Deposition of Inhaled Prolastin in Cystic Fibrosis Patients
Information source: Talecris Biotherapeutics
Information obtained from ClinicalTrials.gov on June 20, 2008
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Cystic Fibrosis
Intervention: Alpha1-Proteinase Inhibitor (Human) (Drug)
Phase: Phase 2
Status: Completed
Sponsored by: Talecris Biotherapeutics Official(s) and/or principal investigator(s):
Matthias Griese, MD, Principal Investigator, Affiliation: Kinderklinik und Kinderpoliklinik im Haunerschen Kinderspital
Summary
The objective of this trial is to determine the optimal region of the lung for depositing
Prolastin (alpha-1 antitrypsin; AAT) by inhalation in order to treat cystic fibrosis (CF).
The AKITA® nebulizer has settings which can be varied to target the inhaled drug to either
the deep lung or to the upper airways in a one to one randomization. The study will measure
how much of the activity of the enzyme elastase is inhibited by AAT.
Clinical Details
Official title: Multicenter, Randomized, Parallel Group Study to Investigate the Optimal Deposition Site for Inhaled Prolastin® in Patients With Cystic Fibrosis (CF)
Study design: Treatment, Randomized, Open Label, Dose Comparison, Parallel Assignment, Efficacy Study
Primary outcome: change in free elastase in induced sputum
Secondary outcome: Standard lung function parameters (FEV1, FVC, FEV1/FVC, MEF)AAT activity in induced sputum total IgG fragments in induced sputum total bacterial load in induced sputum Pseudomonas load in induced sputum Neutrophil number in induced sputum
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.
Eligibility
Minimum age: 8 Years.
Maximum age: N/A.
Gender(s): Both.
Criteria:
Inclusion Criteria:
- Patient with diagnosis of CF
- Age >= 8 years
- 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 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 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
Locations and Contacts
Additional Information
FDA Approved Product Labeling Information FDA Enforcement Report Index (Class I, Class II Recall, Market Alerts and Medical Product Safety Alerts) CF2 Synopsis of Study Results
Starting date: December 2003
Ending date: June 2004
Last updated: May 7, 2008
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