Clinical Trials

Numerous clinical trials evaluate tafamidis across Phase 1 through Phase 4 investigations and non-interventional designs, primarily studying transthyretin amyloid cardiomyopathy, hereditary transthyretin amyloidosis with polyneuropathy, primary cardiomyopathies, cardiac amyloidosis, and healthy volunteer pharmacokinetics. Sponsored by pharmaceutical companies such as Pfizer and Bayer alongside academic centers like Yale University and Heinrich-Heine University Duesseldorf, these active, completed, and upcoming studies include safety surveillance programs and post-orthotopic liver transplantation evaluations.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT06940336 RECRUITING
Transthyretin Amyloid Polyneuropathy
Qilu Pharmaceutical Co., Ltd.
 2025-06-19 PHASE4
NCT07298044 RECRUITING
Transthyretin Amyloid Cardiomyopathy
Bayer
 2026-02-13 PHASE4
NCT05489523 ACTIVE_NOT_RECRUITING
Transthyretin Cardiac Amyloidosis
University of Texas Southwestern Medical Center
 2023-05-01 PHASE4
NCT04849754 RECRUITING
Cardiac Amyloidosis
GCS Ramsay Santé pour l'Enseignement et la Recherche
 2021-02-25
NCT07587697 COMPLETED
Healthy Adults
Pfizer
 2026-05-11 PHASE1
NCT07470866 TERMINATED
Healthy
Pfizer
 2026-03-23 PHASE1
NCT04535349 RECRUITING
Amyloidosis Transthyretin
University Hospital, Caen
 2020-10-08
NCT06705569 COMPLETED
Healthy
Pfizer
 2024-12-20 PHASE1
NCT06273839 COMPLETED
Healthy
Pfizer
 2024-02-27 PHASE1
NCT06086353 Recruiting
Transthyretin Amyloid Cardiomyopathy
Pfizer
 2024-03-25 --
NCT05938218 Not yet recruiting
Amyloidosis Cardiac|Virtual Reality
Heinrich-Heine University Duesseldorf
 2023-12-01 Not Applicable
NCT02791230 COMPLETED
Transthyretin (TTR) Amyloid Cardiomyopathy
Pfizer
 2016-06-13 PHASE3
NCT04814186 COMPLETED
Transthyretin Amyloid Cardiomyopathy
Pfizer
 2021-07-22 PHASE4
NCT05489523 Recruiting
Transthyretin Cardiac Amyloidosis
University of Texas Southwestern Medical Center
 2023-05-01 Phase 4
NCT04828993 COMPLETED
Transthyretin Amyloid Polyneuropathy (ATTR-PN)
Pfizer
 2021-04-28 PHASE4
NCT05498701 COMPLETED
Healthy
Pfizer
 2022-09-29 PHASE1
NCT05482308 COMPLETED
Healthy
Pfizer
 2022-08-29 PHASE1
NCT05560555 Completed
Hereditary Transthyretin Amyloidosis (ATTRv)|Polyneuropathy
Pfizer
 2022-10-24 --
NCT05374564 Recruiting
Cardiomyopathies Primary
Yale University|Pfizer
 2022-08-16 Phase 1
NCT04963985 UNKNOWN
Transthyretin Amyloidosis
Peking University Third Hospital
 2021-06-01 PHASE4
NCT04814186 Completed
Transthyretin Amyloid Cardiomyopathy
Pfizer
 2021-07-22 Phase 4
NCT04575116 COMPLETED
Healthy
Pfizer
 2020-09-17 PHASE1
NCT04253353 COMPLETED
Healthy Volunteers
Pfizer
 2020-02-06 PHASE1
NCT00925002 COMPLETED
ATTR-PN
Pfizer
 2009-08-05 PHASE3
NCT00935012 COMPLETED
ATTR-CM; TTR-CM
Pfizer
 2009-09-30 PHASE3
NCT03662191 COMPLETED
Healthy Volunteers
Pfizer
 2018-09-28 PHASE1
NCT03280173 COMPLETED
Healthy
Pfizer
 2017-09-29 PHASE1
NCT03266705 COMPLETED
Healthy
Pfizer
 2017-09-20 PHASE1
NCT01994889 COMPLETED
Transthyretin (TTR) Amyloid Cardiomyopathy
Pfizer
 2013-12-09 PHASE3
NCT02746926 COMPLETED
Healthy
Pfizer
 2016-04-29 PHASE1
NCT02697864 COMPLETED
Healthy
Pfizer
 2016-04 PHASE1
NCT02534740 COMPLETED
Healthy
Pfizer
 2015-09 PHASE1
NCT02406560 COMPLETED
Healthy Volunteers
Pfizer
 2015-04 PHASE1
NCT02217813 COMPLETED
Healthy Volunteers
Pfizer
 2014-10 PHASE1
NCT02189330 COMPLETED
Healthy Volunteers
Pfizer
 2014-05 PHASE1
NCT01435655 COMPLETED
Transthyretin Familial Amyloid Polyneuropathy
Pfizer
 2011-11 PHASE3
NCT01775761 COMPLETED
TTR Cardiomyopathy
Pfizer
 2013-01 PHASE1
NCT01655511 COMPLETED
TTR Cardiomyopathy
Pfizer
 2012-07 PHASE1
NCT01369836 COMPLETED
Healthy
Pfizer
 2011-07 PHASE1

(data from https://clinicaltrials.gov, updated on 2025-07-30)

Check the Tafamidis product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Tafamidis selectively binds to the thyroxine-binding sites of transthyretin (TTR), kinetically stabilizing the tetrameric protein structure and preventing its dissociation into monomeric subunits. By inhibiting TTR misfolding and downstream amyloid fibril aggregation, this kinetic stabilization halts amyloidogenesis and prevents progressive myocardial and neurological tissue damage in conditions such as transthyretin amyloid cardiomyopathy and polyneuropathy.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

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Note: Technical data last updated: Sep 1, 2026.