Clinical trial NCT05146882: An Extension Study of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATLD)
Official title: A Phase 2 Open-Label Extension Study to Evaluate the Safety and Pharmacodynamics of Belcesiran in Patients With PiZZ Alpha-1 Antitrypsin Deficiency Associated Liver Disease
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE2
Conditions: Alpha 1-Antitrypsin Deficiency
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2021-12-07
Enrollment: 0
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: OTHER

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.


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

Primary outcomes:
measure: The incidence of treatment-emergent adverse events
timeFrame: up to 152 weeks
measure: The change from baseline in pulmonary function tests (PFTs)
timeFrame: up to 152 weeks
measure: The change from baseline in PFTs
timeFrame: up to 152 weeks
measure: The change from baseline in PFTs
timeFrame: up to 152 weeks
measure: The change from baseline in PFTs
timeFrame: up to 152 weeks
measure: The change from baseline in 12-lead electrocardiogram (ECG)
timeFrame: up to 56 weeks
measure: The change from baseline in ECG
timeFrame: up to 56 weeks
measure: The change from baseline in 12-lead ECG
timeFrame: up to 56 weeks
measure: The change from baseline in 12-lead ECG
timeFrame: up to 56 weeks
measure: The change from baseline in 12-lead ECG
timeFrame: up to 56 weeks
measure: The change from baseline in 12-lead ECG
timeFrame: up to 56 weeks
measure: The change from baseline in 12-lead ECG
timeFrame: up to 56 weeks
measure: The change from baseline in physical examination (PE) findings
timeFrame: up to 56 weeks
measure: The change from baseline in PE findings
timeFrame: up to 56 weeks
measure: The change from baseline in PE findings
timeFrame: up to 56 weeks
measure: The change from baseline in vital sign measurements
timeFrame: up to 56 weeks
measure: The change from baseline in vital sign measurements
timeFrame: up to 56 weeks
measure: The change from baseline in vital sign measurements
timeFrame: up to 56 weeks
measure: The change from baseline in vital sign measurements
timeFrame: up to 56 weeks
measure: The change from baseline in clinical laboratory tests: Hematology
timeFrame: up to 152 weeks
measure: The change from baseline in clinical laboratory tests: Clinical Chemistry
timeFrame: up to 152 weeks
measure: The change from baseline in clinical laboratory tests: Coagulation
timeFrame: up to 152 weeks
measure: The change from baseline in clinical laboratory tests: Urinalysis
timeFrame: up to 152 weeks
Secondary outcomes:
measure: Changes in serum AAT protein concentrations over time
timeFrame: up to 152 weeks
Other outcomes:
Locations:
city: Grafton
country: New Zealand
facility: Auckland Clinical Studies
state: Auckland
Study officials:
affiliation: Dicerna Pharmaceuticals
name: Anne-Sophie Sejling, MD
role: STUDY_DIRECTOR
References: