A Study of Belcesiran in Patients With A1ATD-Associated Liver Disease

• Status: Recruiting
• Trial end date: 2023-12-01
• Target enrollment: 0
• Participant gender: All

Summary

This is a multiple dose, randomized, placebo-controlled, double-blind study of belcesiran to evaluate the safety, tolerability, PK, and PD in adult patients with PiZZ A1ATD-associated liver disease.

The study will be conducted in 2 parallel cohorts. A total of up to 27 participants may be enrolled in a single cohort. The 2 cohorts are differentiated only by the duration of the treatment period, the number of doses administered, and the timing of the end-of-treatment (EOT) liver biopsy. Participants in Cohort 1 will undergo EOT biopsy at 24 weeks, and participants in Cohort 2 will undergo EOT biopsy at 48 weeks.

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

Details

• Lead Sponsor: Dicerna Pharmaceuticals, Inc.

Criteria

Inclusion Criteria:

• 18 to 70 years, inclusive. Female participants must be either surgically sterile or postmenopausal. No women of childbearing potential are eligible for enrollment.

• Documented diagnosis of PiZZ-type A1ATD, confirmed by genotyping. Historical genotyping data may be used, if available.

• A1ATD-associated liver disease documented by liver biopsy.

• Consent to undergo paired liver biopsies, one at Screening and one at either 24 weeks or 48 weeks of the first dose of the study intervention

• Lung, renal and liver function within acceptable limits

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

Exclusion Criteria:

• Presence of any condition or comorbidities that would interfere with study compliance or data interpretation or potentially affect patient safety including, but not restricted to:

• Severe intercurrent illness.

• History of chronic liver disease from any cause other than PiZZ-type A1ATD,

• A1AT augmentation therapy in the 6 months prior to Screening

• Routine use of acetaminophen/paracetamol

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

• Participation in any clinical study in which they received an IMP within 4 months before Screening