Cystic FibrosisPhase 2CF2NCT00486837
Deposition of Inhaled Prolastin in Cystic Fibrosis Patients
A Phase 2 study testing Alpha1-Proteinase Inhibitor (Human) in people with Cystic Fibrosis, run by Grifols Therapeutics LLC.
Trial 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). Read more about this study
- Who is it for?
- Ages 8 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Alpha1-Proteinase Inhibitor (Human)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 72 participants
- Where
- No sites listed yet
- Study dates
- Dec 2003to Jun 2004
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
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- 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)
Who cannot join
- 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.
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- 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
About this study
Official title: Multicenter, Randomized, Parallel Group Study to Investigate the Optimal Deposition Site for Inhaled Prolastin® in Patients With Cystic Fibrosis (CF)
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.
Read the 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.
- Conditions
- Cystic Fibrosis
- Being tested
- Alpha1-Proteinase Inhibitor (Human)
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Alpha1-Proteinase Inhibitor (Human)Drug
Also called Prolastin®, Alpha-1 antitrypsin (AAT), BAY x 5747, BAY 10-5233
25 mg of Alpha1-Proteinase Inhibitor (Human) in the lungs, one inhalation per day over 4 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Group 1 | Experimental | Bronchial Deposition Intervention: Alpha1-Proteinase Inhibitor (Human) Dosage: 25 mg in lungs, one inhalation per day over 4 weeks Receives: Drug: Alpha1-Proteinase Inhibitor (Human) |
| Group 2 | Experimental | Peripheral Deposition Intervention: Alpha1-Proteinase Inhibitor (Human) Dosage: 25 mg in lungs, one inhalation per day over 4 weeks Receives: Drug: Alpha1-Proteinase Inhibitor (Human) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Free Elastase in Induced Sputum From Baseline to Week 4
Measured: Baseline vs Week 4
Secondary outcomes (5)
Change in Alpha-1-anti-trypsin (A1AT) Activity in Induced Sputum From Baseline at Week 4
Measured: Baseline vs Week 4
Change in Total Immunoglobulin G (IgG) Fragments in Induced Sputum From Baseline at Week 4
Measured: Baseline vs Week 4
Change in Total Bacterial Load in Induced Sputum From Baseline to Week 4
Measured: Week 4
Change in Pseudomonas Load in Induced Sputum From Baseline at Week 4
Measured: Baseline vs Week 4
Change in Neutrophil Number in Induced Sputum From Baseline at Week 4
Measured: Baseline vs Week 4
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jun 2007 (estimated)
- Study startDec 2003
- Primary completionJun 2004
- Study completionJun 2004
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 72 participants (actual)
Sponsor and investigators
- Lead sponsor
- Grifols Therapeutics LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Matthias Griese, MD, Principal investigator, Kinderklinik und Kinderpoliklinik im Haunerschen Kinderspital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00486837
- Sponsor's protocol ID
- 100452
- Acronym
- CF2
- Status last verified
- Aug 2014
- First submitted
- 13 Jun 2007
- Last updated
- 21 Aug 2014
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
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.
PubMed 17050563
Study at a glance
From the registry record, last updated by the sponsor on 21 Aug 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicenter, Randomized, Parallel Group Study to Investigate the Optimal Deposition Site for Inhaled Prolastin® in Patients With Cystic Fibrosis (CF)
- ClinicalTrials.gov ID
- NCT00486837
- Acronym / sponsor ID
- CF2 / 100452
- Conditions
- Cystic Fibrosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 72 participants (actual)
- Lead sponsor
- Grifols Therapeutics LLCIndustry (pharma, biotech, devices)
- Study start
- Dec 2003
- Primary completion
- Jun 2004 (actual)
- Study completion
- Jun 2004 (actual)
- First posted
- 15 Jun 2007 (estimated)
- Results
- Posted 21 Aug 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group 1 | Experimental | Drug: Alpha1-Proteinase Inhibitor (Human)Bronchial Deposition Intervention: Alpha1-Proteinase Inhibitor (Human) Dosage: 25 mg in lungs, one inhalation per day over 4 weeks |
| Group 2 | Experimental | Drug: Alpha1-Proteinase Inhibitor (Human)Peripheral Deposition Intervention: Alpha1-Proteinase Inhibitor (Human) Dosage: 25 mg in lungs, one inhalation per day over 4 weeks |
Who may qualify
- Ages
- 8 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- 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
Key exclusion criteria (13 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Alpha1-Proteinase Inhibitor (Human)Also: Prolastin®, Alpha-1 antitrypsin (AAT), BAY x 5747, BAY 10-523325 mg of Alpha1-Proteinase Inhibitor (Human) in the lungs, one inhalation per day over 4 weeks. | Drug | Group 1, Group 2 |
Primary outcome
Change in Free Elastase in Induced Sputum From Baseline to Week 4
Time frame: Baseline vs Week 4
5 secondary outcomes
Change in Alpha-1-anti-trypsin (A1AT) Activity in Induced Sputum From Baseline at Week 4
Time frame: Baseline vs Week 4
Change in Total Immunoglobulin G (IgG) Fragments in Induced Sputum From Baseline at Week 4
Time frame: Baseline vs Week 4
Change in Total Bacterial Load in Induced Sputum From Baseline to Week 4
Time frame: Week 4
Change in Pseudomonas Load in Induced Sputum From Baseline at Week 4
Time frame: Baseline vs Week 4
Change in Neutrophil Number in Induced Sputum From Baseline at Week 4
Time frame: Baseline vs Week 4
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Aug 2014.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 17050563Griese 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.
- FDA Approved Product Labeling InformationLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Matthias Griese, MD principal investigator
Kinderklinik und Kinderpoliklinik im Haunerschen Kinderspital
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Alpha1-Proteinase Inhibitor (Human) in people with Cystic Fibrosis, run by Grifols Therapeutics LLC.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 21 Aug 2014. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 21 Aug 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Free Elastase in Induced Sputum From Baseline to Week 4 (time frame: Baseline vs Week 4). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00486837, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Aug 2014.