Phase II, Safety and ELF Study of "Kamada-API for Inhalation"

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2015 by Kamada, Ltd.
Information provided by (Responsible Party):
Kamada, Ltd. Identifier:
First received: November 24, 2013
Last updated: February 8, 2015
Last verified: February 2015

To evaluate different doses of "Kamada-API 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 Alpha-1 Antitrypsin Deficiency.

Condition Intervention Phase
Alpha-1 Antitrypsin Deficiency
Biological: Kamada-API for Inhalation, 80mg
Drug: Placebo
Biological: Kamada-API for Inhalation, 160mg
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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

Resource links provided by NLM:

Further study details as provided by Kamada, Ltd.:

Primary Outcome Measures:
  • Concentration of active AAT (Alpha-1 antitrypsin) in ELF [ Time Frame: 3 months from dosing ] [ Designated as safety issue: No ]
  • Concentration of antigenic AAT (Alpha-1 antitrypsin) in ELF [ Time Frame: 3 months from dosing ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Safety and tolerability [ Time Frame: 3 months and 6 months from dosing ] [ Designated as safety issue: No ]
    Adverse Events, Serious Adverse Events, physical examination, vital signs

  • Concentration of active AAT in plasma [ Time Frame: 3 months from dosing ] [ Designated as safety issue: No ]
  • ELF inflammatory analytes [ Time Frame: 3 months from dosing ] [ Designated as safety issue: No ]
    Cytokines and proteases

Estimated Enrollment: 40
Study Start Date: April 2014
Estimated Primary Completion Date: February 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Active, group A
Kamada-API for Inhalation, 80mg
Biological: Kamada-API for Inhalation, 80mg
Placebo Comparator: Placebo
Placebo administered by inhalation daily
Drug: Placebo
Experimental: Active, group B
Kamada-API for Inhalation, 160mg
Biological: Kamada-API for Inhalation, 160mg


Ages Eligible for Study:   18 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

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].
  • FEV1 (forced expiratory volume at one second) ≥ 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, 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.
  • IgA (immunoglobulin A) Deficiency.
  • Inability to undergo bronchoscopy.
  • Allergy to lidocaine or any other medicines used in the bronchoscopy process
  • Exacerbation of COPD (chronic obstructive pulmonary disease) 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 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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT02001688

United States, Florida
University of Florida, Pulmonary, Critical Care & Sleep Medicine Recruiting
Gainesville, Florida, United States, 32610
Contact: Joanna Nolte, ARNP   
Principal Investigator: Mark Brantly, MD         
United States, Texas
The University of Texas Health Science Center at Tyler Center for Clinical Research Recruiting
Tyler, Texas, United States, 75708
Contact: James Stocks   
Principal Investigator: James Stocks         
Sponsors and Collaborators
Kamada, Ltd.
  More Information

No publications provided

Responsible Party: Kamada, Ltd. Identifier: NCT02001688     History of Changes
Other Study ID Numbers: Kamada-AAT (inhaled)-006
Study First Received: November 24, 2013
Last Updated: February 8, 2015
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Alpha 1-Antitrypsin Deficiency
Digestive System Diseases
Genetic Diseases, Inborn
Liver Diseases
Lung Diseases
Pathologic Processes
Respiratory Tract Diseases
Subcutaneous Emphysema processed this record on October 09, 2015