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Phase 3 open-label randomized trial of ION-682884 vs inotersen in hereditary transthyretin-mediated amyloid polyneuropathy
jRCT2071210009Phase 3Completed
- Sponsor
- Ionis Pharmaceuticals
- Start
- Apr 2021
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
01
Overview
Phase 3, randomised, open-label trial of eplontersen in familial amyloid polyneuropathy (FAP) (2 arms), sponsored by Ionis Pharmaceuticals. Primary endpoints: Change from baseline in mNIS+7 at Week 66, Change from baseline in Norfolk Quality of Life Diabetic Neuropathy Questionnaire at Week 66 and 3 more. Started Apr 2021; status: completed.
02
Drugs
eplontersen
03
Conditions
familial amyloid polyneuropathy (FAP)
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