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

• Status: Enrolling by invitation
• Trial end date: 2025-12-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Dicerna Pharmaceuticals, Inc.

Criteria

Inclusion Criteria:

1. Must be 18 to 75 years of age inclusive, at the time of signing the Informed Consent Form (ICF).

2. Documented diagnosis of PiZZ-type Alpha-1 Antitrypsin deficiency (AATD), confirmed by genotyping.

3. Lung, renal and liver function within acceptable limits.

4. Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.

Exclusion Criteria:

1. Any condition that, in the opinion of the Investigator, would make the participant unsuitable for enrollment or could interfere with participation in or completion of the study

2. Routine use of acetaminophen/paracetamol

3. Use of systemically acting steroids in the month prior to Screening and throughout the study period.

4. Positive SARS-CoV-2 virus test at Screening

5. Any other safety laboratory test result considered clinically significant and unacceptable by the Investigator

6. Inability or unwillingness to comply with the specified study procedures, including lifestyle considerations