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

Kamada

Status and phase

Completed

Phase 2

Conditions

Cystic Fibrosis

Treatments

Drug: Aerosolized, human, plasma-derived Alpha-1 Antitrypsin

Study type

Interventional

Funder types

Industry

Identifiers

NCT00499837

Kamada-AAT (inhaled)-003

Details and patient eligibility

About

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.

Full 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.

Enrollment

21 patients

Sex

All

Ages

5+ years old

Volunteers

No Healthy Volunteers

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