Trial Outcomes & Findings for A Study to Examine the Clinical Effectiveness of Tafamidis in Patients With Mixed Phenotype Hereditary Transthyretin Amyloidosis (NCT NCT05139680)
NCT ID: NCT05139680
Last Updated: 2024-09-19
Results Overview
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.
COMPLETED
10 participants
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)
2024-09-19
Participant Flow
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).
Participant milestones
| Measure |
Tafamidis
Eligible participants with ATTRv-CM who initiated Tafamidis as either VYNDAQEL 80 mg (four 20-mg tafamidis meglumine capsules) orally once daily (QD) or VYNDAMAX 61 mg (one 61-mg tafamidis capsule) orally QD in a real world setting were included in this observational study.
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|---|---|
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Overall Study
STARTED
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10
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Overall Study
COMPLETED
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10
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Overall Study
NOT COMPLETED
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0
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Study to Examine the Clinical Effectiveness of Tafamidis in Patients With Mixed Phenotype Hereditary Transthyretin Amyloidosis
Baseline characteristics by cohort
| Measure |
Tafamidis
n=10 Participants
Eligible participants with ATTRv-CM who initiated Tafamidis as either VYNDAQEL 80 mg (four 20-mg tafamidis meglumine capsules) orally QD or VYNDAMAX 61 mg (one 61-mg tafamidis capsule) orally QD in a real world setting were included in this observational study.
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|---|---|
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Age, Continuous
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72.20 Years
STANDARD_DEVIATION 10.26 • n=5 Participants
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Sex: Female, Male
Female
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5 Participants
n=5 Participants
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Sex: Female, Male
Male
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5 Participants
n=5 Participants
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Ethnicity (NIH/OMB)
Hispanic or Latino
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0 Participants
n=5 Participants
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Ethnicity (NIH/OMB)
Not Hispanic or Latino
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1 Participants
n=5 Participants
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Ethnicity (NIH/OMB)
Unknown or Not Reported
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9 Participants
n=5 Participants
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Race (NIH/OMB)
American Indian or Alaska Native
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0 Participants
n=5 Participants
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Race (NIH/OMB)
Asian
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0 Participants
n=5 Participants
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Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
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0 Participants
n=5 Participants
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Race (NIH/OMB)
Black or African American
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8 Participants
n=5 Participants
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Race (NIH/OMB)
White
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1 Participants
n=5 Participants
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Race (NIH/OMB)
More than one race
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0 Participants
n=5 Participants
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Race (NIH/OMB)
Unknown or Not Reported
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1 Participants
n=5 Participants
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PRIMARY outcome
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)Population: All eligible participants whose data were included and observed in the study. Here "Number of Participants Analyzed" signifies the number of participants who were evaluable for this outcome measure and had non-missing data.
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.
Outcome measures
| Measure |
Tafamidis
n=7 Participants
Eligible participants with ATTRv-CM who initiated Tafamidis as either VYNDAQEL 80 mg (four 20-mg tafamidis meglumine capsules) orally QD or VYNDAMAX 61 mg (one 61-mg tafamidis capsule) orally QD in a real world setting were included in this observational study.
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|---|---|
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Neurologic Disease Progression: Number of Participants According to Muscle Weakness Assessment by Neuropathy Impairment Score (NIS) Subscale Score
No change in scores
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2 Participants
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Neurologic Disease Progression: Number of Participants According to Muscle Weakness Assessment by Neuropathy Impairment Score (NIS) Subscale Score
Increase in scores
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2 Participants
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Neurologic Disease Progression: Number of Participants According to Muscle Weakness Assessment by Neuropathy Impairment Score (NIS) Subscale Score
Decrease in scores
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3 Participants
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PRIMARY outcome
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)Population: All eligible participants whose data were included and observed in the study.
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.
Outcome measures
| Measure |
Tafamidis
n=10 Participants
Eligible participants with ATTRv-CM who initiated Tafamidis as either VYNDAQEL 80 mg (four 20-mg tafamidis meglumine capsules) orally QD or VYNDAMAX 61 mg (one 61-mg tafamidis capsule) orally QD in a real world setting were included in this observational study.
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|---|---|
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Neurologic Disease Progression: Number of Participants According to Walking Capacity Assessment by Polyneuropathy Disability (PND) Score
Decrease in scores
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3 Participants
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Neurologic Disease Progression: Number of Participants According to Walking Capacity Assessment by Polyneuropathy Disability (PND) Score
No change in scores
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6 Participants
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Neurologic Disease Progression: Number of Participants According to Walking Capacity Assessment by Polyneuropathy Disability (PND) Score
Increase in scores
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1 Participants
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PRIMARY outcome
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)Population: All eligible participants whose data were included and observed in the study.
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.
Outcome measures
| Measure |
Tafamidis
n=10 Participants
Eligible participants with ATTRv-CM who initiated Tafamidis as either VYNDAQEL 80 mg (four 20-mg tafamidis meglumine capsules) orally QD or VYNDAMAX 61 mg (one 61-mg tafamidis capsule) orally QD in a real world setting were included in this observational study.
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|---|---|
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Neurologic Disease Progression: Number of Participants According to Muscle Strength Assessment by Medical Research Council (MRC) Scale
No change in scores
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8 Participants
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Neurologic Disease Progression: Number of Participants According to Muscle Strength Assessment by Medical Research Council (MRC) Scale
Increase in scores
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1 Participants
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Neurologic Disease Progression: Number of Participants According to Muscle Strength Assessment by Medical Research Council (MRC) Scale
Decrease in scores
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1 Participants
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SECONDARY outcome
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)Population: All eligible participants whose data were included and observed in the study.
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.
Outcome measures
| Measure |
Tafamidis
n=10 Participants
Eligible participants with ATTRv-CM who initiated Tafamidis as either VYNDAQEL 80 mg (four 20-mg tafamidis meglumine capsules) orally QD or VYNDAMAX 61 mg (one 61-mg tafamidis capsule) orally QD in a real world setting were included in this observational study.
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|---|---|
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Modified Body Mass Index (mBMI)
Pre-Treatment
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1001.06 (kg/m^2)*(g/L)
Standard Deviation 269.52
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Modified Body Mass Index (mBMI)
Post-Treatment
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1070.92 (kg/m^2)*(g/L)
Standard Deviation 232.58
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Adverse Events
Tafamidis
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
- Publication restrictions are in place
Restriction type: OTHER