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

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Grifols

Status and phase

Completed
Phase 3
Phase 2

Conditions

Alpha₁-Antitrypsin Deficiency

Treatments

Biological: Prolastin-C
Biological: Liquid Alpha₁-PI

Study type

Interventional

Funder types

Industry

Identifiers

NCT02282527
GTI1402

Details and patient eligibility

About

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).

Enrollment

32 patients

Sex

All

Ages

18 to 70 years old

Volunteers

No Healthy Volunteers

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

Trial design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Crossover Assignment

Masking

Quadruple Blind

32 participants in 2 patient groups

Treatment Sequence 1
Other group
Description:
Subjects were treated first with Liquid Alpha₁-PI and then treated with Prolastin-C
Treatment:
Biological: Liquid Alpha₁-PI
Biological: Prolastin-C
Treatment Sequence 2
Other group
Description:
Subjects were treated first with Prolastin-C and then treated with Liquid Alpha₁-PI
Treatment:
Biological: Liquid Alpha₁-PI
Biological: Prolastin-C

Trial contacts and locations

6

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Data sourced from clinicaltrials.gov

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