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Transthyretin Stabilizer

Tafamidis for Transthyretin Amyloid Cardiomyopathy

Phase 4
Recruiting
Led By Jan Griffin, MD
Research Sponsored by University of Texas Southwestern Medical Center
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Has a stable immunosuppressive regimen and ≤ 10 mg of prednisone (or equivalent) at time of enrollment.
Has a Karnofsky performance status ≥ 70%
Must not have
Has been treated for acute cellular or antibody mediated rejection in the past 3 months
Has an estimated glomerular filtration rate (eGFR) ≤ 15 ml/min/1.73 m2
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline, 3, 6, 9, and 12 months
Awards & highlights
Drug Has Already Been Approved
No Placebo-Only Group
Pivotal Trial

Summary

This trial will study the safety and efficacy of tafamidis in patients who have undergone heart or combined heart/liver transplantation for ATTR cardiac amyloidosis. The primary endpoint of this study will be serial change in plasma transthyretin (TTR) levels from baseline to 12 months.

Who is the study for?
This trial is for patients who've had a heart transplant at least 12 months ago due to end-stage Transthyretin Amyloid Cardiomyopathy (ATTR-CA). They should be relatively healthy otherwise, with stable organ function and not taking high doses of immune-suppressing drugs. People with active cancers (except certain skin cancers), infections like hepatitis or HIV, severe heart issues post-transplant, recent rejection treatments, very poor kidney function, or other types of amyloidosis can't join.
What is being tested?
The study tests Tafamidis 61 MG's safety and effectiveness in patients after a heart transplant due to ATTR-CA. It's a single-arm trial where all participants receive the drug—no placebo group—focusing on how it affects protein levels related to ATTR-CA over one year and its impact on nerve health, body mass index changes, specific side effects related to the transplant, and how the body processes the drug.
What are the potential side effects?
While specific side effects are not listed here, this study will closely monitor any adverse events that may arise from taking Tafamidis post-heart transplantation. These could include potential impacts on gastrointestinal health and nerves among others.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am on a stable medication regimen for my immune system, taking 10 mg or less of prednisone.
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I am mostly able to care for myself and carry out daily activities.
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I had a heart transplant over a year ago due to advanced ATTR.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have been treated for organ rejection in the last 3 months.
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My kidney function is severely reduced.
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I have been diagnosed with leptomeningeal disease or AL amyloidosis.
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I have an active cancer that is not a non-melanoma skin cancer.
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I have a lymph node condition after a transplant.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline, 3, 6, 9, and 12 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, 3, 6, 9, and 12 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Serial change from baseline in plasma TTR levels at 12 months
Secondary study objectives
Number of hepatic or renal transplant-specific adverse events
Number of transplant-specific adverse events
Serial change from baseline in Composite Autonomic Symptom Score (COMPASS-31) at12 months
+2 more

Awards & Highlights

Drug Has Already Been Approved
The FDA has already approved this drug, and is just seeking more data.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups
Experimental Treatment
Group I: Treatment ArmExperimental Treatment1 Intervention
Tafamidis 61 mg

Find a Location

Who is running the clinical trial?

University of Texas Southwestern Medical CenterLead Sponsor
1,089 Previous Clinical Trials
1,059,658 Total Patients Enrolled
Jan Griffin, MDPrincipal InvestigatorMedical University of South Carolina
Justin Grodin, MDPrincipal InvestigatorUT Southwestern Medical Center

Media Library

Tafamidis (Transthyretin Stabilizer) Clinical Trial Eligibility Overview. Trial Name: NCT05489523 — Phase 4
Transthyretin Amyloid Cardiomyopathy Research Study Groups: Treatment Arm
Transthyretin Amyloid Cardiomyopathy Clinical Trial 2023: Tafamidis Highlights & Side Effects. Trial Name: NCT05489523 — Phase 4
Tafamidis (Transthyretin Stabilizer) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05489523 — Phase 4
~4 spots leftby Apr 2025
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