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