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