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Phase 3 study of inotersen in hereditary transthyretin-mediated amyloid polyneuropathy
2019-001698-10Phase 3Unknown
- Sponsor
- Ionis Pharmaceuticals
- Start
- Oct 2019
- Modality
- Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 1
01
Overview
Phase 3, randomised, open-label trial in familial amyloid polyneuropathy (FAP) comparing Inotersen with Historical placebo control from NEURO-TTR (2 arms). Primary endpoints: Interim Analysis Co-Primary Efficacy Endpoints at Week 35, Change from baseline in serum transthyretin concentration and 2 more. Status: unknown.
02
Drugs
inotersen
03
Conditions
familial amyloid polyneuropathy (FAP)
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