What the trial was testing
The NEURO-TTRansform enrolled 168 patients with hereditary attr amyloidosis. The study was sponsored by Ionis Pharmaceuticals and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was a large trial designed to confirm whether the treatment works well enough for wider use. Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
82% drop in transthyretin protein levels and better nerve function.
JAMA · 2023 · NCT04136184
These findings — that in transthyretin protein and improved nerve function on monthly eplontersen — were published in the JAMA and represent the headline result of the study.
Researchers tracked outcomes across 168 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with hereditary attr amyloidosis, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
Eplontersen (Wainua) is FDA-approved and available now for hereditary ATTR amyloidosis with nerve disease. It is given as a monthly under-the-skin injection that can be done at home. Ask a neurologist about treatment for confirmed ATTR amyloidosis.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open hereditary attr amyloidosis trials
Comprehensive Program for Hereditary Transthyretin Amyloidosis
The Comprehensive Program for Hereditary Transthyretin Amyloidosis describes a prospective observational study focused on understanding hereditary transthyretin amyloidosis (ATTR), a progressive and potentially fatal condition marked by amyloid fibril deposits impacting multiple organs. The trial aims to characterize patient phenotypes, investigate factors affecting disease progression, and identify minimum criteria for disease onset. Conducted at Néstor Kirchner Hospital, the trial enrolls participants over 18 years old with confirmed pathogenic TTR variants. It includes thorough evaluations such as genetic testing sponsored by pharmaceutical companies, clinical assessments, and diverse diagnostic tests.
Efficacy and Safety of Intravenous YOLT-201 for Transthyretin Amyloidosis Cardiomyopathy
This study is a single-arm, open-label, single-dose escalation trial aimed at evaluating the safety and tolerability of YOLT-201 treatment in patients with Transthyretin Amyloidosis Cardiomyopathy （ATTR-CM）, as well as assessing the preliminary efficacy of subjects treated with YOLT-201.