N/A
N=10
A Study to Examine the Clinical Effectiveness of Tafamidis in Patients With Mixed Phenotype Hereditary Transthyretin Amyloidosis
Hereditary Transthyretin Amyloidosis (ATTRv) Cardiomyopathy (CM), Mixed Phenotype
Bottom Line
View on ClinicalTrials.gov: NCT05139680 ↗Enrolled (actual)
10
Serious AEs
—
Results posted
Sep 2024
Primary outcome: Primary: Neurologic Disease Progression: Number of Participants According to Muscle Weakness Assessment by Neuropathy Impairment Score (NIS) Subscale Score — 2; 2; 3 Participants
Study Design & Population
- Study type
- Observational
- Phase
- N/A
- Interventions
- tafamidis (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Pfizer
- Primary completion
- May 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Neurologic Disease Progression: Number of Participants According to Muscle Weakness Assessment by Neuropathy Impairment Score (NIS) Subscale Score |
2; 2; 3 | — |
| PRIMARY Neurologic Disease Progression: Number of Participants According to Walking Capacity Assessment by Polyneuropathy Disability (PND) Score |
6; 1; 3 | — |
| PRIMARY Neurologic Disease Progression: Number of Participants According to Muscle Strength Assessment by Medical Research Council (MRC) Scale |
8; 1; 1 | — |
| SECONDARY Modified Body Mass Index (mBMI) |
1001.06; 1070.92 | — |
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)
Data sourced from ClinicalTrials.gov (NCT05139680). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.