac Failure

11%

Cataract

11%

Onychomycosis

11%

Urinary Tract Infection

11%

Pneumonia

11%

Skin Abrasion

11%

Weight Decreased

11%

Fluid Overload

11%

Pain in Extremity

11%

Fine Motor Skill Dysfunction

11%

Memory Impairment

11%

Syncope

11%

Nephrolithiasis

11%

Dyspnoea

11%

Decubitus Ulcer

11%

Skin Ulcer

11%

Sepsis

11%

Dental Caries

11%

Malaise

11%

Sinusitis

11%

Arthralgia

11%

Chest Pain

Abdominal Pain Lower

Gastrointestinal Disorder

Inguinal Hernia

Asthenia

Implant Site Pain

Cellulitis

Gastrointestinal Motility Disorder

Odynophagia

Vomitting

Bronchitis

Exercise Tolerance Decreased

Hordeolum

Cardiac Amyloidosis

Hyperthyroidism

Amyloidosis

Gastroenteritis

Wrist Fracture

Wound

Hypokalaemia

Heart Transplant

Tachycardia

Deafness

Dry Eye

Pyelonephritis

Rhinitis

Respiratory Tract Infection

Vulvovaginal Mycotic Infection

Skin Laceration

Thermal Burn

Muscle Spasms

Musculoskeletal Stiffness

Neck Pain

Amnesia

Burning Sensation

Headache

Presyncope

Carotid Artery Stenosis

Sciatica

Depression

Insomnia

Dysuria

Benign Prostatic Hyperplasia

Vulvovaginal Pruritus

Night Sweats

Pruritus

Purpura

Hypotension

Peripheral Vascular Disorder

Epilepsy

Cardiac Failure Acute

Disease Progression

Hypovolaemia

Confusional State

Bundle Branch Block Bilateral

Constipation

Gastroenteritis Viral

Gingivitis

Pneumonia Streptococcal

Medication Error

Muscle Injury

Decreased Appetite

Vitamin B12 Deficiency

Vitamin D Deficiency

Arthritis

Foot Deformity

Muscle Atrophy

Musculoskeletal Chest Pain

Haematuria

Hyperhidrosis

Cardiac Arrest

Cardiomyopathy

Gait Disturbance

Diverticulitis

Head Injury

Joint Injury

Alopecia

Burns Second Degree

Influenza

Tooth Fracture

Blood Thyroid Stimulating Hormone Increased

Haemoglobin Increased

Hepatic Enzyme Increased

Eczema

Carpal Tunnel Decompression

100%

80%

60%

40%

20%

Study treatment Arm

NonVal30Met: Tafamidis

Val30Met: Placebo Then Tafamidis

Val30Met: Tafamidis Then Tafamidis

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups

Experimental Treatment

Group I: TafamidisExperimental Treatment1 Intervention

Active treatment - 61 mg or if not available, tafamidis megulmine 80 mg

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Tafamidis

2018

Completed Phase 4

~2400

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Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Transthyretin Amyloid Cardiomyopathy (ATTR-CM) include transthyretin stabilizers like tafamidis, and transthyretin silencers such as patisiran and inotersen. Tafamidis works by stabilizing the transthyretin protein, preventing it from misfolding and forming amyloid deposits in the heart, which helps reduce mortality and cardiovascular-related hospitalizations. Patisiran and inotersen, on the other hand, reduce the production of transthyretin protein by interfering with its genetic expression. These mechanisms are crucial for ATTR-CM patients as they help manage the progression of the disease, improve quality of life, and reduce the burden of symptoms associated with amyloid deposition in the heart.

Navigating the Complex Web of Prescribing Amyloidosis Therapeutics: A Primer.Tafamidis: A First-in-Class Transthyretin Stabilizer for Transthyretin Amyloid Cardiomyopathy.Impact of Tafamidis on Health-Related Quality of Life in Patients With Transthyretin Amyloid Cardiomyopathy (from the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial).