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

Sponsored by:	Kamada, Ltd.
Information provided by:	Kamada, Ltd.
ClinicalTrials.gov Identifier:	NCT00499837

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 II

Genetics Home Reference related topics: cystic fibrosis

MedlinePlus related topics: Cystic Fibrosis

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Randomized, Single Blind, Placebo Control, Parallel Assignment, Safety/Efficacy Study
Official Title:	Single-Blind, Randomized, Placebo-Controlled, Phase II Study of the Safety and Efficacy of Inhaled Alpha-1 Antitrypsin (AAT ) in Cystic Fibrosis Patients

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

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

Please refer to this study by its ClinicalTrials.gov identifier: NCT00499837

Contacts

Contact: Thea Pugatsch, PhD

+972-2-5844702

pthea@hadassah.org.il

Locations

Israel
Hadassah Hebrew University, Medical Center	Recruiting
Jerusalem, Israel, 91240
Contact: Thea Pugatsch, PhD 972-2-5844702 pthea@hanadssah.org.il
Principal Investigator: Eitan Kerem, MD, Prof.
Sub-Investigator: David Shosayov, MD

Sponsors and Collaborators

Kamada, Ltd.

Investigators

Principal Investigator:

Eitan Kerem, MD

Hadassah Hebrew University, Medical Center, Mt. Scopus, Jerusalem

More Information

Study ID Numbers:	Kamada-AAT (inhaled)-003
Study First Received:	July 11, 2007
Last Updated:	October 16, 2007
ClinicalTrials.gov Identifier:	NCT00499837
Health Authority:	Israel: Ministry of Health

Study placed in the following topic categories:

Protein C Inhibitor
Alpha 1-Antitrypsin
Digestive System Diseases
Protein C
Genetic Diseases, Inborn
Respiratory Tract Diseases

Cystic Fibrosis
Fibrosis
Lung Diseases
Infant, Newborn, Diseases
Pancreatic Diseases
Cystic fibrosis

Additional relevant MeSH terms:

Serine Proteinase Inhibitors
Pathologic Processes
Molecular Mechanisms of Pharmacological Action
Trypsin Inhibitors

Enzyme Inhibitors
Pharmacologic Actions
Protease Inhibitors

ClinicalTrials.gov processed this record on January 16, 2009