A Study to Assess Safety and PK of Liquid Alpha₁-Proteinase Inhibitor (Human) in Treating Alpha₁-Antitrypsin Deficiency

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Brief Title

A Study to Assess Safety and PK of Liquid Alpha₁-Proteinase Inhibitor (Human) in Treating Alpha₁-Antitrypsin Deficiency

Official Title

A Multi-center, Randomized, Double-blind, Crossover Study to Assess the Safety and Pharmacokinetics of Liquid Alpha₁-Proteinase Inhibitor (Human) Compared to Prolastin®-C in Subjects With Alpha₁-Antitrypsin Deficiency

Brief Summary

      Grifols Therapeutics Inc. conducted a multi-center, randomized, double-blind, crossover study
      to evaluate the safety, immunogenicity, and pharmacokinetics (PK) of Liquid Alpha₁-PI
      compared to the currently licensed product, Prolastin-C, in subjects with Alpha₁-Antitrypsin
      Deficiency (AATD).
    


Study Phase

Phase 2/Phase 3

Study Type

Interventional


Primary Outcome

AUC(0-7 Days) Based on Antigenic Content

Secondary Outcome

 AUC(0-7 Days) Based on Functional Activity

Condition

Alpha₁-Antitrypsin Deficiency

Intervention

Liquid Alpha₁-PI

Study Arms / Comparison Groups

 Treatment Sequence 1
Description:  Subjects were treated first with Liquid Alpha₁-PI and then treated with Prolastin-C

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

32

Start Date

October 2014

Completion Date

January 2016

Primary Completion Date

January 2016

Eligibility Criteria

        Inclusion Criteria:

          -  Be between 18 and 70 years of age, inclusive

          -  Had a diagnosis of congenital AATD

          -  Had a documented total alpha₁-PI level < 11 µM. If the total alpha₁-PI level had yet
             to be documented, a blood draw for total alpha₁-PI level was obtained at the Screening
             Visit

          -  Had a post-bronchodilator Forced expiratory volume in 1 second (FEV1) ≥ 30% and < 80%
             of predicted and FEV1/forced vital capacity (FVC) < 70%

          -  If the subject had received alpha₁-PI augmentation therapy of any kind, he/she must
             have been be willing to discontinue that treatment at the Week 1 (Baseline) Visit and
             remain off any kind of alpha₁-PI treatment, other than the investigational products
             for this study, while participating in the study

        Exclusion Criteria:

          -  Subject had a moderate or severe pulmonary exacerbation during the 4 weeks before the
             Week 1 (Baseline) Visit

          -  History of lung or liver transplant

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

          -  Liver cirrhosis confirmed by biopsy

          -  Elevated liver enzymes (aspartate transaminase [AST], alanine aminotransferase [ALT],
             and alkaline phosphatase [ALP]) equal to or greater than 2.5 times the upper limit of
             normal

          -  Severe concomitant disease (e.g., congestive heart failure, clinically significant
             pulmonary fibrosis, malignant disease [with the exception of skin cancers other than
             melanoma], history of acute hypersensitivity pneumonitis reaction, or current chronic
             hypersensitivity pneumonitis)

          -  Females who were pregnant, breastfeeding or, if of child-bearing potential, unwilling
             to practice a highly effective method of contraception (oral, injectable or implanted
             hormonal methods of contraception, placement of an intrauterine device (IUD) or
             intrauterine system (IUS), condom or occlusive cap with spermicidal
             foam/gel/film/cream/suppository, male sterilization, or abstinence) throughout the
             study

          -  Known previous infection with or clinical signs and symptoms consistent with current
             hepatitis A virus (HAV), hepatitis B virus (HBV), hepatitis C virus (HCV), or human
             immunodeficiency virus (HIV) infection

          -  Smoking during the past 6 months or a positive urine cotinine test at the Screening
             Visit that is due to smoking

          -  Participation in another investigational drug study within one month prior to the Week
             1 (Baseline) Visit

          -  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 4 weeks prior to the Week 1 (Baseline) Visit
             inhaled steroids are not considered systemic steroids)

          -  Use of systemic or aerosolized antibiotics for an exacerbation within the 4 weeks
             prior to the Week 1 (Baseline) Visit

          -  Known selective or severe Immunoglobulin A (IgA) deficiency
      

Gender

All

Ages

18 Years - 70 Years

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT02282527

Organization ID

GTI1402


Responsible Party

Sponsor

Study Sponsor

Grifols Therapeutics LLC


Study Sponsor

, , 


Verification Date

January 2017