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Deposition of Inhaled Prolastin in Cystic Fibrosis Patients (CF2)

Primary Purpose

Cystic Fibrosis

Status

Completed

Phase

Phase 2

Locations

Study Type

Interventional

Intervention

Alpha1-Proteinase Inhibitor (Human)

About this trial

This is an interventional treatment trial for Cystic Fibrosis

Eligibility Criteria

8 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Arm Label

Group 1

Group 2

Arm Description

Bronchial Deposition Intervention: Alpha1-Proteinase Inhibitor (Human) Dosage: 25 mg in lungs, one inhalation per day over 4 weeks

Peripheral Deposition Intervention: Alpha1-Proteinase Inhibitor (Human) Dosage: 25 mg in lungs, one inhalation per day over 4 weeks

Outcomes

Primary Outcome Measures

Change in Free Elastase in Induced Sputum From Baseline to Week 4

Secondary Outcome Measures

Change in Alpha-1-anti-trypsin (A1AT) Activity in Induced Sputum From Baseline at Week 4

Change in Total Immunoglobulin G (IgG) Fragments in Induced Sputum From Baseline at Week 4

Change in Total Bacterial Load in Induced Sputum From Baseline to Week 4

Change in Pseudomonas Load in Induced Sputum From Baseline at Week 4

Change in Neutrophil Number in Induced Sputum From Baseline at Week 4

Full Information

NCT ID

NCT00486837

First Posted

June 13, 2007

Last Updated

August 5, 2014

Sponsor

Grifols Therapeutics LLC

1. Study Identification

Unique Protocol Identification Number

NCT00486837

Brief Title

Deposition of Inhaled Prolastin in Cystic Fibrosis Patients

Acronym

CF2

Official Title

Multicenter, Randomized, Parallel Group Study to Investigate the Optimal Deposition Site for Inhaled Prolastin® in Patients With Cystic Fibrosis (CF)

Study Type

Interventional

2. Study Status

Record Verification Date

August 2014

Overall Recruitment Status

Completed

Study Start Date

December 2003 (undefined)

Primary Completion Date

June 2004 (Actual)

Study Completion Date

June 2004 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Grifols Therapeutics LLC

4. Oversight

Data Monitoring Committee

5. Study Description

Brief 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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Cystic Fibrosis

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Randomized

Enrollment

72 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Group 1

Arm Type

Experimental

Arm Description

Bronchial Deposition Intervention: Alpha1-Proteinase Inhibitor (Human) Dosage: 25 mg in lungs, one inhalation per day over 4 weeks

Arm Title

Group 2

Arm Type

Experimental

Arm Description

Peripheral Deposition Intervention: Alpha1-Proteinase Inhibitor (Human) Dosage: 25 mg in lungs, one inhalation per day over 4 weeks

Intervention Type

Drug

Intervention Name(s)

Alpha1-Proteinase Inhibitor (Human)

Other Intervention Name(s)

Prolastin®, Alpha-1 antitrypsin (AAT), BAY x 5747, BAY 10-5233, TAL-05-00007, A1AT, NDC 13533-601-30, NDC 13533-601-35

Intervention Description

25 mg of Alpha1-Proteinase Inhibitor (Human) in the lungs, one inhalation per day over 4 weeks.

Primary Outcome Measure Information:

Title

Change in Free Elastase in Induced Sputum From Baseline to Week 4

Time Frame

Baseline vs Week 4

Secondary Outcome Measure Information:

Title

Change in Alpha-1-anti-trypsin (A1AT) Activity in Induced Sputum From Baseline at Week 4

Time Frame

Baseline vs Week 4

Title

Change in Total Immunoglobulin G (IgG) Fragments in Induced Sputum From Baseline at Week 4

Time Frame

Baseline vs Week 4

Title

Change in Total Bacterial Load in Induced Sputum From Baseline to Week 4

Time Frame

Week 4

Title

Change in Pseudomonas Load in Induced Sputum From Baseline at Week 4

Time Frame

Baseline vs Week 4

Title

Change in Neutrophil Number in Induced Sputum From Baseline at Week 4

Time Frame

Baseline vs Week 4

10. Eligibility

Sex

All

Minimum Age & Unit of Time

8 Years

Accepts Healthy Volunteers

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Matthias Griese, MD

Organizational Affiliation

Kinderklinik und Kinderpoliklinik im Haunerschen Kinderspital

Official's Role

Principal Investigator

12. IPD Sharing Statement

Citations:

PubMed Identifier

17050563

Citation

Griese M, Latzin P, Kappler M, Weckerle K, Heinzlmaier T, Bernhardt T, Hartl D. alpha1-Antitrypsin inhalation reduces airway inflammation in cystic fibrosis patients. Eur Respir J. 2007 Feb;29(2):240-50. doi: 10.1183/09031936.00047306. Epub 2006 Oct 18.

Results Reference

result

Links:

URL

http://www.talecris-pi.info/inserts/Prolastin.pdf

Description

FDA Approved Product Labeling Information

Learn more about this trial

Deposition of Inhaled Prolastin in Cystic Fibrosis Patients

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