ARALAST NP Alpha-1 Lung Density Chronic Obstructive Pulmonary Disease-Emphysema (COPD-E) Study

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Brief Title

ARALAST NP Alpha-1 Lung Density Chronic Obstructive Pulmonary Disease-Emphysema (COPD-E) Study

Official Title

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)

Brief Summary

      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.
    


Study Phase

Phase 4

Study Type

Interventional


Primary Outcome

Annual Rate of the Physiologically Adjusted Lung Density Change

Secondary Outcome

 Number of Moderate or Severe Exacerbations of Chronic Obstructive Pulmonary Disease (COPD)

Condition

Chronic Obstructive Pulmonary Disease

Intervention

ARALAST NP

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.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Biological

Estimated Enrollment

168

Start Date

March 8, 2021

Completion Date

September 12, 2025

Primary Completion Date

September 12, 2025

Eligibility Criteria

        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
      

Gender

All

Ages

18 Years - 65 Years

Accepts Healthy Volunteers

No

Contacts

Study Director, +1 866 842 5335, ClinicalTransparency@shire.com



Administrative Informations


NCT ID

NCT04440488

Organization ID

TAK-883-3001


Responsible Party

Sponsor

Study Sponsor

Shire


Study Sponsor

Study Director, Study Director, Shire


Verification Date

June 2020