Plain-English translation of NCT02510261 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 , an investigational medication, in people who had already completed earlier studies of this treatment for a hereditary nerve disease called familial amyloidotic polyneuropathy. Researchers wanted to see how safe and effective the medication remained when given over a longer period of time. Participants continued receiving the same doses they had been getting, given as an infusion into the vein every three weeks.
Early studies showed promise for this medication in treating this serious hereditary nerve condition, but researchers needed to understand whether it would continue to work safely and effectively over many years of ongoing treatment.
Participants who joined this trial had already been in earlier studies. They continued receiving the same medication dose—0.3 milligrams per kilogram—as an infusion into a vein once every three weeks. Depending on which earlier study they came from, they received this treatment for between 61 and 66 months (about 5 to 5.5 years) while researchers monitored their safety and how well the medication worked over this extended period.
AI-generated summary from trial data · Sep 24, 2026 · Not medical advice
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