Real-World Effectiveness of High-Dose Tafamidis on Neurol... | NCT06393465

Studies
Real-World Effectiveness of High-Dose Tafamidis on Neurologic Disease Progression in Mixed-Phenotype Transthyretin Amyloid Cardiomyopathy

NCT06393465

Sponsor

Pfizer

Status

Completed

Last Update Posted

Apr 29, 2026Actual

Enrollment

50Actual

Phase

Not provided

Conditions

Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)

Interventions

Tafamidis

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

ID	Type	Description	Link
NCT06393465	Registry Identifier	ClinicalTrials.gov

Status Module

Record Verification Date

Apr 2026

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jun 15, 2024Actual

Primary Completion Date

Apr 7, 2026Actual

Completion Date

Apr 7, 2026Actual

First Submitted Date

Apr 26, 2024

First Submission Date that Met QC Criteria

Apr 26, 2024

First Posted Date

May 1, 2024Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Apr 24, 2026

Last Update Posted Date

Apr 29, 2026Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

50Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Patients with mixed phenotype ATTR-CM		ATTR-CM patients presenting with mixed phenotype	Drug: Tafamidis

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Tafamidis	Drug	61 milligrams under real world conditions	Patients with mixed phenotype ATTR-CM

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Rate of neurologic disease progression	Describe and compare the rate of neurologic disease progression before and after initiation of tafamidis in patients with mixed-phenotype ATTR-CM receiving tafamidis 61 mg daily in a real world setting.	Baseline through at least 12 months of treatment

Secondary Outcomes

Measure	Description	Time Frame
Change from Baseline in modified Body Mass Index (BMI)	Assess change from BL in mBMI in patients with mixed phenotype ATTR-CM 61 mg tafamidis	Baseline (BL) through at least 12 months of treatment

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Age ≥18 years at diagnosis.
Diagnosed with ATTRv-CM or ATTRwt-CM, mixed phenotype.
Treated with tafamidis, as VYNDAMAX 61 mg (one 61-mg tafamidis capsule) orally once daily for ≥12 months or started on tafamidis 20 mg then switched to VYNDAMAX 61 mg for ≥12 months
Have had ≥1 pre- and ≥1 post-treatment neurologic assessments.

Exclusion Criteria:

History of any organ transplant.
Individuals who are non-ambulatory.
Prior or current treatment with any disease-modifying therapy (investigational or approved) alone or in combination, except tafamidis, as VYNDAQEL 80 mg [four 20-mg tafamidis meglumine capsules] orally once daily or VYNDAMAX 61 mg [one 61-mg tafamidis capsule] orally once daily.
Peripheral neuropathy attributed to causes other than ATTR amyloidosis (eg, diabetes mellitus, B12 deficiency, hypothyroidism, shingles,Lyme disease, HIV infection, secondary to injury, chronic kidney disease).
Patient's data fails to pass data quality checks.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

Not provided

Standard Ages

AdultOlder Adult

Study Population

The study will include patients with mixed-phenotype ATTR-CM who are treated in the Amyloidosis Program at MedStar in the U.S and three other study sites (Canada, Portugal and Italy).

Sampling Method

Non-Probability Sample

Name	Affiliation	Role
Pfizer CT.gov Call Center	Pfizer	Study Director

Facility	Status	City	State	ZIP	Country	Contacts
Pfizer		New York	New York	10001	United States

Label	URL
To obtain contact information for a study center near you, click here.	View source

IPD Sharing Statement Module

Plan to Share IPD

Description

Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical\_trials/trial\_data\_and\_results/data\_requests.

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided