An observational study in Hereditary Transthyretin Amyloidosis (ATTRv) Cardiomyopathy (CM), Mixed Phenotype, sponsored by Pfizer. Completed at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-09-19.
Sponsored by Pfizer · Observational
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.
122 studies on the registry are indexed under Amyloid Neuropathies, Familial; 71 are open to participants now.
This study's enrollment of 10 is below the median of 180 across 57 observational studies indexed under Amyloid Neuropathies, Familial.
Browse Amyloid Neuropathies, Familial studies →Pfizer is the lead sponsor of 3,244 studies on the registry; 139 are open to participants now.
Of its 582 completed or terminated interventional studies of FDA-regulated products, 381 (65%) have results posted.
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The study will include patients with mixed-phenotype ATTRv-CM who are treated in the Amyloidosis Program at MedStar.
Exclusion Criteria:
Hereditary ATTR-CM patients presenting with mixed phenotype
Drug: tafamidis
80 or 61 milligrams (mg)
Also known as: Vyndaqel, Vyndamax
Neurologic Disease Progression: Number of Participants According to Muscle Weakness Assessment by Neuropathy Impairment Score (NIS) Subscale Score
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.
Time frame: 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: Number of Participants According to Walking Capacity Assessment by Polyneuropathy Disability (PND) Score
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.
Time frame: 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: Number of Participants According to Muscle Strength Assessment by Medical Research Council (MRC) Scale
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.
Time frame: 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)
Modified Body Mass Index (mBMI)
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.
Time frame: 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)
Data of participants with mixed-phenotype variant transthyretin amyloid cardiomyopathy (ATTRv-CM) who initiated Tafamidis as either VYNDAQEL 80 milligram (mg) or VYNDAMAX 61 mg in a real world setting, were observed retrospectively in this study. Anonymized data was extracted from participant electronic medical records (EMR): MedStar health system in Washington D.C.(08-March-2023 to 19-May-2023).
| Milestone | Tafamidis |
|---|---|
| Started | 10 |
| Completed | 10 |
| Not completed | 0 |
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.
| Participants | Tafamidis |
|---|---|
| No change in scores | 2 |
| Increase in scores | 2 |
| Decrease in scores | 3 |
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.
| Participants | Tafamidis |
|---|---|
| No change in scores | 6 |
| Increase in scores | 1 |
| Decrease in scores | 3 |
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.
| Participants | Tafamidis |
|---|---|
| No change in scores | 8 |
| Increase in scores | 1 |
| Decrease in scores | 1 |
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.
| (kg/m^2)*(g/L) | Tafamidis |
|---|---|
| Pre-Treatment | 1001.06 ± 269.52 |
| Post-Treatment | 1070.92 ± 232.58 |
Collected over Not applicable as safety data was not planned to be collected for the study.. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Tafamidis | — | — | — |
Baseline analysis population included all eligible participants whose data were included and observed in the study.
| Age, Continuous(Years) | Tafamidis |
|---|---|
| Mean | 72.20 ± 10.26 |
| Sex: Female, Male(Participants) | Tafamidis |
|---|---|
| Female | 5 |
| Male | 5 |
| Ethnicity (NIH/OMB)(Participants) | Tafamidis |
|---|---|
| Hispanic or Latino | 0 |
| Not Hispanic or Latino | 1 |
| Unknown or Not Reported | 9 |
| Race (NIH/OMB)(Participants) | Tafamidis |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 8 |
| White | 1 |
| More than one race | 0 |
| Unknown or Not Reported | 1 |
Documents are hosted by the registry — open the source record to download them.
Plan to share: No — Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
This study is completed, as verified in Apr 2024. You cannot join it, but the record below documents what was studied.
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