Phase II, Safety and Efficacy Study of Kamada-alpha-1-antitrypsin (AAT) for Inhalation”

Related Clinical Trial
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

Phase II, Safety and Efficacy Study of Kamada-alpha-1-antitrypsin (AAT) for Inhalation"

Official Title

Phase II, Double-Blind, Placebo-Controlled Study to Explore the ELF and Plasma Concentration as Well as Safety of Inhaled Alpha-1 Antitrypsin in Alpha-1 Antitrypsin Deficiency Subjects

Brief Summary

      To evaluate different doses of "Kamada-AAT for Inhalation" on the levels of alpha
      1-proteinase inhibitor and other analytes in epithelial lining fluid (ELF) and serum and to
      assess the safety of the treatment in subjects with AAT Deficiency.
    


Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Change From Baseline in the Concentration of Antigenic Alpha-1 Antitrypsin (AAT) in the Lung Epithelial Lining Fluid (ELF)

Secondary Outcome

 Change From Baseline in Levels of M Specific AAT in Plasma (PiM)

Condition

Alpha-1 Antitrypsin Deficiency

Intervention

Kamada-AAT for Inhalation, 80mg

Study Arms / Comparison Groups

 Kamada-AAT for Inhalation, 80mg
Description:  Daily inhalation of Kamada-AAT for Inhalation, 80mg

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

Drug

Estimated Enrollment

36

Start Date

April 2014

Completion Date

May 2016

Primary Completion Date

February 2016

Eligibility Criteria

        Inclusion Criteria:

          -  Male or female patients between 18 and 65 years of age (inclusive).

          -  Able and willing to sign informed consent.

          -  Males, and non-pregnant, non-lactating females whose screening pregnancy test is
             negative and who are using contraceptive methods deemed reliable by the investigator
             or who are post-menopausal or surgically sterilized.

          -  Diagnosis of alpha1-antitrypsin deficiency [only individuals with a ZZ or Z null
             classification].

          -  Forced expiratory volume in one second (FEV1) ≥ 50% of predicted post bronchodilator

          -  No respiratory exacerbations within 6 weeks of baseline. Subjects can be re-screened
             if exacerbations exist at the time of enrollment.

          -  No signs of chronic and/or acute Hepatitis A, Hepatitis B, Hepatitis C, HIV infection
             and Parvovirus B19, by NAT (for Parvovirus B19, nucleic acid testing (NAT) result must
             be < 10^4 IU/mL).

          -  No significant abnormalities in serum hematology, serum chemistry, serum inflammatory
             / immunogenic markers and urinalysis.

          -  No significant abnormalities in ECG.

          -  Not on intravenous augmentation therapy for at least 8 weeks prior to initial dosing
             with study drug/placebo and willing to forego intravenous augmentation therapy for the
             duration of the study.

        Exclusion Criteria:

          -  Clinically significant intercurrent illnesses (except for respiratory or liver disease
             secondary to AAT deficiency), including: cardiac, hepatic, renal, endocrine,
             neurological, hematological, neoplastic, immunological, skeletal or other) that in the
             opinion of the investigator, could interfere with the safety, compliance or other
             aspects of this study. Patients with well-controlled, chronic diseases could possibly
             be included after consultation with the treating physician and the sponsor.

          -  History of life threatening allergy, anaphylactic reaction, or systemic response to
             human plasma derived products.

          -  History of life threatening transfusion reactions.

          -  History of lung transplant.

          -  Current or previous (up to 8 weeks from baseline) use of AAT augmentation therapy or
             by any other route

          -  Current use of oral or parenteral glucocorticoids in doses exceeding 10mg of
             prednisone daily or equivalent generics (substance and dose).

          -  Any lung surgery within the past two years.

          -  On any thoracic surgery waiting list.

          -  Active smoking during the last 12 months from screening date.

          -  Pregnancy or lactation.

          -  Woman of child-bearing potential not taking adequate contraception deemed reliable by
             the investigator.

          -  Presence of psychiatric/ mental disorder or any other medical disorder which might
             impair the patient's ability to give informed consent or to comply with the
             requirements of the study protocol.

          -  Evidence of alcohol abuse or history of alcohol abuse or illegal and/or legally
             prescribed drugs.

          -  Immunoglobulin A (IgA) Deficiency.

          -  Inability to undergo bronchoscopy.

          -  Allergy to lidocaine or any other medicines used in the bronchoscopy process

          -  Exacerbation of chronic obstructive pulmonary disease (COPD) in the previous 6 weeks.

          -  Participation in another clinical trial involving investigational medication or
             interventional treatment within 30 days prior to baseline visit.

          -  Participation in observational clinical trial which involves any invasive procedure
             scheduled to occur during the AAT inhaled study period. If participating in an
             observational clinical trial that already completed all diagnostic procedures (e.g.
             liver biopsy), any adverse events (AEs) experienced must have returned to baseline
             within 30 days prior to baseline visit.

          -  Inability to attend scheduled clinic visits and/or comply with the study protocol.

          -  Any other factor that, in the opinion of the investigator, would prevent the patient
             form complying with the requirements of the protocol.
      

Gender

All

Ages

18 Years - 65 Years

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT02001688

Organization ID

Kamada-AAT (inhaled)-006


Responsible Party

Sponsor

Study Sponsor

Kamada, Ltd.


Study Sponsor

, , 


Verification Date

December 2019