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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 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
6%
Abdominal Pain Lower
6%
Gastrointestinal Disorder
6%
Inguinal Hernia
6%
Asthenia
6%
Implant Site Pain
6%
Cellulitis
6%
Gastrointestinal Motility Disorder
6%
Odynophagia
6%
Vomitting
6%
Bronchitis
6%
Exercise Tolerance Decreased
6%
Hordeolum
6%
Cardiac Amyloidosis
6%
Hyperthyroidism
6%
Amyloidosis
6%
Gastroenteritis
6%
Wrist Fracture
6%
Wound
6%
Hypokalaemia
6%
Heart Transplant
6%
Tachycardia
6%
Deafness
6%
Dry Eye
6%
Pyelonephritis
6%
Rhinitis
6%
Respiratory Tract Infection
6%
Vulvovaginal Mycotic Infection
6%
Skin Laceration
6%
Thermal Burn
6%
Muscle Spasms
6%
Musculoskeletal Stiffness
6%
Neck Pain
6%
Amnesia
6%
Burning Sensation
6%
Headache
6%
Presyncope
6%
Carotid Artery Stenosis
6%
Sciatica
6%
Depression
6%
Insomnia
6%
Dysuria
6%
Benign Prostatic Hyperplasia
6%
Vulvovaginal Pruritus
6%
Night Sweats
6%
Pruritus
6%
Purpura
6%
Hypotension
6%
Peripheral Vascular Disorder
6%
Epilepsy
6%
Cardiac Failure Acute
6%
Disease Progression
6%
Hypovolaemia
6%
Confusional State
6%
Bundle Branch Block Bilateral
6%
Constipation
6%
Gastroenteritis Viral
6%
Gingivitis
6%
Pneumonia Streptococcal
6%
Medication Error
6%
Muscle Injury
6%
Decreased Appetite
6%
Vitamin B12 Deficiency
6%
Vitamin D Deficiency
6%
Arthritis
6%
Foot Deformity
6%
Muscle Atrophy
6%
Musculoskeletal Chest Pain
6%
Haematuria
6%
Hyperhidrosis
6%
Cardiac Arrest
6%
Cardiomyopathy
6%
Gait Disturbance
6%
Diverticulitis
6%
Head Injury
6%
Joint Injury
6%
Alopecia
6%
Burns Second Degree
6%
Influenza
6%
Tooth Fracture
6%
Blood Thyroid Stimulating Hormone Increased
6%
Haemoglobin Increased
6%
Hepatic Enzyme Increased
6%
Eczema
6%
Carpal Tunnel Decompression
100%
80%
60%
40%
20%
0%
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

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).

Find a Location

Who is running the clinical trial?

PfizerLead Sponsor
4,661 Previous Clinical Trials
17,843,256 Total Patients Enrolled
4 Trials studying Transthyretin Amyloid Cardiomyopathy
400 Patients Enrolled for Transthyretin Amyloid Cardiomyopathy
Pfizer CT.gov Call CenterStudy DirectorPfizer
3,546 Previous Clinical Trials
14,884,412 Total Patients Enrolled
4 Trials studying Transthyretin Amyloid Cardiomyopathy
400 Patients Enrolled for Transthyretin Amyloid Cardiomyopathy

Media Library

Tafamidis (Transthyretin Stabilizer) Clinical Trial Eligibility Overview. Trial Name: NCT02791230 — Phase 3
Transthyretin Amyloid Cardiomyopathy Research Study Groups: Tafamidis
Transthyretin Amyloid Cardiomyopathy Clinical Trial 2023: Tafamidis Highlights & Side Effects. Trial Name: NCT02791230 — Phase 3
Tafamidis (Transthyretin Stabilizer) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02791230 — Phase 3
~184 spots leftby Dec 2025