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

Alnylam Pharmaceuticals

Status and phase

Terminated

Phase 1

Conditions

Antitrypsin Deficiency Liver Disease

Treatments

Drug: ALN-AAT

Drug: Sterile Normal Saline (0.9% NaCl)

Study type

Interventional

Funder types

Industry

Identifiers

NCT02503683

ALN-AAT-001

Details and patient eligibility

About

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.

Enrollment

26 patients

Sex

All

Ages

18 to 65 years old

Volunteers

Accepts Healthy Volunteers

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