Overview

Deposition of Inhaled Prolastin in Cystic Fibrosis Patients

Status:
Completed
Trial end date:
2004-06-01
Target enrollment:
0
Participant gender:
All
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.
Phase:
Phase 2
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Grifols Therapeutics Inc.
Grifols Therapeutics LLC
Treatments:
Alpha 1-Antitrypsin
Protease Inhibitors
Criteria
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