- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00499837
Phase II Study of the Safety and Efficacy of Inhaled Alpha-1 Antitrypsin (AAT ) in Cystic Fibrosis Patients
Double-Blind, Randomized, Placebo-Controlled, Phase II Study of the Safety and Efficacy of Inhaled Alpha-1 Antitrypsin (AAT ) in Cystic Fibrosis Patients
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.
Study Overview
Status
Conditions
Intervention / Treatment
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.
Study Type
Enrollment (Actual)
Phase
- Phase 2
Contacts and Locations
Study Locations
-
-
-
Jerusalem, Israel, 91240
- Hadassah Hebrew University, Medical Center
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: RANDOMIZED
- Interventional Model: PARALLEL
- Masking: QUADRUPLE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
EXPERIMENTAL: 80 mg/kg AAT inhaled
|
|
PLACEBO_COMPARATOR: Placebo inhaled
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Safety and airway inflammation
Time Frame: days 1,7,14,21,28,35,42,49,56,63
|
days 1,7,14,21,28,35,42,49,56,63
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Sputum microbiology, pulmonary function and serum CRP
Time Frame: days 1,7,14,21,28,35,42,49,56,63
|
days 1,7,14,21,28,35,42,49,56,63
|
Collaborators and Investigators
Sponsor
Investigators
- Principal Investigator: Eitan Kerem, MD, Hadassah Hebrew University, Medical Center, Mt. Scopus, Jerusalem
Study record dates
Study Major Dates
Study Start
Primary Completion (ACTUAL)
Study Completion (ACTUAL)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (ESTIMATE)
Study Record Updates
Last Update Posted (ESTIMATE)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Digestive System Diseases
- Pathologic Processes
- Respiratory Tract Diseases
- Lung Diseases
- Infant, Newborn, Diseases
- Liver Diseases
- Genetic Diseases, Inborn
- Pancreatic Diseases
- Subcutaneous Emphysema
- Emphysema
- Fibrosis
- Cystic Fibrosis
- Alpha 1-Antitrypsin Deficiency
- Molecular Mechanisms of Pharmacological Action
- Enzyme Inhibitors
- Protease Inhibitors
- Serine Proteinase Inhibitors
- Trypsin Inhibitors
- Alpha 1-Antitrypsin
- Protein C Inhibitor
Other Study ID Numbers
- Kamada-AAT (inhaled)-003
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Cystic Fibrosis
-
Hospital de Clinicas de Porto AlegreUnknownCystic Fibrosis | Cystic Fibrosis Pulmonary Exacerbation | Cystic Fibrosis in Children | Cystic Fibrosis With ExacerbationBrazil
-
University of Colorado, DenverCystic Fibrosis FoundationTerminatedCystic Fibrosis-related Diabetes | Cystic Fibrosis Pulmonary Exacerbation | Cystic Fibrosis in ChildrenUnited States
-
Royal College of Surgeons, IrelandThe Hospital for Sick Children; Imperial College London; Erasmus Medical Center; University College Dublin and other collaboratorsActive, not recruitingCystic Fibrosis | Adherence, Medication | Cystic Fibrosis Gastrointestinal Disease | Cystic Fibrosis in Children | Cystic Fibrosis Liver DiseaseUnited Kingdom, Ireland
-
Herlev and Gentofte HospitalCopenhagen University Hospital, DenmarkActive, not recruitingMyocardial Infarction | Heart Diseases | Heart Failure | Stroke | Cystic Fibrosis | Heart Failure, Diastolic | Heart Failure, Systolic | Left Ventricular Dysfunction | Cystic Fibrosis-related Diabetes | Cystic Fibrosis Gastrointestinal Disease | Cystic Fibrosis of Pancreas | Cystic Fibrosis, Pulmonary | Cystic...Denmark
-
The Hospital for Sick ChildrenCanadian Cystic Fibrosis FoundationActive, not recruitingCystic Fibrosis | Cystic Fibrosis Gastrointestinal Disease | Cystic Fibrosis in ChildrenCanada
-
Arrowhead PharmaceuticalsTerminatedCystic Fibrosis, PulmonaryAustralia, New Zealand
-
AzurRx SASCompletedCystic Fibrosis | Cystic Fibrosis Gastrointestinal Disease | Cystic Fibrosis of PancreasTurkey, Hungary
-
Dartmouth-Hitchcock Medical CenterTrustees of Dartmouth CollegeWithdrawnCystic Fibrosis-related Diabetes | Cystic Fibrosis Liver Disease | CF - Cystic FibrosisUnited States
-
University Hospital, BordeauxCompleted
-
University of PortsmouthUniversity Hospital Southampton NHS Foundation Trust; Loughborough University; Queen Alexandra HospitalTerminated
Clinical Trials on Aerosolized, human, plasma-derived Alpha-1 Antitrypsin
-
Baxalta now part of ShireArriva Pharmaceuticals, Inc.CompletedAlpha1-antitrypsin DeficiencyUnited States
-
Baxalta now part of ShireArriva Pharmaceuticals, Inc.CompletedAlpha1-antitrypsin DeficiencyUnited States
-
Michael Campos, MDCSL BehringCompletedAlpha 1 Antitrypsin DeficiencyUnited States
-
Grifols Therapeutics LLCInstituto Grifols, S.A.CompletedCongenital AfibrinogenemiaIndia, United States, Italy, Lebanon
-
Baxalta now part of ShireDynPort Vaccine Company LLC, A GDIT CompanyCompletedHealthy Volunteers | Chemical Terrorism | Chemical WarfareUnited States
-
Baxalta now part of ShireDynPort Vaccine Company LLC, A GDIT CompanyCompletedHealthy VolunteersUnited States
-
Third Affiliated Hospital, Sun Yat-Sen UniversityUnknownAcute-On-Chronic Liver Failure | Hepatitis BChina
-
National Institute of Allergy and Infectious Diseases...Juvenile Diabetes Research Foundation; Immune Tolerance Network (ITN)WithdrawnDiabetes Mellitus, Type 1United States
-
Kamada, Ltd.CompletedType 1 Diabetes MellitusIsrael
-
Rabin Medical CenterUnknownLung Transplantation | Bronchiolitis Obliterable Syndrome