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

NCT ID: NCT00499837

Last Updated: 2016-06-09

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 21 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2007-09-30
• Study Completion Date: 2008-07-31

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.

Conditions

Cystic Fibrosis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

80 mg/kg AAT inhaled

80 mg/kg AAT inhaled

Group Type: EXPERIMENTAL

Aerosolized, human, plasma-derived Alpha-1 Antitrypsin

Intervention Type: DRUG

Placebo inhaled

Placebo inhaled

Group Type: PLACEBO_COMPARATOR

Aerosolized, human, plasma-derived Alpha-1 Antitrypsin

Intervention Type: DRUG

Interventions

Aerosolized, human, plasma-derived Alpha-1 Antitrypsin

Intervention Type: DRUG

Eligibility 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

• Minimum Eligible Age: 5 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Kamada, Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Eitan Kerem, MD

Role: PRINCIPAL_INVESTIGATOR

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

Locations

Hadassah Hebrew University, Medical Center

Jerusalem, , Israel

Countries

Israel

Other Identifiers

Kamada-AAT (inhaled)-003

Identifier Type: -

Identifier Source: org_study_id