This Study Will Examine the Clinical Effectiveness of Tafamidis in Patients With Mixed Phenotype Hereditary Transthyretin Amyloidosis
- Conditions
- Hereditary Transthyretin Amyloidosis (ATTRv) Cardiomyopathy (CM), Mixed Phenotype
- Interventions
- Registration Number
- NCT05139680
- Lead Sponsor
- Pfizer
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 10
- 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
- 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)
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Patients with mixed phenotype ATTRv-CM tafamidis Hereditary ATTR-CM patients presenting with mixed phenotype
- Primary Outcome Measures
Name Time Method Neurologic Disease Progression: Number of Participants According to Muscle Weakness Assessment by Neuropathy Impairment Score (NIS) Subscale Score 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) Neurologic disease progression meant worsening of neurologic function over the time, is assessed by NIS subscale score in this outcome measure. NIS is a composite neurologic impairment score that assesses muscle weakness, sensation, and reflexes by physical exam. NIS subscale for muscle weakness assessment has a score range from 0 (minimum value) to 192 (maximum value). Lower scores indicates normal to mild impairment. Participants were classified as: no change, increase or decrease in NIS subscale scores from pre-treatment baseline period to post-treatment period. Pre-treatment baseline period: at least 6 months and up to 12 months before treatment initiation. Post-treatment period: at least 6 months after treatment initiation with a +/- 3 months window.
Neurologic Disease Progression: Number of Participants According to Walking Capacity Assessment by Polyneuropathy Disability (PND) Score 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) Neurologic disease progression meant worsening of neurologic function over the time, is assessed by PND score in this outcome measure. PND is a scoring system to assess participants' walking capacity. It consists of four stages from stage 0 (no impairment) to stage IV (confined to a wheelchair or bedridden). Lower scores indicates normal to mild impairment. Participants were classified as: no change, increase or decrease in PND scores from pre-treatment to post -treatment period. Pre-treatment baseline period: at least 6 months and up to 12 months before treatment initiation. Post-treatment period: at least 6 months after treatment initiation with a +/- 3 months window.
Neurologic Disease Progression: Number of Participants According to Muscle Strength Assessment by Medical Research Council (MRC) Scale 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) Neurologic disease progression meant worsening of neurologic function over the time, is assessed by MRC scale in this outcome measure. MRC assessed muscle strength using a score of 0 (no contraction) to 5 (normal power), to grade the power of a particular muscle group in relation to the movement of a single joint. The higher scores mean a better outcome. Participants were classified as: no change, increase or decrease in MRC scale score from pre-treatment to post-treatment period. Pre-treatment baseline period: at least 6 months and up to 12 months before treatment initiation. Post-treatment period: at least 6 months after treatment initiation with a +/- 3 months window.
- Secondary Outcome Measures
Name Time Method Modified Body Mass Index (mBMI) 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) mBMI was calculated as the product of BMI in kilogram per meter square (kg/m\^2) and serum albumin in gram per litre (g/L) to compensate for peripheral edema. Pre-treatment baseline period: at least 6 months and up to 12 months before treatment initiation. Post-treatment period: at least 6 months after treatment initiation with a +/- 3 months window.
Trial Locations
- Locations (1)
Pfizer
🇺🇸New York, New York, United States