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Phase 2 Randomized Double-blind Placebo-controlled Study of Belcesiran in AATLD
2020-003313-35Phase 2Completed
- Sponsor
- Dicerna Pharmaceuticals, Inc.
- Start
- Nov 2020
- Modality
- Gene Therapies, Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 1
01
Overview
Phase 2, randomised, double-blind trial in alpha-1 antitrypsin deficiency (AATD) comparing Belcesiran with Placebo (2 arms). Primary endpoint: Primary safety and tolerability. Started Nov 2020; status: completed.
02
Drugs
belcesiran
03
Conditions
alpha-1 antitrypsin deficiency (AATD)
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