A Study of an Investigational Drug, ALN-AAT, in Healthy Adult Subjects and Patients With ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease
This trial is active, not recruiting.
|Condition||antitrypsin deficiency liver disease|
|Treatments||aln-aat, sterile normal saline (0.9% nacl)|
|Phase||phase 1/phase 2|
|Start date||July 2015|
|End date||January 2017|
|Trial size||66 participants|
|Trial identifier||NCT02503683, ALN-AAT-001|
The purpose of this study is to evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Subcutaneously Administered ALN-AAT in Healthy Adult Subjects and Patients with ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease.
|Endpoint classification||safety study|
|Intervention model||parallel assignment|
|Masking||single blind (subject)|
The safety of ALN-AAT evaluated by the proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs), and AEs leading to study drug discontinuation
time frame: Part A (SAD phase): through day 70; Part B (MAD) phase: through Day 154; Part C: through Day 224
Profile of Pharmacokinetics (PK) of ALN-AAT
time frame: Part A (SAD) phase: up to 21 days; Part B (MAD) phase: up to 105 days; Part C: up to 161 days
The effect of ALN-AAT on serum levels of AAT protein
time frame: Part A (SAD) phase: through day 70; Part B (MAD) phase: through Day 154; Part C: through Day 224
Male or female participants from 18 years up to 65 years old.
Inclusion Criteria: - Adequate complete blood counts, liver and renal function. - 12-lead electrocardiogram (ECG) within normal limits - Female subjects must be of non-childbearing potential; e.g. post-menopausal or pre-menopausal with surgical sterilization - Male subjects agree to use appropriate contraception - Willing to provide written informed consent and willing to comply with study requirements - Nonsmokers for at least 5 years before screening Exclusion Criteria: - Any uncontrolled or serious disease, or any medical or surgical condition, that may interfere with participation in the clinical study and/or put the subject at significant risk - Received an investigational agent within 90 days before the first dose of study drug or are in follow-up of another clinical study - Active serious mental illness or psychiatric disorder requiring current pharmacological intervention - History or evidence of alcohol or drug abuse within 12 months before screening. - History of intolerance to SC injection
|Official title||A Phase 1/2, Randomized, Single-blind, Placebo-Controlled, Single Ascending Dose and Multiple Dose, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Subcutaneously Administered ALN-AAT in Healthy Adult Subjects and Patients With ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease|
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