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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00499837
Recruitment Status : Completed
First Posted : July 12, 2007
Last Update Posted : June 9, 2016
Information provided by (Responsible Party):
Kamada, Ltd.

Brief Summary:

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 or disease Intervention/treatment Phase
Cystic Fibrosis Drug: Aerosolized, human, plasma-derived Alpha-1 Antitrypsin Phase 2

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.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 21 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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
Study Start Date : September 2007
Actual Primary Completion Date : July 2008
Actual Study Completion Date : July 2008

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arm Intervention/treatment
Experimental: 80 mg/kg AAT inhaled
80 mg/kg AAT inhaled
Drug: Aerosolized, human, plasma-derived Alpha-1 Antitrypsin
Placebo Comparator: Placebo inhaled
Placebo inhaled
Drug: Aerosolized, human, plasma-derived Alpha-1 Antitrypsin

Primary Outcome Measures :
  1. Safety and airway inflammation [ Time Frame: days 1,7,14,21,28,35,42,49,56,63 ]

Secondary Outcome Measures :
  1. Sputum microbiology, pulmonary function and serum CRP [ Time Frame: days 1,7,14,21,28,35,42,49,56,63 ]

Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00499837

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

Responsible Party: Kamada, Ltd. Identifier: NCT00499837     History of Changes
Other Study ID Numbers: Kamada-AAT (inhaled)-003
First Posted: July 12, 2007    Key Record Dates
Last Update Posted: June 9, 2016
Last Verified: April 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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