Real-World data shows High-Dose tafamidis may slow nerve damage in ATTR-CM patients
NCT ID NCT06393465
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study looked at medical records of 50 people with a rare heart condition called ATTR-CM to see if high-dose tafamidis (Vyndamax) slows nerve damage. Researchers compared how fast nerve problems got worse before and after starting the drug. The goal was to see if the medication works in everyday medical practice, not just in controlled studies.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- tafamidis (Vyndamax)
- What this could lead to
- If successful, this could confirm that high-dose tafamidis helps slow nerve damage in people with mixed-phenotype ATTR-CM, supporting its use in real-world care.
- What could go wrong
- This is a small, completed study that only looked back at existing medical records, not a controlled trial. Results may not apply to all patients, and tafamidis does not cure the disease.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Participants
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50 people
The number who actually took part.
- Started
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Jun 2024
- Finished
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Apr 2026
- Lead sponsor
-
A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
Who is studied
The study will include patients with mixed-phenotype ATTR-CM who are treated in the Amyloidosis Program at MedStar in the U.S and three other study sites (Canada, Portugal and Italy).
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Age ≥18 years at diagnosis. * Diagnosed with ATTRv-CM or ATTRwt-CM, mixed phenotype. * Treated with tafamidis, as VYNDAMAX 61 mg (one 61-mg tafamidis capsule) orally once daily for ≥12 months or started on tafamidis 20 mg then switched to VYNDAMAX 61 mg for ≥12 months * Have had ≥1 pre- and ≥1 post-treatment neurologic assessments. Exclusion Criteria: * History of any organ transplant. * Individuals who are non-ambulatory. * Prior or current treatment with any disease-modifying therapy (investigational or approved) alone or in combination, except tafamidis, as 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 (eg, diabetes mellitus, B12 deficiency, hypothyroidism, shingles,Lyme disease, HIV infection, secondary to injury, chronic kidney disease). * Patient's data fails to pass data quality checks.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Pfizer
New York, New York, 10001, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.