- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00486837
Deposition of Inhaled Prolastin in Cystic Fibrosis Patients (CF2)
Multicenter, Randomized, Parallel Group Study to Investigate the Optimal Deposition Site for Inhaled Prolastin® in Patients With Cystic Fibrosis (CF)
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
The optimum deposition site (bronchial or peripheral) in CF patients for AAT will be investigated by measuring several parameters in induced sputum. The study will start with a 2 week run-in period in which the planned 60 patients inhale isotonic saline once daily. This period is followed by a 4 week treatment period where 30 patients inhale AAT for peripheral deposition and 30 patients inhale AAT for bronchial deposition. Six patients in each group will be asked to collect spontaneous sputum at home.
Twenty-five milligrams of AAT will be deposited at one of the two target sites using the AKITA® device. The inhalation should take place in the evening between 18.00 and 23.00 h.
Patients will inhale saline once daily for 2 weeks (run-in period) followed by 4 weeks of once daily inhalation of AAT. Induced sputum will be collected at visits to the clinic at the start of the run-in, at the start of AAT treatment, and at 2 and 4 weeks after the start of AAT treatment.
Study Type
Enrollment (Actual)
Phase
- Phase 2
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- Patient with diagnosis of CF
- Age >= 8 years
- Forced expiratory volume at one second (FEV1) > 25 % of predicted value
- Free elastase activity checked at visit 1 must be positive (free elastolytic activity in the sample, 2 standard deviations above of the negative blank samples in the assay.) .
- Patient must be positive at least 3 times for pseudomonas in the last 2 years
- Patient must be positive for pseudomonas at Visit 1
- Patient must be able to perform reliable spirometry
- Patient must be on stable concomitant therapy at least 2 weeks prior to visit 1 and during the study
- Written informed consent of the patient or legal representative(s)
Exclusion Criteria:
- FEV1 < 25% of predicted value post-bronchodilator
- History of lung transplant
- Any lung surgery within the past 2 years
- On any thoracic surgery waiting list
- Severe concomitant disease (serious malignant disease, congestive heart failure New York Heart Association (NYHA) III/IV, cor pulmonale with the need of oxygen therapy)
- Severe liver cirrhosis with ascites, hypersplenism or grade III/IV esophageal varices.
- Known selective immunoglobulin A (IgA) deficiency with known antibody against IgA (anti-IgA antibody)
- Active pulmonary exacerbation within the 4 weeks prior to screening
- Current Smoking
- Pregnancy or lactation
- Women of child-bearing age without adequate contraception
- Any medical condition which the investigator feels will prohibit the patient from completing the trial
- Participation in another clinical trial within 30 days prior to inclusion at visit 1
Study Plan
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: RANDOMIZED
- Interventional Model: PARALLEL
- Masking: NONE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
EXPERIMENTAL: Group 1
Bronchial Deposition Intervention: Alpha1-Proteinase Inhibitor (Human) Dosage: 25 mg in lungs, one inhalation per day over 4 weeks
|
25 mg of Alpha1-Proteinase Inhibitor (Human) in the lungs, one inhalation per day over 4 weeks.
Other Names:
|
EXPERIMENTAL: Group 2
Peripheral Deposition Intervention: Alpha1-Proteinase Inhibitor (Human) Dosage: 25 mg in lungs, one inhalation per day over 4 weeks
|
25 mg of Alpha1-Proteinase Inhibitor (Human) in the lungs, one inhalation per day over 4 weeks.
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Change in Free Elastase in Induced Sputum From Baseline to Week 4
Time Frame: Baseline vs Week 4
|
Baseline vs Week 4
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Change in Alpha-1-anti-trypsin (A1AT) Activity in Induced Sputum From Baseline at Week 4
Time Frame: Baseline vs Week 4
|
Baseline vs Week 4
|
Change in Total Immunoglobulin G (IgG) Fragments in Induced Sputum From Baseline at Week 4
Time Frame: Baseline vs Week 4
|
Baseline vs Week 4
|
Change in Total Bacterial Load in Induced Sputum From Baseline to Week 4
Time Frame: Week 4
|
Week 4
|
Change in Pseudomonas Load in Induced Sputum From Baseline at Week 4
Time Frame: Baseline vs Week 4
|
Baseline vs Week 4
|
Change in Neutrophil Number in Induced Sputum From Baseline at Week 4
Time Frame: Baseline vs Week 4
|
Baseline vs Week 4
|
Collaborators and Investigators
Sponsor
Investigators
- Principal Investigator: Matthias Griese, MD, Kinderklinik und Kinderpoliklinik im Haunerschen Kinderspital
Publications and helpful links
Helpful Links
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
- Genetic Diseases, Inborn
- Pancreatic Diseases
- Fibrosis
- Cystic Fibrosis
- Molecular Mechanisms of Pharmacological Action
- Enzyme Inhibitors
- Serine Proteinase Inhibitors
- Trypsin Inhibitors
- Protease Inhibitors
- Alpha 1-Antitrypsin
Other Study ID Numbers
- 100452
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.
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