Efficacy and Safety of Alpha1-Proteinase Inhibitor (Human), Modified Process (Alpha-1 MP) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin Deficiency (AATD)



Status:Recruiting
Conditions:Chronic Obstructive Pulmonary Disease, Pulmonary
Therapuetic Areas:Pulmonary / Respiratory Diseases
Healthy:No
Age Range:18 - 70
Updated:2/13/2019
Start Date:November 2013
End Date:November 2023
Contact:Susan Sorrells
Email:Susan.Sorrells@grifols.com

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A Randomized, Double-Blind, Placebo Controlled Study to Assess the Efficacy and Safety of Two Dose Regimens (60 mg/kg and 120 mg/kg) of Weekly Intravenous Alpha1 Proteinase Inhibitor (Human) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin Deficiency

This is a multi-center, randomized, placebo-controlled, double blind clinical study to assess
the efficacy and safety of two separate dose regimens of Alpha-1 MP versus placebo for 156
weeks (i.e., 3 years) using computed tomography (CT) of the lungs as the main measure of
efficacy. The two Alpha-1 MP doses to be tested are 60 mg/kg and 120 mg/kg administered
weekly by IV infusion for 156 weeks. The study consists of a Screening Phase, a 156-week
Treatment Phase, and an End of Study Visit at Week 160.


Inclusion Criteria:

- Have a documented total alpha1-PI serum level < 11 µM.

- Have a diagnosis of congenital AATD with an allelic combination of ZZ, SZ, Z(null),
(null)(null), S(null), or "at-risk" alleles.

- At the Screening (Week -3) Visit, have a post-bronchodilator forced expiratory volume
in 1 second (FEV1) ≥ 30% and < 80% of predicted and FEV1/forced vital capacity (FVC) <
70% (Global Initiative for Chronic Obstructive Lung Disease [GOLD] stage II or III).

- Have a carbon monoxide diffusing capacity (DLCO) ≤ 60% of predicted (corrected for
HgB) within the past 2 years OR evidence of pulmonary emphysema on CT scan within the
past 2 years per the Investigator's judgment.

- Have clinical evidence of pulmonary emphysema per the Investigator's judgment.

Exclusion Criteria:

- Has received alpha1-PI augmentation therapy for more than 1 month within the six
months prior to the Screening Visit.

- Has received alpha1-PI augmentation therapy within one month of the Screening Visit.

- Has had a chronic obstructive pulmonary disease (COPD) exacerbation within the 5 weeks
prior to the Screening Visit or during the Screening Phase.

- Unable to physically (e.g., unable to fit inside the CT scanner) or mentally (e.g.,
claustrophobic) undergo a CT scan.

- History of lung or liver transplant.

- Any lung surgery during the past 2 years (excluding lung biopsy).

- On the waiting list for lung surgery, including lung transplant.

- Smoking during the past 12 months or a positive urine cotinine test at screening that
is due to smoking. Maybe on Nicotine replacement, including vapor cigarettes.

- History of anaphylaxis or severe systemic response to any plasma-derived alpha1-PI
preparation or other blood product(s).

- Use of systemic steroids above a stable dose equivalent to 5 mg/day prednisone (i.e.,
10 mg every 2 days) within the 5 weeks prior to the Screening Visit (inhaled steroids
are not considered systemic steroids) or during the Screening Phase.

- Use of systemic or aerosolized antibiotics for a COPD exacerbation within the 5 weeks
prior to the Screening Visit or during the Screening Phase.

- Known selective or severe Immunoglobulin A (IgA) deficiency.
We found this trial at
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350 W Thomas Rd
Phoenix, Arizona 85013
(602) 406-3000
Principal Investigator: Rajat Walia, MD
St. Joseph's Hospital and Medical Center St. Joseph's is a nationally recognized center for quality...
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Gainesville, Florida 32610
(352) 392-3261
Principal Investigator: Mark Brantly, MD
University of Florida The University of Florida (UF) is a major, public, comprehensive, land-grant, research...
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1601 Northwest 12th Avenue
Miami, Florida 33136
(305) 243-6545
Principal Investigator: Michael Campos, MD
University of Miami Miller School of Medicine The University of Miami Leonard M. Miller School...
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3181 Southwest Sam Jackson Park Road
Portland, Oregon 97239
503 494-8311
Principal Investigator: Alan Barker, MD
Oregon Health and Science University In 1887, the inaugural class of the University of Oregon...
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171 Ashley Avenue
Charleston, South Carolina 29425
843-792-1414
Principal Investigator: Tatsiana Beiko, MD
Medical University of South Carolina The Medical University of South Carolina (MUSC) has grown from...
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Hershey, Pennsylvania 17033
Principal Investigator: Timothy Craig, DO
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660 S Euclid Ave
Saint Louis, Missouri 63110
(314) 362-5000
Washington University School of Medicine Washington University Physicians is the clinical practice of the School...
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Tyler, Texas 75708
Principal Investigator: James Stocks, MD
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Vicente Lopez, Buenos Aires
Principal Investigator: Mariano Fernandez Acquier
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Wilmington, North Carolina 28401
Principal Investigator: Mitchell D Lee, MD
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