Safety, Efficacy, and Pharmacokinetics of Tafamidis in Patients With Transthyretin-mediated Amyloidosis Post Orthotopic Heart Transplantation

Launched by UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER · Aug 2, 2022

Keywords

ClinConnect Summary

This clinical trial is studying a medication called tafamidis in patients who have recently received a heart transplant due to a serious condition known as transthyretin cardiac amyloidosis (ATTR-CA). This disease can lead to severe heart problems, and while heart transplantation can significantly help, many patients still face challenges, especially with non-heart-related symptoms. The trial aims to find out if tafamidis is safe and effective for these patients, particularly in reducing symptoms and improving their overall health after heart transplant.

To be eligible for this trial, participants must have had a heart transplant at least 12 months ago and be in stable health with a good performance status. This means they should be able to perform daily activities fairly well. They should also be on a stable treatment plan for their immune system and not have received certain other ATTR treatments recently. If someone joins the trial, they will take tafamidis and be monitored regularly for changes in their health and any side effects. This study could provide valuable insights into how tafamidis can help improve the lives of patients after heart transplantation for ATTR-CA.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Has received orthotopic heart transplantation for end-stage ATTRv or ATTRwt ≥12 months prior to screening. Concomitant hepatic and renal transplantation with adequate allograft function are included.
• • Has a stable immunosuppressive regimen and ≤ 10 mg of prednisone (or equivalent) at time of enrollment.
• • Has a Karnofsky performance status ≥ 70%
• Exclusion Criteria:
• • Has previously received inotersen within the past 180 days, patisiran within the past 90 days, tafamidis within the past 14 days, or diflunisal in the past 14 days.
• • Participating in a clinical trial for ATTR targeted therapies.
• • Has an estimated glomerular filtration rate (eGFR) ≤ 15 ml/min/1.73 m2
• • Has known leptomeningeal or AL amyloidosis
• • Has active post-transplant lymphoproliferative disease
• • Excluding non-melanomatous skin cancers, has an active malignancy.
• • Has active infection with hepatitis B, hepatitis C, human immunodeficiency virus, or cytomegalovirus (CMV). For CMV, donor/ recipient exposure status and prior treated CMV disease on stable doses of antiviral therapies are not excluded.
• • Has cardiac allograft dysfunction defined by left ventricular ejection fraction (LVEF) \<50% by echocardiogram within the past 3 months
• • Has been treated for acute cellular or antibody mediated rejection in the past 3 months
• • Has criteria to meet International Society for Heart and Lung Transplantation standardized nomenclature for severe coronary allograft vasculopathy ("ISHLT CAV3")