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

Related Clinical Trial
A Phase 1, First-in-human Study of VX-668 Study to Check the Safety of Fazirsiran and Learn if Fazirsiran Can Help People With Liver Disease and Scarring (Fibrosis) Due to an Abnormal Version of Alpha-1 Antitrypsin Protein A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype A Phase 1, First-in-human Study of VX-634 A Phase I of OsrhAAT in Healthy Volunteers Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of Disease An Extension Study of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATLD) Functional and Structural Lung Imaging in Chronic Obstructive Pulmonary Disease A Study of Belcesiran in Patients With A1ATD-Associated Liver Disease A Study to Evaluate Safety, Tolerability and Pharmacokinetics of Two Different Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% in Participants With Alpha1-Antitrypsin Deficiency Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype A Single Ascending and Repeated Dose Study of Oral ZF874 in Healthy Volunteers and PiMZ Subjects ARALAST NP Alpha-1 Lung Density Chronic Obstructive Pulmonary Disease-Emphysema (COPD-E) Study Early Access Program Using Alpha 1 Antitrypsin Infusion for Patients With Steroid Refractory Acute GvHD After Hematopoietic Stem Cell Transplantation (HSCT) Characterization of the Pathobiology of Early Lung Destruction in Alpha 1-Antitrypsin Deficient Individuals COPD Exacerbation Blood and Urine Biomarkers Study Alpha-1 Carrier Genomics Study Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype Study of ARO-AAT in Normal Adult Volunteers Safety Study of Alfalastin (Human Alpha-1 Antitrypsin) Administered at Home AL1TER™: Alpha-1 Therapy, Evaluation, and Research Patient Registry Respreeza® Self-administration and Learning Program (AmAREtTI Study) Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) Deficiency Lung Disease and Its Affect on the Work of White Blood Cells in the Lungs A 12-week Study Treating Participants Who Have alpha1-antitrypsin-related COPD With Alvelestat (MPH966) or Placebo. Environment Effect on Six-Minute Walk Test Performance Alpha-1 Foundation DNA and Tissue Bank Alpha1-antitrypsin Deficiency Registry Alpha-1 Research Registry Stage 1 Study of ARALAST NP and GLASSIA in A1PI Deficiency Alpha-1 Coded Testing(ACT) Study Long-Term Follow-up Study of ADVM-043 Safety Dose Finding Study of ADVM-043 Gene Therapy to Treat Alpha-1 Antitrypsin (A1AT) Deficiency 4-PBA: Will it Increase the Level of Alpha 1-Antitrypsin(AAT) in Persons With AAT Deficiency? Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin Deficiency Management of Patients With Alpha-1 Antitrypsin Deficiency Associated Emphysema Phase II, Safety and Efficacy Study of Kamada-alpha-1-antitrypsin (AAT) for Inhalation” EARCO REGISTRY. History Of Patients With Alpha-1 Antitrypsin GLASSIA Safety, Immunogenicity, and Bronchoalveolar Lavage Study Safety, Tolerability and Effect of ARC-AAT Injection on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels Efficacy/Safety of HA Inhalation Solution for Hereditary Emphysema in Patients With Alpha-1 Antitrypsin Deficiency Safety and Pharmacokinetics of Alpha-1 MP in Patients With Alpha1-Antitrypsin Deficiency Phase II/III Study of an Alpha-1 Proteinase Inhibitor (Kamada-API) in Individuals With Alpha-1 Antitrypsin Deficiency Study of the Effect of Aerosolized, Recombinant Alpha 1-Antitrypsin on Epithelial Lining Fluid Analytes in Subjects With Alpha 1-Antitrypsin Deficiency Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiency The Safety and Tolerability of Alpha-1 Modified Process (MP) In Subjects With Alpha-1-antitrypsin (AAT) Deficiency Lung Volume Reduction Coils for Emphysema in Alpha-1 Antitrypsin Deficiency Study of Genotype and Phenotype in Patients With Alpha 1-Antitrypsin Deficiency Alpha-1 Foundation Research Registry Targeting Pulmonary Perfusion in Alpha-1 Antitrypsin Deficiency Effects of Exercise Training in Chronic Obstructive Pulmonary Disease Versus Alpha-1-Antitrypsin-deficiency-patients Evaluate Efficacy and Safety of “Kamada-AAT for Inhalation” in Patients With AATD A Study of DCR-A1AT in Healthy Adult Volunteers and Patients With A1ATD-Associated Liver Disease Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency Effects of Different Exercise Training Modalities in Alpha-1 Antitrypsin Deficiency Patients Safety Study of an Aerosolized, Recombinant Alpha 1-Antitrypsin in Subjects With Alpha 1-Antitrypsin Deficiency Study Comparing Weekly Intravenous Administration of OctaAlpha1 With a Marketed Preparation Glassia® in Subjects With Alpha-1-antitrypsin Deficiency Long-term Safety of Alpha1-Proteinase Inhibitor (Human) in Japanese Subjects With Alpha1 Antitrypsin Deficiency (GTI1401-OLE) Efficacy and Safety Study of Augmentation Therapy With ARALAST Fraction IV-1 (Human Alpha 1 – Proteinase Inhibitor) Aralast alpha1-proteinase Inhibitor Surveillance Study Pharmacokinetic Study of ARALAST (Human Alpha1- PI) The Use of High Resolution Chest Computed Tomography in Alpha-1 Antitrypsin Deficiency Prevalence of Alpha-1 Antitrypsin Deficiency in Chronic Obstructive Pulmonary Disease (COPD) Comparison of Pharmacokinetic, Safety, Tolerability of Alpha-1 MP and Prolastin In Alpha1-antitrypsin Deficient Adults Phase 1 Study to Assess the Safety, PK and PD of INBRX-101 in Adults With Alpha-1 Antitrypsin Deficiency Safety and Pharmacokinetics of Alpha-1 Proteinase Inhibitor in Subjects With Alpha1-Antitrypsin Deficiency The Impact of Delayed Diagnosis of Alpha-1 Antitrypsin Deficiency Epigenetic Regulation of Immunity in Alpha-1 Anti-trypsin Deficiency Microbioma in Sputa From COPD With Alpha-1 Antitrypsin Deficiency A Study of ARC-AAT in Healthy Volunteer Subjects and Patients With Alpha-1 Antitrypsin Deficiency (AATD) A Study to Assess Safety and PK of Liquid Alpha₁-Proteinase Inhibitor (Human) in Treating Alpha₁-Antitrypsin Deficiency

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