Cystic FibrosisPhase 2NCT02010411
Alpha1 Antitrypsin Aerosol Therapy in Cystic Fibrosis
A Phase 2 study testing Prolastin (drug) in people with Cystic Fibrosis, run by Université de Sherbrooke at 1 site in Canada.
Trial summary
The hypothesis being tested is that inhibition of the enzyme known as elastase in the airways of patients with cystic fibrosis will help decrease the number of bacteria. Alpha1 antitrypsin, an elastase inhibitor, will be given to patients with cystic fibrosis by aerosol therapy twice in 1 day and sputum will be… Read more about this study
- Who is it for?
- Ages 14 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Prolastin (drug)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 17 participants
- Where
- 1 siteCanada
- Study dates
- Jun 2004to Oct 2005
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 14 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of cystic fibrosis
- Age 14 years and older
- Women must have a negative pregnancy test and used effective contraception
- Must be able to produce sputum
- Sputum culture positive for Pseudomonas aeruginosa
Who cannot join
- -
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Effects of Prolastin Aerosol Therapy on Bacterial Density in the Airways of Patients With Cystic Fibrosis
The hypothesis being tested is that inhibition of the enzyme known as elastase in the airways of patients with cystic fibrosis will help decrease the number of bacteria. Alpha1 antitrypsin, an elastase inhibitor, will be given to patients with cystic fibrosis by aerosol therapy twice in 1 day and sputum will be collected to measure the density of bacteria
Read the detailed description
Cystic fibrosis is usually characterized by chronic bacterial infections of the airways. Neutrophils release the enzyme elastase in the airways and this enzyme can prevent the ingestion and killing of bacteria by the airway phagocytic cells. The hypothesis being tested is that inhibition of elastase in the airways will help neutrophils decrease the number of bacteria. Each subject with cystic fibrosis will first undergo aerosol therapy with a sterile saline solution and sputum will be collected 2, 4 and 6 hours after the aerosol therapy to measure the density of bacteria. Subsequently, alpha1 antitrypsin, an elastase inhibitor, will be given to the same patients by aerosol therapy twice in 1 day and sputum will be collected at 2, 4 and 6 hours after treatment to measure the density of bacteria. The results will be compared to those obtained after after aerosol therapy with saline solution.
Study phase II
Study type Interventional
Study design Purpose - treatment Allocation - nonrandomized trial Masking - open Control - active Assignment - cross-over Endpoint - efficacy
Primary Outcome Measure Bacterial density in sputum as determined by colony forming units at 2, 4 and 6 hours after Prolastin therapy.
- Conditions
- Cystic Fibrosis
- Being tested
- Prolastin (drug)
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Prolastin (drug)Drug
Also called Alpha1 antitrypsin
Outcomes compared to saline or no intervention in each subject.
| Study group | Role | What they receive |
|---|---|---|
| Prolastin | Active comparator | Prolastin 250 mg nebulized BID, 10 days Receives: Drug: Prolastin (drug) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Bacterial density in sputum as determined by colony forming units at 2, 4 and 6 hours after Prolastin therapy.
Measured: 6 hours after Prolastin
Secondary outcomes (3)
Neutrophil burden in airways as determined by sputum myeloperoxidase;
Measured: 6 hours after Prolastin
Sputum elastase activity
Measured: 6 hours after Prolastin
Alpha1 antitrypsin in sputum
Measured: 6 hours after Prolastin
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Dec 2013 (estimated)
- Study startJun 2004
- Primary completionOct 2004
- Study completionOct 2005
Why it stopped: Technical problems with measure of CFU's. Outcomes published: PMID:16986483
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Université de SherbrookeThe sponsor's other registered studies.
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- Université de Sherbrooke
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, André Cantin, Université de Sherbrooke
- Investigators
- Andre Cantin, md, Principal investigator, Centre de recherche du Centre hospitalier universitaire de Sherbrooke
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02010411
- Sponsor's protocol ID
- 04-038
- Status last verified
- Dec 2013
- First submitted
- 9 Dec 2013
- Last updated
- 12 Dec 2013
- Results posted
- No
- Data monitoring committee
- No
Publications
McElvaney NG, Hubbard RC, Birrer P, Chernick MS, Caplan DB, Frank MM, Crystal RG. Aerosol alpha 1-antitrypsin treatment for cystic fibrosis. Lancet. 1991 Feb 16;337(8738):392-4. doi: 10.1016/0140-6736(91)91167-s.
