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 2023 Phase 4 trial • 53 Patients • NCT04814186

23%

COVID-19

11%

Cardiac failure

11%

Diarrhoea

Hyperuricaemia

Vomiting

Hepatic function abnormal

Nasopharyngitis

Cardiac failure acute

Pneumonia

Haematuria

Cough

Disease progression

Respiratory failure

Atrial thrombosis

Cardiorenal syndrome

Hereditary neuropathic amyloidosis

Oedema peripheral

Erysipelas

Gastroenteritis

Respiratory tract infection

Electrolyte imbalance

Hyperkalaemia

Dizziness

Haemorrhagic cerebral infarction

Syncope

Chronic kidney disease

100%

80%

60%

40%

20%

Study treatment Arm

All Participants

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

Do I qualify?Apply below to find out

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).