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

NCT00499837 | Completed Phase 2

• 1 other identifier: Kamada-AAT (inhaled)-003
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 1

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.

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

• Safety and airway inflammation

  days 1,7,14,21,28,35,42,49,56,63

Secondary Outcomes (1)

• Sputum microbiology, pulmonary function and serum CRP

  days 1,7,14,21,28,35,42,49,56,63

Study Arms (2)

80 mg/kg AAT inhaled

EXPERIMENTAL

80 mg/kg AAT inhaled

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

Placebo inhaled

PLACEBO COMPARATOR

Placebo inhaled

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

Interventions

Aerosolized, human, plasma-derived Alpha-1 Antitrypsin DRUG

80 mg/kg AAT inhaled; Placebo inhaled

Eligibility Criteria

• Age: 5 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Kamada, Ltd.: lead

Study Sites (1)

Hadassah Hebrew University, Medical Center

Jerusalem, 91240, Israel

Location

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Eitan Kerem, MD

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

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 11, 2007
• First Posted: July 12, 2007
• Study Start: September 1, 2007
• Primary Completion: July 1, 2008
• Study Completion: July 1, 2008
• Last Updated: June 9, 2016

Record last verified: 2016-04

Data Sharing

• IPD Sharing: Will not share

Locations

IL (1)