Phase 2 N=72 Randomized Treatment

Deposition of Inhaled Prolastin in Cystic Fibrosis Patients

Cystic Fibrosis

Bottom Line

View on ClinicalTrials.gov: NCT00486837 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

Aug 2014

Primary outcome: Primary: Change in Free Elastase in Induced Sputum From Baseline to Week 4 — -7.42; 6.11 ug/mL — p=0.197

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Alpha1-Proteinase Inhibitor (Human) (Drug)
Age: Pediatric, Adult, Older Adult · 8+ yrs
Sex: All
Sponsor: Grifols Therapeutics LLC
Primary completion: Jun 2004

Outcome Measures

Outcome	Result	p-value
PRIMARY Change in Free Elastase in Induced Sputum From Baseline to Week 4	-7.42; 6.11	0.197
SECONDARY Change in Alpha-1-anti-trypsin (A1AT) Activity in Induced Sputum From Baseline at Week 4	16741; 6469	—
SECONDARY Change in Total Immunoglobulin G (IgG) Fragments in Induced Sputum From Baseline at Week 4	0.005; -0.48	—
SECONDARY Change in Total Bacterial Load in Induced Sputum From Baseline to Week 4	22673275.8; 5021353.2	—
SECONDARY Change in Pseudomonas Load in Induced Sputum From Baseline at Week 4	-40.1; -22.3	—
SECONDARY Change in Neutrophil Number in Induced Sputum From Baseline at Week 4	-20.8; -8.2	—

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.

Eligibility 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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00486837). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.