Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Patients with mixed phenotype ATTRv-CM | Hereditary ATTR-CM patients presenting with mixed phenotype |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| tafamidis | Drug | 80 or 61 milligrams (mg) |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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. | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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) |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
The study will include patients with mixed-phenotype ATTRv-CM who are treated in the Amyloidosis Program at MedStar.
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Pfizer CT.gov Call Center | Pfizer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Pfizer | New York | New York | 10017 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 38521883 | Derived | Streicher N, Amass L, Wang R, Stephens JM, LeMasters T, Raina R, Merrill E, Sheikh FH. Real-World Effectiveness of High-Dose Tafamidis on Neurologic Disease Progression in Mixed-Phenotype Variant Transthyretin Amyloid Cardiomyopathy. Cardiol Ther. 2024 Jun;13(2):359-368. doi: 10.1007/s40119-024-00362-9. Epub 2024 Mar 23. |
| Label | URL |
|---|---|
| To obtain contact information for a study center near you, click here. | View source |
Not provided
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.
Not provided
Not provided
Not provided
Not provided
Not provided
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).
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | 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. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | May 31, 2023 | Apr 16, 2024 |
Not provided
Not provided
Not provided
Not provided
| 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) |
| COMPLETED |
|
| NOT COMPLETED |
|
Baseline analysis population included all eligible participants whose data were included and observed in the study.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Tafamidis | 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. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | 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. | Posted | Count of Participants | 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) |
|
|
| ||||||||||||||||||||||||||||||
| Primary | 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. | All eligible participants whose data were included and observed in the study. | Posted | Count of Participants | 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) |
|
| |||||||||||||||||||||||||||||||
| Primary | 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. | All eligible participants whose data were included and observed in the study. | Posted | Count of Participants | 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) |
|
| |||||||||||||||||||||||||||||||
| Secondary | 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. | All eligible participants whose data were included and observed in the study. | Posted | Mean | Standard Deviation | (kg/m^2)*(g/L) | 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) |
|
|
Not applicable as safety data was not planned to be collected for the study.
Due to nature of data in data sources for the study, individual participant data were not retrieved or validated, and it was not possible to link (i.e., identify a potential association between) a particular product and medical event for any individual. Thus, the minimum criteria for reporting an adverse event (AE) (ie, identifiable participant, identifiable reporter, a suspect product, and event) could not be met.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Tafamidis | 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. | 0 | 0 | 0 | 0 | 0 | 0 |
Not provided
Not provided
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.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Pfizer ClinicalTrials.gov Call Center | Pfizer Inc. | 1-800-718-1021 | ClinicalTrials.gov_Inquiries@pfizer.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jun 9, 2023 | Apr 16, 2024 | SAP_001.pdf |
| ID | Term |
|---|---|
| C567782 | Amyloidosis, Hereditary, Transthyretin-Related |
| D009202 | Cardiomyopathies |
| D000686 | Amyloidosis |
| D011115 | Polyneuropathies |
| D018450 | Disease Progression |
| ID | Term |
|---|---|
| D006331 | Heart Diseases |
| D002318 | Cardiovascular Diseases |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D010523 | Peripheral Nervous System Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
Not provided
Not provided
| ID | Term |
|---|---|
| C547076 | tafamidis |
Not provided
Not provided
Not provided
| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Participants |
|
|
| Participants |
|
|
|