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A Study of Belcesiran in Patients With AATLD

NCT04764448 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 16

Last updated 2026-04-07

Study results available
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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 AATD-associated liver disease (AATLD).

The study will be conducted in 3 separate cohorts. A total of up to 16 participants may be enrolled in Cohort 1 and 2. A total number of 30 subjects will be enrolled in cohort 3. The 3 cohorts are differentiated by the duration of the treatment period, the number of doses administered, and the timing of the second liver biopsy.

Conditions

Interventions

DRUG

Belcesiran

Administered multiple fixed doses of belcesiran by subcutaneous (sc) injection for 24 weeks. Extension offered to participants.

OTHER

Placebo

Comparator: Placebo Cohort 1 Administered sterile normal saline (0.9% NaCl) matching volume of belcesiran by subcutaneous (sc) injection for 24 weeks. Extension offered to participants.

DRUG

Belcesiran

Administered multiple fixed doses of belcesiran by subcutaneous (sc) injection for 48 weeks. Extension offered to participants.

OTHER

Placebo

Administered sterile normal saline (0.9% NaCl) matching volumes of belcesiran by subcutaneous (sc) injection for 48 weeks. Extension offered to participants.

DRUG

Belcesiran

Administered multiple fixed doses of belcesiran by subcutaneous (sc) injection for 96 weeks.

OTHER

Placebo

Administered sterile normal saline (0.9% NaCl) matching volumes of belcesiran by subcutaneous (sc) injection for 96 weeks.

Sponsors & Collaborators

  • Dicerna Pharmaceuticals, Inc., a Novo Nordisk company

    lead INDUSTRY

Principal Investigators

  • Thomas Bowman, MD · Dicerna Pharmaceuticals / Novo Nordisk

Study Design

Allocation
RANDOMIZED
Purpose
OTHER
Masking
TRIPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
75 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-02-12
Primary Completion
2023-12-08
Completion
2024-05-29
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Austria
  • Belgium
  • Canada
  • France
  • Germany
  • Ireland
  • Netherlands
  • New Zealand
  • Portugal
  • Spain
  • Sweden
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04764448 on ClinicalTrials.gov