Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)NCT06393465
Real-World Effectiveness of High-Dose Tafamidis on Neurologic Disease Progression in Mixed-Phenotype Transthyretin Amyloid Cardiomyopathy
An observational study of Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM), run by Pfizer at 1 site in United States.
Trial summary
This study will examine the clinical effectiveness of Tafamidis in patients with Mixed Phenotype Transthyretin Amyloidosis using data that already exist in patients' medical records Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)
- What is being tested?
- Tafamidis
- How many people
- 50 participants
- Where
- 1 siteUnited States
- Study dates
- 15 Jun 2024to 7 Apr 2026
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: 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).
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Real-World Effectiveness of High-Dose Tafamidis on Neurologic Disease Progression in Mixed-Phenotype Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
This study will examine the clinical effectiveness of Tafamidis in patients with Mixed Phenotype Transthyretin Amyloidosis using data that already exist in patients' medical records
- Being tested
- Tafamidis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TafamidisDrug
Also called Vyndamax
61 milligrams under real world conditions
| Study group | Role | What they receive |
|---|---|---|
| Patients with mixed phenotype ATTR-CM | ATTR-CM patients presenting with mixed phenotype Receives: Drug: Tafamidis |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of neurologic disease progression
Describe and compare the rate of neurologic disease progression before and after initiation of tafamidis in patients with mixed-phenotype ATTR-CM receiving tafamidis 61 mg daily in a real world setting.
Measured: Baseline through at least 12 months of treatment
Secondary outcomes (1)
Change from Baseline in modified Body Mass Index (BMI)
Assess change from BL in mBMI in patients with mixed phenotype ATTR-CM 61 mg tafamidis
Measured: Baseline (BL) through at least 12 months of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 May 2024
- Study start15 Jun 2024
- Primary completion7 Apr 2026
- Study completion7 Apr 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)Studies of the same condition, open and under way.
- More trials run by PfizerThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 50 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pfizer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Pfizer CT.gov Call Center, Study director, Pfizer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06393465
- Sponsor's protocol ID
- B3461121
- Other IDs
- NCT06393465
- Status last verified
- Apr 2026
- First submitted
- 26 Apr 2024
- Last updated
- 29 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- 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.
Publications
Fine NM, Streicher NS, Conceicao I, Agostinho J, Obici L, Palladini G, Carlsson M, Amass L, Sheikh FH. Neurologic Stability During Tafamidis Treatment in Transthyretin Amyloidosis with a Mixed Phenotype: A Multicenter Real-World Study. Cardiol Ther. 2026 Oct 7. doi: 10.1007/s40119-026-00479-z. Online ahead of print.
PubMed 42844536