An Extension Study of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATLD)
- Registration Number
- NCT05146882
- Lead Sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- WITHDRAWN
- Sex
- All
- Target Recruitment
- Not specified
- Must be 18 to 75 years of age inclusive, at the time of signing the Informed Consent Form (ICF).
- Documented diagnosis of PiZZ-type Alpha-1 Antitrypsin deficiency (AATD), confirmed by genotyping.
- 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.
- 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
- Routine use of acetaminophen/paracetamol
- Use of systemically acting steroids in the month prior to Screening and throughout the study period.
- Positive SARS-CoV-2 virus test at Screening
- Any other safety laboratory test result considered clinically significant and unacceptable by the Investigator
- Inability or unwillingness to comply with the specified study procedures, including lifestyle considerations
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description belcesiran Belcesiran Participants who completed the DCR-A1AT-201 treatment period will receive open-label belcesiran administered subcutaneously
- Primary Outcome Measures
Name Time Method The change from baseline in physical examination (PE) findings up to 56 weeks body weight
The change from baseline in clinical laboratory tests: Clinical Chemistry up to 152 weeks Clinical Chemistry is collected to evaluate the long-term safety of belcesiran
The incidence of treatment-emergent adverse events up to 152 weeks The change from baseline in 12-lead electrocardiogram (ECG) up to 56 weeks heart rate
The change from baseline in ECG up to 56 weeks ventricular rate
The change from baseline in 12-lead ECG up to 56 weeks corrected QT interval (QTcF, Fridericia correction)
The change from baseline in PE findings up to 56 weeks physical examination to assess skin, lungs, cardiovascular system, and abdomen (liver and spleen) based on the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE; version 5.0) grading scale
The change from baseline in clinical laboratory tests: Hematology up to 152 weeks Hematology is collected to evaluate the long-term safety of belcesiran
The change from baseline in clinical laboratory tests: Coagulation up to 152 weeks Coagulation is collected to evaluate the long-term safety of belcesiran
The change from baseline in vital sign measurements up to 56 weeks oral temperature
The change from baseline in clinical laboratory tests: Urinalysis up to 152 weeks Urinalysis is collected to evaluate the long-term safety of belcesiran
The change from baseline in pulmonary function tests (PFTs) up to 152 weeks Forced expiratory volume in 1 second (FEV1)
The change from baseline in PFTs up to 152 weeks diffusing capacity for carbon monoxide (DLCO)
- Secondary Outcome Measures
Name Time Method Changes in serum AAT protein concentrations over time up to 152 weeks
Trial Locations
- Locations (1)
Auckland Clinical Studies
🇳🇿Grafton, Auckland, New Zealand
Auckland Clinical Studies🇳🇿Grafton, Auckland, New Zealand