Evaluating tafamidis for patients with transthyretin cardiac amyloidosis after heart transplantation
An Open-label Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Tafamidis in Patients With Transthyretin-mediated Amyloidosis Post Orthotopic Heart Transplantation
PHASE4 · University of Texas Southwestern Medical Center · NCT05489523
This study is testing if tafamidis can help people with transthyretin cardiac amyloidosis feel better after they have had a heart or heart/liver transplant.
Quick facts
| Phase | PHASE4 |
|---|---|
| Study type | Interventional |
| Enrollment | 25 (estimated) |
| Ages | 18 Years to 90 Years |
| Sex | All |
| Sponsor | University of Texas Southwestern Medical Center (other) |
| Drugs / interventions | prednisone |
| Locations | 4 sites (Beverly Hills, California and 3 other locations) |
| Trial ID | NCT05489523 on ClinicalTrials.gov |
What this trial studies
This clinical trial aims to assess the safety and efficacy of tafamidis in patients who have undergone heart or combined heart/liver transplantation for transthyretin cardiac amyloidosis. The study will monitor plasma transthyretin levels over 12 months of therapy and evaluate the impact of tafamidis on neurological impairment, activity limitations, and transplantation-related adverse events. It is a single-arm intervention trial, meaning all participants will receive tafamidis without a placebo group. The trial seeks to provide insights into the pharmacokinetics of tafamidis in this patient population.
Who should consider this trial
Good fit: Ideal candidates are individuals who have received orthotopic heart transplantation for end-stage transthyretin cardiac amyloidosis at least 12 months prior to screening.
Not a fit: Patients with active malignancies, severe renal impairment, or those who have recently received other specific ATTR therapies may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could improve the management and outcomes for patients with transthyretin cardiac amyloidosis post-transplantation.
How similar studies have performed: While tafamidis has shown promise in other contexts, this specific application in post-heart transplantation patients is novel and has not been extensively tested.
Eligibility criteria
Show full inclusion / exclusion 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")
Where this trial is running
Beverly Hills, California and 3 other locations
- Cedars-Sinai — Beverly Hills, California, United States (RECRUITING)
- Columbia University Medical Center — New York, New York, United States (RECRUITING)
- Cleveland Clinic — Cleveland, Ohio, United States (RECRUITING)
- UT Southwestern Medical Center — Dallas, Texas, United States (RECRUITING)
Study contacts
- Principal investigator: Justin Grodin, MD — University of Texas Southwestern Medical Center
- Study coordinator: Amir Mehdizadeh
- Email: Amir.Mehdizadeh@utsouthwestern.edu
- Phone: 214-648-7507
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Transthyretin Cardiac Amyloidosis, heart transplantation, transthyretin amyloidosis, ATTR, tafamidis