Alpha 1-Antitrypsin Deficiency
Conditions
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.
Interventions
DRUGBelcesiran
Belcesiran will be administered subcutaneously (SC) in the treatment arm.
Sponsors
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
OTHER
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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 incidence of treatment-emergent adverse events | up to 152 weeks | — |
| 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 | Forced vital capacity (FVC) |
| 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 | RR interval |
| The change from baseline in physical examination (PE) findings | up to 56 weeks | body weight |
| The change from baseline in PE findings | up to 56 weeks | body-mass index (BMI) (using height from DCR-A1AT-201 study) |
| The change from baseline in vital sign measurements | up to 56 weeks | blood pressure |
| 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: Clinical Chemistry | up to 152 weeks | Clinical Chemistry 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 |
Secondary
| Measure | Time frame |
|---|---|
| Changes in serum AAT protein concentrations over time | up to 152 weeks |
Countries
New Zealand
Outcome results
None listed