A Study of the Long-term Safety of Experimental Tafamidis For Transthyretin Cardiomyopathy

Enter your info and the study team will contact you soon!
Your data is securely stored and only shared with the research team
Logo Image
"Volunteer for research at UC Davis and contribute to discoveries that may improve health care for you, your family, and your community!"
Age: 18 years or older
Gender:
Any
Healthy Volunteers: No
Keywords: Cardiomyopathy, Transthyretin Amyloid Cardiomyopathy, heart failure, enlarged heart
Type: Drug study, Phase 3
Target:
1 Participants
Investigator:
Description
You have been diagnosed with transthyretin amyloid cardiomyopathy. This is a life-threatening disease involving misfolded protein in the heart. Symptoms include: heart failure, manifested by dyspnea, fatigue, orthostatic hypotension, and/or syncope. Currently there are no FDA approved therapies specific to treating amyloid cardiomyopathy.

In a clinical trial, experimental tafamidis was shown to be superior to placebo. It reduced the rate of hospitalizations from cardiac events. It also reduced the rate of death from this disease. The trial enrollment period has ended for this trial. Pfizer is currently pursuing approval in the United States of this drug for TTR amyloid disease.

It is not approved by the U.S. Food and Drug Administration (FDA) or any other regulatory agency in the United States.

During this study, you will be monitored by your physician in clinic to see how well you are responding to the study drug. The safety of tafamidis will also be evaluated by tracking any adverse events.
This study requires

If the FDA approves your use of tafamidis, the drug will be shipped directly to the Investigational Drug Pharmacy at UC Davis Medical Center. The medication will then be dispensed to you through the UC Davis Investigational Pharmacy and your physician. You will be prescribed the study drug, tafamidis, by your physician. The prescriptions will be refilled over an indefinite period of time. This is unless you transition to a commercial version of the drug or do not tolerate the drug well.

You will be taking a 80 mg dose of tafamidis orally, once a day, from the date you are enrolled until you are no longer enrolled in the study. The dose may be reduced to 40mg daily if the treating physician believes it is clinically indicated by your condition.

If you agree to take this drug, you will be followed closely by your treating physician approximately every 3 months. You may see your treating physician more or less often based on their judgment.

To monitor any effects of the drugs, your treating physician may perform: - physical exams, - draw blood, - order EKGs, - echocardiograms, and - do other tests based on your individual condition.

Whether or not they choose to do these tests will be at their discretion. Some of these procedures and tests may be done in between your visits with your treating physician should you choose so and will be interpreted by your treating physician shortly after results become available.

Who can participate

Inclusion criteria:

Cohort A: Completion of 30 months of study treatment on Pfizer Protocol B3461028 Cohort B: Patients in specific countries diagnosed with ATTR-CM who did not previously participate in Pfizer Study B3461028

Exclusion criteria:

-Liver and/or heart transplant, or implanted cardiac mechanical assist device

Benefits and risks of participating
BENEFITS:

We cannot promise any benefits to you as a result of taking part in this research study.


RISKS:

The study doctor will discuss the risks associated with taking part in this research study.
Compensation
You will not be paid to take part in this research study.
Resources
Schedule
Study duration and period
We expect that you will be in this research study indefinitely.
Recruitment period
From March 19, 2019
Location
UC Davis Medical Center
2315 Stockton Boulevard
Sacramento, CA 95817
Contact
Clinical Trials Office
Research Topic
Conditions:
  • Transthyretin (TTR) Amyloid Cardiomyopathy

Have any questions or want to learn more? Leave your contact details below and the research team will reach out to you.


Sending

Send SMS with Study Info:

SMS Sent!

Share Study Info via Email:


E-mail Preview