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

Related Clinical Trial
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 Evaluate Safety, Tolerability and Pharmacokinetics of Two Different Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% in Participants With Alpha1-Antitrypsin Deficiency

Official Title

A Multi-Center, Single-Dose and Repeat-Dose Over Eight Weeks, Sequential Cohort Study to Evaluate Safety and Tolerability as Well as Pharmacokinetics of Two Different Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% Administered Subcutaneously in Subjects With Alpha1-Antitrypsin Deficiency

Brief Summary

      The purpose of this study is to evaluate the safety and tolerability of 72 milligrams per
      kilogram (mg/kg) and 144 mg/kg Alpha-1 15%, administered as a single-dose subcutaneous (SC)
      infusion and subsequently as weekly SC infusions over 8 weeks in participants with
      Alpha1-Antitrypsin Deficiency (AATD).
    


Study Phase

Phase 1/Phase 2

Study Type

Interventional


Primary Outcome

Number of Participants With Adverse Events (AEs)


Condition

Alpha1-Antitrypsin Deficiency

Intervention

Alpha-1 15%

Study Arms / Comparison Groups

 Cohort 1: Treatment Period 1 (Alpha-1 15%, 72 mg/kg)
Description:  Participants will receive Alpha-1 15% 72 mg/kg, single weekly subcutaneous (SC) infusion in treatment-period 1 (Single-Dose) at Week 1.

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

16

Start Date

August 13, 2021

Completion Date

February 5, 2023

Primary Completion Date

February 5, 2023

Eligibility Criteria

        Inclusion Criteria:

          -  Have a diagnosis of congenital Alpha1-antitrypsin deficiency (AATD) with an allelic
             combination of ZZ, SZ, Z(null), (null)(null), S(null), or "at-risk" alleles (subjects
             with "at-risk" alleles must be individually evaluated for eligibility by the Medical
             Monitor).

          -  Have a documented pre-Alpha1-Proteinase Inhibitor (PI) augmentation therapy serum
             alpha-1 antitrypsin (AAT) level <11 micrometer (μM) (80 milligrams per decilitre
             (mg/dL) if measured by radial immunodiffusion or 50 mg/dL if measured by
             nephelometry).

          -  Currently receiving Alpha1-PI augmentation therapy or has received Alpha1-PI
             augmentation therapy within the past. If the subject is currently receiving 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 IPs for this study, while participating in the study. Note: Subjects must not
             be naïve to Alpha1-PI augmentation therapy for study participation.

          -  At the Screening Visit, have a post-bronchodilator forced expiratory volume (FEV1)
             ≥30% and <80% of predicted and FEV1/forced vital capacity (FVC) <70% (Global
             Initiative for Chronic Obstructive Lung Disease (GOLD) stage II or III).

        Exclusion Criteria:

          -  Have had a moderate or severe Chronic obstructive pulmonary disease (COPD)
             exacerbation during the 4 weeks before the Week 1 (Baseline) Visit.

          -  Have history of lung or liver transplant.

          -  Have any lung surgery during the past 2 years (excluding lung biopsy).

          -  Have severe concomitant disease (example, congestive heart failure, clinically
             significant pulmonary fibrosis, malignant disease [except for 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 true abstinence) throughout
             the study.

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

          -  Participate in another Investigational product (IP) study within one month prior to
             the Week 1 (Baseline) Visit.

          -  Have history of anaphylaxis or severe systemic response to any plasma-derived
             Alpha1-PI preparation or other blood product(s).

          -  Use 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. It is
             recommended to maintain the same dose throughout the study.

          -  Use systemic or aerosolized antibiotics for a chronic COPD exacerbation within the 4
             weeks prior to the Week 1 (Baseline) Visit.

          -  Have known selective or severe Immunoglobulin A (IgA) deficiency.
      

Gender

All

Ages

18 Years - 70 Years

Accepts Healthy Volunteers

No

Contacts

, +1 919 316 6693, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT04722887

Organization ID

GC2008


Responsible Party

Sponsor

Study Sponsor

Grifols Therapeutics LLC


Study Sponsor

, , 


Verification Date

October 2021