A Study of an Investigational Drug, ALN-AAT, in Healthy Adult Subjects and Patients With ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease

• Status: Terminated
• Trial end date: 2018-01-03
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 1
• Accepts Healthy Volunteers?: Accepts Healthy Volunteers

Details

• Lead Sponsor: Alnylam Pharmaceuticals
• Treatments: Alpha 1-Antitrypsin; Protein C Inhibitor

Criteria

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