Phase II Study of the Safety and Efficacy of Inhaled Alpha-1 Antitrypsin (AAT ) in Cystic Fibrosis Patients

This study has been completed.
Sponsor:
Information provided by:
Kamada, Ltd.
ClinicalTrials.gov Identifier:
NCT00499837
First received: July 11, 2007
Last updated: December 30, 2009
Last verified: December 2009
  Purpose

Cystic Fibrosis (CF) is an inherited disorder in which mucus-secreting glands in the lungs produce considerable quantity of thick, sticky secretions that clog the airways, promote bacterial growth and lead to chronic obstruction, inflammation and destruction of the airways.

The purpose of this study is to collect data about the resolution of the chronic inflammatory state in addition to assure the safety of the therapy in CF patients.


Condition Intervention Phase
Cystic Fibrosis
Drug: Aerosolized, human, plasma-derived Alpha-1 Antitrypsin
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double-Blind
Primary Purpose: Treatment
Official Title: Double-Blind, Randomized, Placebo-Controlled, Phase II Study of the Safety and Efficacy of Inhaled Alpha-1 Antitrypsin (AAT ) in Cystic Fibrosis Patients

Resource links provided by NLM:


Further study details as provided by Kamada, Ltd.:

Primary Outcome Measures:
  • Safety and airway inflammation

Secondary Outcome Measures:
  • Sputum microbiology, pulmonary function and serum CRP

Estimated Enrollment: 21
Study Start Date: September 2007
Study Completion Date: July 2008
Detailed Description:

In CF patients the unregulated inflammatory response overwhelms the normal protease (elastase)/antiprotease (AAT) balance, leading to the accumulation of elastase in the lung, destruction of the lung architecture, severe pulmonary dysfunction, and ultimately death.

Administration of AAT is to address the elastase/antiprotease imbalance in order to prevent destruction of the lung tissue and reduce the inflammatory dysregulation that causes pulmonary dysfunction.

  Eligibility

Ages Eligible for Study:   5 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosis of CF by clinical symptoms and positive sweat test or disease inducing mutation.
  • Age >5 yrs
  • Proven ability to perform reproducible PFTs
  • FEV1 >25% predicted
  • Steady disease state for 3 months and no decrease in lung function exceeding 10% during that period
  • Colonization
  • Stable concomitant therapy >2 weeks prior to visit 1 and during the study
  • Non-tobacco user of any kind
  • Ability for sputum induction
  • Written informed consent

Exclusion Criteria:

  • Severe CF with an FEV1 of <25% predicted
  • History of lung transplant
  • Active allergic bronchopulmonary aspergillosis (ABPA)
  • Treatment with additional antibiotics (beyond standard CF treatment) for a period of 14 days before study entry (routine antibiotics permitted)
  • Treatment with additional oral and/or IV steroids (beyond standard CF treatment) for a period of 14 days before study entry (screening day)
  • Known hypersensitivity to plasma products
  • IgA deficiency
  • Uncontrolled hypertension
  • Lung surgery in the previous two years
  • Being on any thoracic surgery waiting list
  • Severe concomitant disease
  • Hospitalization within 1 month before study entry, not due to an airway disease
  • Severe liver cirrhosis with ascites
  • Hypersplenism
  • Grade III/IV oesophageal varices
  • Active pulmonary exacerbation within the 4 weeks prior to screening
  • History of significant hemoptysis within the previous year
  • Use of tobacco products or recreational drugs
  • Pregnancy or breastfeeding
  • Any serious or active medical or psychiatric illness which, in the opinion of the investigator, would interfere with patient treatment, assessment, or compliance with the protocol.
  • Being a female of child-bearing age without adequate contraception
  • Participation in research study within 1 month
  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 ClinicalTrials.gov identifier: NCT00499837

Locations
Israel
Hadassah Hebrew University, Medical Center
Jerusalem, Israel, 91240
Sponsors and Collaborators
Kamada, Ltd.
Investigators
Principal Investigator: Eitan Kerem, MD Hadassah Hebrew University, Medical Center, Mt. Scopus, Jerusalem
  More Information

No publications provided

ClinicalTrials.gov Identifier: NCT00499837     History of Changes
Other Study ID Numbers: Kamada-AAT (inhaled)-003
Study First Received: July 11, 2007
Last Updated: December 30, 2009
Health Authority: Israel: Ministry of Health

Additional relevant MeSH terms:
Cystic Fibrosis
Fibrosis
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Pathologic Processes
Alpha 1-Antitrypsin
Protein C Inhibitor
Trypsin Inhibitors
Serine Proteinase Inhibitors
Protease Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions

ClinicalTrials.gov processed this record on August 25, 2014