Trial record 20 of 48 for:    "alpha-1 antitrypsin deficiency"

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

This study is not yet open for participant recruitment.
Verified December 2013 by Kamada, Ltd.
Sponsor:
Information provided by (Responsible Party):
Kamada, Ltd.
ClinicalTrials.gov Identifier:
NCT02001688
First received: November 24, 2013
Last updated: December 5, 2013
Last verified: December 2013
  Purpose

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 serum [ 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: December 2013
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

  Eligibility

Ages Eligible for Study:   18 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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 [subjects with deficient serum A1-PI levels who carry deficient disease known mutation].
  • 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 or acute Hepatitis B, Hepatitis C or HIV infection (negative serologies for HIV and viral hepatitis). Positive serologies due to hepatitis immunizations are acceptable.
  • 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 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
Please refer to this study by its ClinicalTrials.gov identifier: NCT02001688

Locations
United States, Florida
University of Florida, Pulmonary, Critical Care & Sleep Medicine Not yet recruiting
Gainesville, Florida, United States, 32610
Contact: Mark Brantly       mark.brantly@medicine.ufl.edu   
Principal Investigator: Mark Brantly, MD         
United States, South Carolina
Medical University of South Carolina, Pulmonary and Critical Care Medicine Not yet recruiting
Charleston, South Carolina, United States, 29425
Contact: Charles Strange       strangec@musc.edu   
Principal Investigator: Charles Strange         
United States, Texas
The University of Texas Health Science Center at Tyler Center for Clinical Research Not yet recruiting
Tyler, Texas, United States, 75708
Contact: James Stocks       James.Stocks@uthct.edu   
Principal Investigator: James Stocks         
Sponsors and Collaborators
Kamada, Ltd.
  More Information

No publications provided

Responsible Party: Kamada, Ltd.
ClinicalTrials.gov Identifier: NCT02001688     History of Changes
Other Study ID Numbers: Kamada-AAT (inhaled)-006
Study First Received: November 24, 2013
Last Updated: December 5, 2013
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Alpha 1-Antitrypsin Deficiency
Liver Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Subcutaneous Emphysema
Emphysema
Pathologic Processes

ClinicalTrials.gov processed this record on April 15, 2014