Real-World Effectiveness of Tafamidis on Neurologic Disease Progression in Patients With Mixed Phenotype Hereditary Transthyretin Amyloidosis Cardiomyopathy

Sponsor

Pfizer (Industry)

Overall Status

Not yet recruiting

CT.gov ID

NCT05139680

Collaborator

(none)

150

Enrollment

Location

Anticipated Duration (Months)

76.1

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This will be an observational, retrospective cohort study using structured secondary anonymized data. Patients with mixed-phenotype ATTRv-CM receiving high dose tafamidis for at least 12 months will be identified. Relevant data will be extracted through at least 12 months following the initiation of tafamidis.

Condition or Disease	Intervention/Treatment	Phase
Hereditary Transthyretin Amyloidosis (ATTRv) Cardiomyopathy (CM), Mixed Phenotype	Drug: tafamidis

Detailed Description

Transthyretin amyloidosis (ATTR amyloidosis) is a rare, life-threatening disease caused by the deposition of transthyretin-derived amyloid fibrils in the peripheral nerves, heart, and other organs. ATTR amyloidosis may arise from mutations in the transthyretin (TTR) gene (ATTRv amyloidosis), or from the aggregation of wild-type TTR (ATTRwt amyloidosis). ATTRv amyloidosis is now widely recognized as a spectrum of disease that can manifest as polyneuropathy, cardiomyopathy, or a mixed phenotype. This non-interventional study will examine the value of high dose tafamidis for delaying neurologic disease progression in mixed-phenotype ATTRv-CM patients.

Study Design

Study Type:

Observational

Anticipated Enrollment :

150 participants

Observational Model:

Cohort

Time Perspective:

Retrospective

Official Title:

Real-World Effectiveness of Tafamidis 80 mg or 61 mg on Neurologic Disease Progression in Patients With Mixed-Phenotype Hereditary Transthyretin Amyloid Cardiomyopathy

Anticipated Study Start Date :

Nov 1, 2022

Anticipated Primary Completion Date :

Dec 31, 2022

Anticipated Study Completion Date :

Dec 31, 2022

Arms and Interventions

Arm	Intervention/Treatment
Patients with mixed phenotype ATTRv-CM Hereditary ATTR-CM patients presenting with mixed phenotype	Drug: tafamidis 80 or 61 milligrams (mg) Other Names: Vyndaqel Vyndamax

Arm

Intervention/Treatment

Patients with mixed phenotype ATTRv-CM

Hereditary ATTR-CM patients presenting with mixed phenotype

Drug: tafamidis

80 or 61 milligrams (mg)

Other Names:

Vyndaqel

Vyndamax

Outcome Measures

Primary Outcome Measures

Rate of neurologic disease progression [Baseline through at least 12 months of treatment]
Describe and compare the rate of neurologic disease progression before and after initiation of tafamidis in patients with mixed-phenotype ATTRv-CM receiving tafamidis 80 mg or 61 mg daily in a real-world setting.

Secondary Outcome Measures

Change from Baseline in modified Body Mass Index (BMI) [Baseline (BL) through at least 12 months of treatment]
Assess change from BL in mBMI in patients with mixed-phenotype ATTRv-CM receiving 80 mg or 61 mg tafamidis

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Age ≥18 years at diagnosis
Diagnosed with hereditary ATTR-CM, mixed phenotype
Treated with tafamidis (VYNDAQEL 80 mg [four 20-mg tafamidis meglumine capsules] orally once daily or VYNDAMAX 61 mg [one 61-mg tafamidis capsule] orally once daily) for ≥12 months
Have had ≥1 pre- and ≥2 post-treatment neurologic assessments

Exclusion Criteria:

History of organ transplant
Wild-type TTR genotype
Individuals who are non-ambulatory
Prior treatment with any disease-modifying therapy (investigational or approved) alone or in combination, except tafamidis, as either 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 (e.g., diabetes mellitus, B12 deficiency, HIV infection)