Phase 3, open label study designed to obtain additional safety data for 20mg and 80mg tafamidis or tafamidis 61mg where available, continue providing to subjects who have completed 30 months of blinded treatment on Protocol B3461028 with tafamidis for up to 60 additional months, or until subject has access to tafamidis for ATTR-CM via prescription. Additional group of patients who have not previously participated in B3461028 included to received tafamidis until local availability by precription.

Phase 1

• Conditions: Transthyretin amyloid cardiomyopathy (ATTR-CM); MedDRA version: 20.0Level: LLTClassification code 10002020Term: Amyloid cardiomyopathySystem Organ Class: 100000004849; Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]

• Registration Number: EUCTR2016-000868-42-DE
• Lead Sponsor: Pfizer Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2016-10-11
• Last Update Posted: 8 July 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 1400

Inclusion Criteria

1. Male and female subjects with TTR amyloid cardiomyopathy who have completed 30 months of study treatment on Protocol B3461028.
2. Evidence of a personally signed and dated informed consent document indicating that the subject has been informed of all pertinent aspects of the study.
3. Subjects who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.
4. Male subjects able to father children and female subjects of childbearing potential and at risk for pregnancy must agree to use 2 highly effective methods of contraception hroughout the study and for at least 28 days after the last dose of assigned treatment.
Female subjects who are not of childbearing potential (ie, meet at least 1 of the following criteria):
? Have undergone a documented hysterectomy and/or bilateral oophorectomy;
? Have medically confirmed ovarian failure; or
? Achieved postmenopausal status, defined as follows: cessation of regular menses for at least 12 consecutive months with no alternative pathological or physiological cause; status may be confirmed by having a serum folliclestimulating hormone (FSH) level confirming the post-menopausal state.
All other female subjects (including females with tubal ligations) will be considered o be of childbearing potential.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 70
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1330

Exclusion Criteria

1. Chronic use of diflunisal, TTR stabilizer, tauroursodeoxycholate, doxycycline, digitalis, calcium channel blockers, investigational drug(s) or other experimental interventions, other than tafamidis, independently or as part of a study within 30 days prior to enrollment.
2. Use of certain non-steroidal anti-inflammatory drugs (NSAIDs)
3. Liver and/or heart transplant, or implanted cardiac mechanical assist device.
4. Pregnant females (or planning to become pregnant during the study interval); breastfeeding females; male subjects with partners currently pregnant.
5. Require initiation of treatment with calcium channel blockers.
6. Urinary retention requiring chronic self-catheterization.
7. Breach of compliance with treatment/significant protocol violations during conduct of B3461028 for which the subject was accountable.
8. Subjects who are investigational site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the investigator, or subjects who are Pfizer employees directly involved in the conduct of he study.
9. Other severe acute or chronic medical or psychiatric condition or laboratory bnormality that may increase the risk associated with study participation or investigational product administration, or that may interfere with the interpretation of study results and, in the judgment of the investigator, would make the subject an appropriate for entry into this study.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: ? To obtain additional, long-term, safety data for tafamidis in subjects with transthyretin amyloid cardiomyopathy (TTR-CM).<br>? To provide investigational product, tafamidis, to TTR-CM subjects who complete 30 months of blinded treatment on protocol B3461028.;Secondary Objective: Not Applicable;Primary end point(s): Safety as measured by:<br>? All-cause mortality.<br>? Incidence of treatment-emergent adverse events.;Timepoint(s) of evaluation of this end point: Both primary end points will be assessed throughout the study.