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

Primary Purpose

Cystic Fibrosis

Status
Completed
Phase
Phase 2
Locations
Israel
Study Type
Interventional
Intervention
Aerosolized, human, plasma-derived Alpha-1 Antitrypsin
Sponsored by
Kamada, Ltd.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cystic Fibrosis

Eligibility Criteria

5 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

  • Hadassah Hebrew University, Medical Center

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

80 mg/kg AAT inhaled

Placebo inhaled

Arm Description

80 mg/kg AAT inhaled

Placebo inhaled

Outcomes

Primary Outcome Measures

Safety and airway inflammation

Secondary Outcome Measures

Sputum microbiology, pulmonary function and serum CRP

Full Information

First Posted
July 11, 2007
Last Updated
June 8, 2016
Sponsor
Kamada, Ltd.
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1. Study Identification

Unique Protocol Identification Number
NCT00499837
Brief Title
Phase II Study of the Safety and Efficacy of Inhaled Alpha-1 Antitrypsin (AAT ) in Cystic Fibrosis Patients
Official Title
Double-Blind, Randomized, Placebo-Controlled, Phase II Study of the Safety and Efficacy of Inhaled Alpha-1 Antitrypsin (AAT ) in Cystic Fibrosis Patients
Study Type
Interventional

2. Study Status

Record Verification Date
April 2016
Overall Recruitment Status
Completed
Study Start Date
September 2007 (undefined)
Primary Completion Date
July 2008 (Actual)
Study Completion Date
July 2008 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Kamada, Ltd.

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
21 (Actual)

8. Arms, Groups, and Interventions

Arm Title
80 mg/kg AAT inhaled
Arm Type
Experimental
Arm Description
80 mg/kg AAT inhaled
Arm Title
Placebo inhaled
Arm Type
Placebo Comparator
Arm Description
Placebo inhaled
Intervention Type
Drug
Intervention Name(s)
Aerosolized, human, plasma-derived Alpha-1 Antitrypsin
Primary Outcome Measure Information:
Title
Safety and airway inflammation
Time Frame
days 1,7,14,21,28,35,42,49,56,63
Secondary Outcome Measure Information:
Title
Sputum microbiology, pulmonary function and serum CRP
Time Frame
days 1,7,14,21,28,35,42,49,56,63

10. Eligibility

Sex
All
Minimum Age & Unit of Time
5 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Eitan Kerem, MD
Organizational Affiliation
Hadassah Hebrew University, Medical Center, Mt. Scopus, Jerusalem
Official's Role
Principal Investigator
Facility Information:
Facility Name
Hadassah Hebrew University, Medical Center
City
Jerusalem
ZIP/Postal Code
91240
Country
Israel

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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