PubMed 1671425
Study at a glance
From the registry record, last updated by the sponsor on 12 Dec 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effects of Prolastin Aerosol Therapy on Bacterial Density in the Airways of Patients With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT02010411
- Acronym / sponsor ID
- 04-038
- Conditions
- Cystic Fibrosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Technical problems with measure of CFU's. Outcomes published: PMID:16986483
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Université de SherbrookeUniversity, hospital or other
- Study start
- Jun 2004
- Primary completion
- Oct 2004 (actual)
- Study completion
- Oct 2005 (actual)
- First posted
- 12 Dec 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The hypothesis being tested is that inhibition of the enzyme known as elastase in the airways of patients with cystic fibrosis will help decrease the number of bacteria. Alpha1 antitrypsin, an elastase inhibitor, will be given to patients with cystic fibrosis by aerosol therapy twice in 1 day and sputum will be collected to measure the density of bacteria
Detailed description
Cystic fibrosis is usually characterized by chronic bacterial infections of the airways. Neutrophils release the enzyme elastase in the airways and this enzyme can prevent the ingestion and killing of bacteria by the airway phagocytic cells. The hypothesis being tested is that inhibition of elastase in the airways will help neutrophils decrease the number of bacteria. Each subject with cystic fibrosis will first undergo aerosol therapy with a sterile saline solution and sputum will be collected 2, 4 and 6 hours after the aerosol therapy to measure the density of bacteria. Subsequently, alpha1 antitrypsin, an elastase inhibitor, will be given to the same patients by aerosol therapy twice in 1 day and sputum will be collected at 2, 4 and 6 hours after treatment to measure the density of bacteria. The results will be compared to those obtained after after aerosol therapy with saline solution.
Study phase II
Study type Interventional
Study design Purpose - treatment Allocation - nonrandomized trial Masking - open Control - active Assignment - cross-over Endpoint - efficacy
Primary Outcome Measure Bacterial density in sputum as determined by colony forming units at 2, 4 and 6 hours after Prolastin therapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Prolastin | Active comparator | Drug: Prolastin (drug)Prolastin 250 mg nebulized BID, 10 days |
Who may qualify
- Ages
- 14 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Diagnosis of cystic fibrosis
- Age 14 years and older
- Women must have a negative pregnancy test and used effective contraception
- Must be able to produce sputum
- Sputum culture positive for Pseudomonas aeruginosa
Key exclusion criteria (1 listed)
- -
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 |
|---|---|---|
| Prolastin (drug)Also: Alpha1 antitrypsinOutcomes compared to saline or no intervention in each subject. | Drug | Prolastin |
Primary outcome
Bacterial density in sputum as determined by colony forming units at 2, 4 and 6 hours after Prolastin therapy.
Time frame: 6 hours after Prolastin
3 secondary outcomes
Neutrophil burden in airways as determined by sputum myeloperoxidase;
Time frame: 6 hours after Prolastin
Sputum elastase activity
Time frame: 6 hours after Prolastin
Alpha1 antitrypsin in sputum
Time frame: 6 hours after Prolastin
Results and safety
The study was stopped early (reason given: Technical problems with measure of CFU's. Outcomes published: PMID:16986483). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Dec 2013.
- Background: PubMed 1671425McElvaney NG, Hubbard RC, Birrer P, Chernick MS, Caplan DB, Frank MM, Crystal RG. Aerosol alpha 1-antitrypsin treatment for cystic fibrosis. Lancet. 1991 Feb 16;337(8738):392-4. doi: 10.1016/0140-6736(91)91167-s.
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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.
Andre Cantin, md principal investigator
Centre de recherche du Centre hospitalier universitaire de Sherbrooke
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Prolastin (drug) in people with Cystic Fibrosis, run by Université de Sherbrooke at 1 site in Canada.
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 5 inclusion and 1 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: stopped early, as of the sponsor's last update on 12 Dec 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
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?
The study was stopped early (reason given: Technical problems with measure of CFU's. Outcomes published: PMID:16986483). Results are not available in the source record.
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?
Bacterial density in sputum as determined by colony forming units at 2, 4 and 6 hours after Prolastin therapy. (time frame: 6 hours after Prolastin). 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 NCT02010411, 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 12 Dec 2013.