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

Alpha1-antitrypsin Deficiency Clinical Trial

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02282527
• Other study ID #: GTI1402
• Status: Completed
• Phase: Phase 2/Phase 3
• First received: October 31, 2014
• Last updated: January 29, 2016
• Start date: October 2014
• Est. completion date: January 2016

Study information

• Verified date: January 2016
• Source: Grifols Therapeutics Inc.
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

Grifols Therapeutics Inc. is conducting 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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 32
• Est. completion date: January 2016
• Est. primary completion date: January 2016
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 70 Years

Eligibility

Inclusion Criteria:

• Be between 18 and 70 years of age, inclusive

• Have a diagnosis of congenital AATD

• Have a documented total alpha1-PI level < 11 µM. If the total alpha1-PI level has yet to be documented, a blood draw for total alpha1-PI level will be obtained at the Screening Visit

• Have 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 has received alpha1-PI augmentation therapy of any kind, he/she must 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 has 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 are 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

Study Design

Allocation: Randomized, Endpoint Classification: Pharmacokinetics Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Alpha 1-Antitrypsin Deficiency
• Alpha1-antitrypsin Deficiency

Intervention

Biological:
• Liquid Alpha1-PI
Liquid Alpha1-PI, 60 mg/kg, 8-weekly intravenous infusion
• Prolastin-C
Prolastin-C, 60 mg/kg, 8-weekly intravenous infusion

Locations

Country	Name	City	State
United States	National Jewish Health	Denver	Colorado
United States	University of Florida Gainesville	Gainesville	Florida
United States	University of Miami - Miller School of Medicine	Miami	Florida
United States	Oregon Health and Science University	Portland	Oregon
United States	University of Texas Health Science Center	Tyler	Texas
United States	PMG Research of Wilmington	Wilmington	North Carolina

Sponsors (1)

Lead Sponsor	Collaborator
Grifols Therapeutics Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Area under the concentration vs. time curve (AUC)		7 days	No
Secondary	Adverse events (AEs)	Subjects will be monitored for AEs	20 weeks	Yes