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

Dicerna Pharmaceuticals

Status and phase: Withdrawn; Phase 2

Conditions: Alpha 1-Antitrypsin Deficiency

Treatments: Drug: Belcesiran

Study type: Interventional

Funder types: Industry

Identifiers

NCT05146882

STARLIGHT (Other Identifier)

DCR-A1AT-202

Details and patient eligibility

About

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.

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Other

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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0 participants in 2 patient groups

belcesiran

Experimental group

Description:

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

Treatment:

Drug: Belcesiran

Observational

No Intervention group

Description:

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)