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Safety and PK/PD of Sapablursen in Healthy Volunteers
NCT03165864Phase 1Completed
- Sponsor
- Ionis Pharmaceuticals
- Start
- May 2017
- Primary completion
- Sep 2018
- Modality
- Gene Editing, Gene Silencing, Small Interfering RNA (siRNA)
- Countries
- 1
01
Overview
Phase 1, randomised, double-blind trial in beta thalassemia comparing Sapablursen with Placebo (2 arms). Primary endpoint: Adverse Events up to 148 Days. Started May 2017; status: completed.
02
Drugs
sapablursen
03
Conditions
beta thalassemia
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