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This Study Will Examine the Clinical Effectiveness of Tafamidis in Patients With Mixed Phenotype Hereditary Transthyretin Amyloidosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT05139680
Recruitment Status : Active, not recruiting
First Posted : December 1, 2021
Last Update Posted : April 21, 2023
Information provided by (Responsible Party):

Brief 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.

Condition or disease Intervention/treatment
Hereditary Transthyretin Amyloidosis (ATTRv) Cardiomyopathy (CM), Mixed Phenotype Drug: tafamidis

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Study Type : Observational
Actual Enrollment : 1 participants
Observational Model: Cohort
Time Perspective: Retrospective
Official Title: Real-World Effectiveness of Tafamidis 80 mg or 61 mg on Neurologic Disease Progression in Patients With Mixed-Phenotype Variant Transthyretin Amyloid Cardiomyopathy (ATTRv-CM)
Actual Study Start Date : March 8, 2023
Estimated Primary Completion Date : November 1, 2023
Estimated Study Completion Date : November 1, 2023

Group/Cohort Intervention/treatment
Patients with mixed phenotype ATTRv-CM
Hereditary ATTR-CM patients presenting with mixed phenotype
Drug: tafamidis
80 or 61 milligrams (mg)
Other Names:
  • Vyndaqel
  • Vyndamax

Primary Outcome Measures :
  1. Rate of neurologic disease progression [ Time Frame: Baseline through at least 6 months of treatment ]
    Describe and compare the rate of neurologic disease progression before and after initiation of tafamidis in patients with mixed-phenotype ATTRv-CM receiving tafamidis 80 mg or 61 mg daily in a real-world setting.

Secondary Outcome Measures :
  1. Change from Baseline in modified Body Mass Index (BMI) [ Time Frame: Baseline (BL) through at least 6 months of treatment ]
    Assess change from BL in mBMI in patients with mixed-phenotype ATTRv-CM receiving 80 mg or 61 mg tafamidis

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
The study will include patients with mixed-phenotype ATTRv-CM who are treated in the Amyloidosis Program at MedStar.

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)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05139680

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United States, New York
New York, New York, United States, 10017
Sponsors and Collaborators
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Study Director: Pfizer CT.gov Call Center Pfizer
Additional Information:
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Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT05139680    
Other Study ID Numbers: B3461099
First Posted: December 1, 2021    Key Record Dates
Last Update Posted: April 21, 2023
Last Verified: April 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: 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.
Keywords provided by Pfizer:
Additional relevant MeSH terms:
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Amyloid Neuropathies, Familial
Heart Diseases
Cardiovascular Diseases
Proteostasis Deficiencies
Metabolic Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Nervous System Diseases
Amyloid Neuropathies
Peripheral Nervous System Diseases
Neuromuscular Diseases
Genetic Diseases, Inborn
Amyloidosis, Familial
Metabolism, Inborn Errors