Status:
TERMINATED
A Study of ALN-AAT02 in Healthy Participants and Participants With ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease
Lead Sponsor:
Alnylam Pharmaceuticals
Conditions:
ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease
Eligibility:
All Genders
18-65 years
Phase:
PHASE1
PHASE2
Brief Summary
The purpose of this study is to evaluate the safety and tolerability of single or multiple doses of ALN-AAT02. The study will be conducted in 2 sequential phases in which Part A will be a single-ascen...
Eligibility Criteria
Inclusion
- Male or female, aged 18 to 65 years, inclusive;
- Has normal 12-lead electrocardiogram (ECG);
- Has body mass index (BMI) between 18 and 30 kg/m\^2, inclusive;
- Has been a nonsmoker for at least 5 years before screening;
- Part A only: Has Alpha-1 antitrypsin (AAT) levels within normal limits;
- Part A only: Has adequate Forced Expiratory Volume in 1 second (FEV1) and adequate FEV1/forced vital capacity ratio;
- Part B only: Has documented ZZ type AAT by genotype;
- Part B only: Has liver biopsy within 90 days of the first dose of study drug demonstrating ZZ type alpha-1 antitrypsin deficiency (PiZZ AATD) liver disease;
- Part B only: Has adequate post-bronchodilator FEV1 and adequate diffusing capacity of the lung for carbon monoxide;
- Part B only: If on any maintenance medication, is likely to be able to remain on a stable medication regimen for the duration of the study (no new medications within 30 days prior to first dose of study drug).
Exclusion
- Has known human immunodeficiency virus (HIV), hepatitis C virus (HCV) or hepatitis B virus (HBV) infection;
- Has clinically significant abnormal laboratory results;
- Received an experimental drug within 30 days of dosing;
- Has a history of multiple drug allergies or history of allergic reaction to an oligonucleotide or N-acetylgalactosamine (GalNAc);
- Part A only: Has estimated glomerular filtration equal to or below 60 mL/min/1.73 m\^2 at screening;
- Part A only: Has a history of asthma or recurrent or chronic lung disease, excluding resolved childhood asthma;
- Part A only: Has a history of chronic liver disease;
- Part B only: Has estimated glomerular filtration equal to or below 45 mL/min/1.73 m\^2 at screening;
- Part B only: Received an augmentation therapy for AAT deficiency within 8 weeks of first dose of study drug;
- Part B only: Has a history of chronic liver disease from any known cause other than ZZ type AAT deficiency;
- Part B only: Has a history of hepatic encephalopathy;
- Part B only: Has a history of gastrointestinal bleeding or ascites.
Key Trial Info
Start Date :
December 5 2018
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 25 2020
Estimated Enrollment :
32 Patients enrolled
Trial Details
Trial ID
NCT03767829
Start Date
December 5 2018
End Date
June 25 2020
Last Update
April 27 2021
Active Locations (1)
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1
Clinical Trial Site
London, United Kingdom