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

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

Status

Terminated

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT02503683

Enrollment

Registered

2015-07-21

Start date

2015-07-09

Completion date

2018-01-03

Last updated

2019-01-07

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Antitrypsin Deficiency Liver Disease

Keywords

RNAi therapeutic, AAT, Alpha-1 Antitrypsin Deficiency

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.

Interventions

DRUGALN-AAT

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

DRUGSterile Normal Saline (0.9% NaCl)

calculated volume to match active comparator

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

SINGLE (Subject)

Eligibility

Sex/Gender

ALL

Age

18 Years to 65 Years

Healthy volunteers

Yes

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

Design outcomes

Primary

Measure	Time frame
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	Part A (SAD phase): through day 70; Part B (MAD) phase: through Day 154; Part C: through Day 224

Secondary

Measure	Time frame	Description
Profile of Pharmacokinetics (PK) of ALN-AAT	Part A (SAD) phase: up to 21 days; Part B (MAD) phase: up to 105 days; Part C: up to 161 days	Cmax, tmax, AUC, t1/2
The effect of ALN-AAT on serum levels of AAT protein	Part A (SAD) phase: through day 70; Part B (MAD) phase: through Day 154; Part C: through Day 224	—

Countries

United Kingdom

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026