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

NCT ID: NCT02503683

Last Updated: 2019-01-07

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE1
• Total Enrollment: 26 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-07-09
• Study Completion Date: 2018-01-03

Brief 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.

Conditions

Antitrypsin Deficiency Liver Disease

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: SINGLE

Study Groups

ALN-AAT

Group Type: ACTIVE_COMPARATOR

ALN-AAT

Intervention Type: DRUG

Single or multiple doses of ALN-AAT by subcutaneous (sc) injection

Sterile Normal Saline (0.9% NaCl)

Group Type: PLACEBO_COMPARATOR

Sterile Normal Saline (0.9% NaCl)

Intervention Type: DRUG

calculated volume to match active comparator

Interventions

ALN-AAT

Single or multiple doses of ALN-AAT by subcutaneous (sc) injection

Intervention Type: DRUG

Sterile Normal Saline (0.9% NaCl)

calculated volume to match active comparator

Intervention Type: DRUG

Eligibility 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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

Alnylam Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Patrick Haslett, MD

Role: STUDY_DIRECTOR

Alnylam Pharmaceuticals

Locations

Richmond Pharmacology, Ltd.

London, , United Kingdom

Countries

United Kingdom

Other Identifiers

ALN-AAT-001

Identifier Type: -

Identifier Source: org_study_id