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

NCT ID: NCT05146882

Last Updated: 2024-09-19

Basic Information

• Recruitment Status: WITHDRAWN
• Clinical Phase: PHASE2
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-12-31
• Study Completion Date: 2022-05-23

Brief 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

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: OTHER
• Blinding Strategy: NONE

Study Groups

belcesiran

Participants who completed the DCR-A1AT-201 treatment period will receive open-label belcesiran administered subcutaneously

Group Type: EXPERIMENTAL

Belcesiran

Intervention Type: DRUG

Belcesiran will be administered subcutaneously (SC) in the treatment arm.

Observational

Participants who completed the DCR-A1AT-201 Conditional Follow-up period will enter DCR-A1AT-202 for continued follow-up (will not receive open-label belcesiran)

Group Type: NO_INTERVENTION

No interventions assigned to this group

Interventions

Belcesiran

Belcesiran will be administered subcutaneously (SC) in the treatment arm.

Intervention Type: DRUG

Eligibility 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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Dicerna Pharmaceuticals, Inc., a Novo Nordisk company

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Anne-Sophie Sejling, MD

Role: STUDY_DIRECTOR

Dicerna Pharmaceuticals

Locations

Auckland Clinical Studies

Grafton, Auckland, New Zealand

Countries

New Zealand

Other Identifiers

STARLIGHT

Identifier Type: OTHER

Identifier Source: secondary_id

DCR-A1AT-202

Identifier Type: -

Identifier Source: org_study_id