N/A
Completed 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
Source: ClinicalTrials.gov NCT05139680 ↗
Enrolled (actual)
10
Serious AEs
—
Results posted
Sep 2024
Primary outcomePrimary: Neurologic Disease Progression: Number of Participants According to Muscle Weakness Assessment by Neuropathy Impairment Score (NIS) Subscale Score — 2; 2; 3 Participants
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.
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 | — |
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. Informational only — not medical advice.