ARALAST NP Alpha-1 Lung Density Chronic Obstructive Pulmonary Disease-Emphysema (COPD-E) Study
A Prospective, Randomized, Double-Blind, Parallel Group Study to Evaluate the Safety and Efficacy of ARALAST NP 60 mg/kg and 120 mg/kg for Alpha-1 Proteinase Inhibitor (A1PI) Augmentation Therapy in Subjects With A1PI Deficiency and Chronic Obstructive Pulmonary Disease-Emphysema (COPD-E)
The purpose of this study is to evaluate the efficacy of ARALAST NP A1PI augmentation therapy 120 milligrams per kilogram (mg/kg) body weight (BW)/week compared with an external placebo comparator on the loss of emphysematous lung tissue measured by lung density change in participants with A1PI deficiency and COPD-E.
Annual Rate of the Physiologically Adjusted Lung Density Change
Number of Moderate or Severe Exacerbations of Chronic Obstructive Pulmonary Disease (COPD)
Chronic Obstructive Pulmonary Disease
Study Arms / Comparison Groups
ARALAST NP 120 mg/kg
Description: Participants will receive 120 mg/kg BW of ARALAST NP intravenous (IV) infusion once in a week for a total of 104 weeks which will be compared with an external placebo arm.
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
March 8, 2021
September 12, 2025
Primary Completion Date
September 12, 2025
Inclusion Criteria: - Adults 18 to 65 years of age at the time of screening - Diagnosis of A1PI deficiency with endogenous plasma A1PI level less than (<) 11 micromoles(μM) (< 0598 milligram per milliliter [mg/mL]) following 4-week minimum washout from previous last dose of A1PI augmentation therapy The screening plasma A1PI level may be repeated if a participant is determined to have an exclusionary value that maybe due to an inadequate washout of augmentation therapy Participants eligible for enrollment include newly diagnosed, previously untreated, currently treated and currently not on treatment but received treatment in the past - Participants should have a documented A1PI genotype and if not, A1PI genotyping will be offered at the time of screening The purpose for genotyping is for sub-group analysis of study results only - Clinically evident COPD-E (according to GOLD criteria (2020) for diagnosis, Stage I-III) (Global Initiative for Chronic Obstructive Lung Disease [COPD]) at the time of screening defined as follows: Forced expiratory volume in 1 second (FEV1) is greater than or equal to (>or=) 35% and less than or equal to (
28 days) of parenteral corticosteroids or oral corticosteroids at doses greater than 10 mg/day of prednisone or its equivalent - Receiving chronic 24 hours/day oxygen supplementation (other than for an acute COPD exacerbation, or supplemental oxygen with continuous positive airway pressure [CPAP], or bi-level positive airway pressure [BiPAP] for acute respiratory failure) - Known selective immunoglobulin A (IgA) deficiency (IgA level < 7 milligrams per deciliter [mg/dL] at screening) with anti-IgA antibodies and a history of hypersensitivity reaction - Known history of hypersensitivity following infusions of human immunoglobulins, human albumin, blood or blood components - Presence of clinically significant laboratory abnormalities at the screening that in the opinion of the investigator would impact the participant's safety, if enrolled in the study - Presence of any of the following that in the opinion of the investigator, would affect participant's safety or compliance or confound the results of the study, including known clinically significant medical, psychiatric, or cognitive illness, is a recreational drug/alcohol user, or has any other uncontrolled medical condition (eg, unstable angina, transient ischemic attack, uncontrolled hypertension) - Known exposure to another IP within 28 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study - Participant is a family member or employee of the investigator - If female, participant is pregnant or nursing at the time of enrollment
18 Years - 65 Years
Accepts Healthy Volunteers
Study Director, ,
Study Director, Study Director, Shire