Plain-English translation of NCT04136184 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Read our Hereditary ATTR amyloidosis research guide →Phase 3 — Testing in thousands of people, comparing the treatment against what doctors currently use. This is the last big step before approval.
This trial tested a medication called in people with hereditary transthyretin-mediated amyloid polyneuropathy, a rare genetic condition that damages nerves over time. Researchers compared —given as an injection under the skin every 4 weeks—against , a medication that was injected weekly. The trial studied whether this treatment could slow or improve nerve damage in about 168 patients.
Hereditary transthyretin-mediated amyloid polyneuropathy is a serious genetic disease with limited treatment options. Researchers wanted to test whether the new medication could offer patients a more convenient treatment option that requires fewer injections while still being effective and safe.
Participants received injections under the skin either once a week or once every 4 weeks, depending on which treatment group they were assigned to, for up to 81 weeks. All participants also took daily vitamin A supplements. Those who started on the weekly injection medication were switched to the newer medication (given every 4 weeks) partway through the trial. Participants attended regular clinic visits for monitoring, blood tests, and assessments of their nerve function and symptoms.
AI-generated summary from trial data · Oct 12, 2026 · Not medical advice
United States