Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.

NCT05489523 · ClinicalTrials.gov registry record · Phase 4

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

A Phase 4 study of Transthyretin Cardiac Amyloidosis, sponsored by University of Texas Southwestern Medical Center.

Recruiting
Registry status
Phase 4
Development phase
25
Enrollment target
4
Study locations

NCT05489523 is a Phase 4 study of Transthyretin Cardiac Amyloidosis that is actively recruiting participants, run by University of Texas Southwestern Medical Center. The registered enrollment target is 25 participants, below the 630-participant average among 4 other Transthyretin Cardiac Amyloidosis trials with a reported enrollment target (96% lower). The trial reports 4 study locations across 4 states.

View on ClinicalTrials.gov ↗

View your shortlist →

The verdict

NCT05489523, a Phase 4 study of Transthyretin Cardiac Amyloidosis, is actively recruiting participants, sponsored by University of Texas Southwestern Medical Center.

RECRUITING
Registry status
Phase 4
Development phase
25 participants
Enrollment target
4
Study locations

Study Summary

Transthyretin cardiac amyloidosis (ATTR-CA) is a relentlessly progressive disease that can progress to end stage heart failure, at which point recently approved transthyretin production silencing or structure stabilizing therapies provide no clinical benefit. For well-selected individuals, heart transplantation is an excellent therapeutic option to improve survival. Historically, concomitant liver transplantation has been used to halt the progression of non-cardiac transthyretin amyloidosis (ATTR) manifestations, especially for individuals with TTR genotypes associated with significant neuropathy. However, despite this, patients continue to experience progressive non-cardiac manifestations, particularly gastrointestinal and neuropathic, which can have a substantial influence on post-heart transplantation morbidity. Concomitant liver transplantation is also associated with substantial morbidity and its future therapeutic role is questionable with recently established therapies for ATTR. Therefore, there is a clear unmet need to determine the utility and safety of ATTR targeted therapies for patients with recent heart transplantation for end-stage ATTR-CA. The central hypothesis of this proposal is that in patients who have received a heart transplantation for end-stage ATTR-CA, tafamidis therapy will be efficacious and well-tolerated. We aim to determine the safety and efficacy of tafamidis in stable patients who have undergone heart or combined heart/liver transplantation for ATTR (wild-type or variant) cardiac amyloidosis. The proposed study will be a single-arm intervention clinical trial with tafamidis. Because of the efficacy of tafamidis for both variant ATTR-CA and wild-type ATTR-CA, there is no clinical equipoise for an inactive-comparator placebo arm. The primary endpoint of this study will be serial change in plasma transthyretin (TTR) levels from baseline to 12 months at 3-month intervals. The secondary endpoints of this study will include serial changes i

Interventions

  • DRUG Tafamidis 61 MG

Study Locations (4)

California

  • Cedars-Sinai - Beverly Hills

New York

  • Columbia University Medical Center - New York

Ohio

  • Cleveland Clinic - Cleveland

Texas

  • UT Southwestern Medical Center - Dallas

Trial Details

FieldValue
Enrollment Target 25 participants
Start Date 2023-05-01
Est. Completion 2026-05-01
Phase Phase 4

What the Registry Record Tells You About NCT05489523

The ClinicalTrials.gov registry entry for NCT05489523 describes a study currently listed as recruiting, categorized as Phase 4. The registered enrollment target is 25 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 630-participant average among 4 other Transthyretin Cardiac Amyloidosis trials with a reported enrollment target (96% lower). The listed sponsor is University of Texas Southwestern Medical Center, which has 837 total studies on file at ClinicalTrials.gov.

The record links to 1 condition, with Transthyretin Cardiac Amyloidosis appearing as the primary indexed condition, and to 1 intervention - of which Tafamidis 61 MG is the first listed.

NCT05489523 reports 4 study locations spanning 4 distinct geographic areas - top geographies include California, New York, Ohio.

Frequently Asked Questions

What is clinical trial NCT05489523 about?

NCT05489523 is a clinical study titled "Safety, Efficacy, and Pharmacokinetics of Tafamidis in Patients With Transthyretin-mediated Amyloidosis Post Orthotopic Heart Transplantation". Transthyretin cardiac amyloidosis (ATTR-CA) is a relentlessly progressive disease that can progress to end stage heart failure, at which point recently approved transthyretin production silencing or structure stabilizing therapies provide no clinical benefit. For well-selected individuals, heart tra...

What is the current status of trial NCT05489523?

This trial is currently recruiting. It is a Phase 4 study. The enrollment target is 25 participants. The study started on 2023-05-01. Estimated completion is 2026-05-01.

What conditions does trial NCT05489523 study?

This clinical trial studies the following conditions: Transthyretin Cardiac Amyloidosis.

What interventions are being tested in trial NCT05489523?

The interventions under investigation include: Tafamidis 61 MG (DRUG).

Who is sponsoring clinical trial NCT05489523?

This trial is sponsored by University of Texas Southwestern Medical Center, which has 837 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT05489523 being conducted?

This trial has 4 study locations across California, New York, Ohio, Texas. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

Similar trials for Transthyretin Cardiac Amyloidosis

Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.

Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.