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

Tracking Information
First Submitted Date ICMJE	July 11, 2007
First Posted Date ICMJE	July 12, 2007
Last Update Posted Date	June 9, 2016
Study Start Date ICMJE	September 2007
Actual Primary Completion Date	July 2008 (Final data collection date for primary outcome measure)
Current Primary Outcome Measures ICMJE; (submitted: June 8, 2016)	Safety and airway inflammation [ Time Frame: days 1,7,14,21,28,35,42,49,56,63 ]
Original Primary Outcome Measures ICMJE; (submitted: July 11, 2007)	Safety and airway inflammation
Change History	Complete list of historical versions of study NCT00499837 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures ICMJE; (submitted: June 8, 2016)	Sputum microbiology, pulmonary function and serum CRP [ Time Frame: days 1,7,14,21,28,35,42,49,56,63 ]
Original Secondary Outcome Measures ICMJE; (submitted: July 11, 2007)	Sputum microbiology, pulmonary function and serum CRP
Current Other Outcome Measures ICMJE	Not Provided
Original Other Outcome Measures ICMJE	Not Provided
Descriptive Information
Brief Title ICMJE	Phase II Study of the Safety and Efficacy of Inhaled Alpha-1 Antitrypsin (AAT ) in Cystic Fibrosis Patients
Official Title ICMJE	Double-Blind, Randomized, Placebo-Controlled, Phase II Study of the Safety and Efficacy of Inhaled Alpha-1 Antitrypsin (AAT ) in Cystic Fibrosis Patients
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.
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 ICMJE	Interventional
Study Phase	Phase 2
Study Design ICMJE	Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
Condition ICMJE	Cystic Fibrosis
Intervention ICMJE	Drug: Aerosolized, human, plasma-derived Alpha-1 Antitrypsin
Study Arms	Experimental: 80 mg/kg AAT inhaled 80 mg/kg AAT inhaled Intervention: Drug: Aerosolized, human, plasma-derived Alpha-1 Antitrypsin; Placebo Comparator: Placebo inhaled Placebo inhaled Intervention: Drug: Aerosolized, human, plasma-derived Alpha-1 Antitrypsin
Publications *	Not Provided
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status ICMJE	Completed
Actual Enrollment ICMJE; (submitted: July 11, 2007)	21
Original Estimated Enrollment ICMJE	Same as current
Actual Study Completion Date	July 2008
Actual Primary Completion Date	July 2008 (Final data collection date for primary outcome measure)
Eligibility Criteria ICMJE	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
Sex/Gender	Sexes Eligible for Study: All
Ages	5 Years and older (Child, Adult, Senior)
Accepts Healthy Volunteers	No
Contacts ICMJE	Contact information is only displayed when the study is recruiting subjects
Listed Location Countries ICMJE	Israel
Removed Location Countries
Administrative Information
NCT Number ICMJE	NCT00499837
Other Study ID Numbers ICMJE	Kamada-AAT (inhaled)-003
Has Data Monitoring Committee	No
U.S. FDA-regulated Product	Not Provided
IPD Sharing Statement	Plan to Share IPD: No
Responsible Party	Kamada, Ltd.
Study Sponsor ICMJE	Kamada, Ltd.
Collaborators ICMJE	Not Provided
Investigators ICMJE	Principal Investigator: Eitan Kerem, MD Hadassah Hebrew University, Medical Center, Mt. Scopus, Jerusalem
PRS Account	Kamada, Ltd.
Verification Date	April 2016
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP