Clinical trial NCT05139680: A Study to Examine the Clinical Effectiveness of Tafamidis in Patients With Mixed Phenotype Hereditary Transthyretin Amyloidosis
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
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hereditary Transthyretin Amyloidosis (ATTRv) Cardiomyopathy (CM), Mixed Phenotype
Keywords: transthyretin
amyloidosis
cardiomyopathy
tafamidis
polyneuropathy
progression
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2021-12-01
Enrollment: 10
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The study will include patients with mixed-phenotype ATTRv-CM who are treated in the Amyloidosis Program at MedStar.

Brief summary: This study will examine the clinical effectiveness of Tafamidis in patients with Mixed Phenotype Hereditary Transthyretin Amyloidosis using data that already exist in patients' medical records.


Eligibility criteria: Inclusion Criteria:

* Age ≥18 years at diagnosis
* Diagnosed with ATTRv-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 ≥6 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)

Primary outcomes:
measure: Neurologic Disease Progression: Number of Participants According to Muscle Weakness Assessment by Neuropathy Impairment Score (NIS) Subscale Score
timeFrame: Pre-treatment baseline period to post-treatment period (data for specified duration was extracted from medical records and observed retrospectively in this study from 08-Mar-2023 to 19-May-2023)
measure: Neurologic Disease Progression: Number of Participants According to Walking Capacity Assessment by Polyneuropathy Disability (PND) Score
timeFrame: Pre-treatment baseline period to post-treatment period (data for specified duration was extracted from medical records and observed retrospectively in this study from 08-Mar-2023 to 19-May-2023)
measure: Neurologic Disease Progression: Number of Participants According to Muscle Strength Assessment by Medical Research Council (MRC) Scale
timeFrame: Pre-treatment baseline period to post-treatment period (data for specified duration was extracted from medical records and observed retrospectively in this study from 08-Mar-2023 to 19-May-2023)
Secondary outcomes:
measure: Modified Body Mass Index (mBMI)
timeFrame: Pre-treatment baseline period, post-treatment period (data for specified duration was extracted from medical records and observed retrospectively in this study from 08-Mar-2023 to 19-May-2023)
Other outcomes:
Locations:
city: New York
country: United States
facility: Pfizer
state: New York
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: