Overview

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
Sponsor Alnylam Pharmaceuticals
Start date July 2015
End date January 2017
Trial size 66 participants
Trial identifier NCT02503683, ALN-AAT-001

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety study
Intervention model parallel assignment
Masking single blind (subject)
Primary purpose treatment
Arm
(Active Comparator)
aln-aat
Single or multiple doses of ALN-AAT by subcutaneous (sc) injection
(Placebo Comparator)
sterile normal saline (0.9% nacl)
calculated volume to match active comparator

Primary Outcomes

Measure
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

Secondary Outcomes

Measure
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

Eligibility Criteria

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

Additional Information

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
Trial information was received from ClinicalTrials.gov and was last updated in May 2016.
Information provided to ClinicalTrials.gov by Alnylam Pharmaceuticals.