NCT05146882

An Extension Study of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATLD)

A Phase 2 Open-Label Extension Study to Evaluate the Safety and Pharmacodynamics of Belcesiran in Patients With PiZZ Alpha-1 Antitrypsin Deficiency Associated Liver Disease

• Status: Withdrawn
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Dicerna Pharmaceuticals, Inc., a Novo Nordisk company · Industry
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase 2, multicenter, open-label extension of Study DCR-A1AT-201, designed to evaluate the long-term safety and further characterize the pharmacodynamics (PD) of belcesiran in adult patients with PiZZ AATLD.

Conditions

• Alpha 1-Antitrypsin Deficiency

Interventions

Type	Name	Description
DRUG	Belcesiran	Belcesiran will be administered subcutaneously (SC) in the treatment arm.

Timeline

• Start date: 2021-12-01
• Primary completion: 2022-05-23
• Completion: 2022-05-23
• First posted: 2021-12-07
• Last updated: 2024-09-19

Locations

1 site across 1 country: New Zealand