Cystic FibrosisPhase 2NCT00499837
Phase II Study of the Safety and Efficacy of Inhaled Alpha-1 Antitrypsin (AAT ) in Cystic Fibrosis Patients
A Phase 2 study testing Aerosolized, human, plasma-derived Alpha-1 Antitrypsin in people with Cystic Fibrosis, run by Kamada, Ltd. at 1 site in Israel.
Trial summary
Cystic Fibrosis (CF) is an inherited disorder in which mucus-secreting glands in the lungs produce considerable quantity of thick, sticky secretions that clog the airways, promote bacterial growth and lead to chronic obstruction, inflammation and destruction of the airways. Read more about this study
- Who is it for?
- Ages 5 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Aerosolized, human, plasma-derived Alpha-1 Antitrypsin
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 21 participants
- Where
- 1 siteIsrael
- Study dates
- Sep 2007to Jul 2008
- 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 5 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of CF by clinical symptoms and positive sweat test or disease inducing mutation.
- Age >5 yrs
- Proven ability to perform reproducible PFTs
- FEV1 >25% predicted
- Steady disease state for 3 months and no decrease in lung function exceeding 10% during that period
- Colonization
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- Stable concomitant therapy >2 weeks prior to visit 1 and during the study
- Non-tobacco user of any kind
- Ability for sputum induction
- Written informed consent
Who cannot join
- Severe CF with an FEV1 of <25% predicted
- History of lung transplant
- Active allergic bronchopulmonary aspergillosis (ABPA)
- Treatment with additional antibiotics (beyond standard CF treatment) for a period of 14 days before study entry (routine antibiotics permitted)
- Treatment with additional oral and/or IV steroids (beyond standard CF treatment) for a period of 14 days before study entry (screening day)
- Known hypersensitivity to plasma products
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- IgA deficiency
- Uncontrolled hypertension
- Lung surgery in the previous two years
- Being on any thoracic surgery waiting list
- Severe concomitant disease
- Hospitalization within 1 month before study entry, not due to an airway disease
- Severe liver cirrhosis with ascites
- Hypersplenism
- Grade III/IV oesophageal varices
- Active pulmonary exacerbation within the 4 weeks prior to screening
- History of significant hemoptysis within the previous year
- Use of tobacco products or recreational drugs
- Pregnancy or breastfeeding
- Any serious or active medical or psychiatric illness which, in the opinion of the investigator, would interfere with patient treatment, assessment, or compliance with the protocol.
- Being a female of child-bearing age without adequate contraception
- Participation in research study within 1 month
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Double-Blind, Randomized, Placebo-Controlled, Phase II Study of the Safety and Efficacy of Inhaled Alpha-1 Antitrypsin (AAT ) in Cystic Fibrosis Patients
Cystic Fibrosis (CF) is an inherited disorder in which mucus-secreting glands in the lungs produce considerable quantity of thick, sticky secretions that clog the airways, promote bacterial growth and lead to chronic obstruction, inflammation and destruction of the airways.
The purpose of this study is to collect data about the resolution of the chronic inflammatory state in addition to assure the safety of the therapy in CF patients.
Read the detailed description
In CF patients the unregulated inflammatory response overwhelms the normal protease (elastase)/antiprotease (AAT) balance, leading to the accumulation of elastase in the lung, destruction of the lung architecture, severe pulmonary dysfunction, and ultimately death.
Administration of AAT is to address the elastase/antiprotease imbalance in order to prevent destruction of the lung tissue and reduce the inflammatory dysregulation that causes pulmonary dysfunction.
- Conditions
- Cystic Fibrosis
- Being tested
- Aerosolized, human, plasma-derived Alpha-1 Antitrypsin
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Aerosolized, human, plasma-derived Alpha-1 AntitrypsinDrug
| Study group | Role | What they receive |
|---|---|---|
| 80 mg/kg AAT inhaled | Experimental | 80 mg/kg AAT inhaled Receives: Drug: Aerosolized, human, plasma-derived Alpha-1 Antitrypsin |
| Placebo inhaled | Placebo comparator | Placebo inhaled Receives: Drug: Aerosolized, human, plasma-derived Alpha-1 Antitrypsin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and airway inflammation
Measured: days 1,7,14,21,28,35,42,49,56,63
Secondary outcomes (1)
Sputum microbiology, pulmonary function and serum CRP
Measured: days 1,7,14,21,28,35,42,49,56,63
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jul 2007 (estimated)
- Study startSep 2007
- Primary completionJul 2008
- Study completionJul 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kamada, Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Eitan Kerem, MD, Principal investigator, Hadassah Hebrew University, Medical Center, Mt. Scopus, Jerusalem
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00499837
- Sponsor's protocol ID
- Kamada-AAT (inhaled)-003
- Status last verified
- Apr 2016
- First submitted
- 11 Jul 2007
- Last updated
- 9 Jun 2016
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Jun 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Double-Blind, Randomized, Placebo-Controlled, Phase II Study of the Safety and Efficacy of Inhaled Alpha-1 Antitrypsin (AAT ) in Cystic Fibrosis Patients
- ClinicalTrials.gov ID
- NCT00499837
- Acronym / sponsor ID
- Kamada-AAT (inhaled)-003
- Conditions
- Cystic Fibrosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 21 participants (actual)
- Lead sponsor
- Kamada, Ltd.Industry (pharma, biotech, devices)
- Study start
- Sep 2007
- Primary completion
- Jul 2008 (actual)
- Study completion
- Jul 2008 (actual)
- First posted
- 12 Jul 2007 (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.
Cystic Fibrosis (CF) is an inherited disorder in which mucus-secreting glands in the lungs produce considerable quantity of thick, sticky secretions that clog the airways, promote bacterial growth and lead to chronic obstruction, inflammation and destruction of the airways.
The purpose of this study is to collect data about the resolution of the chronic inflammatory state in addition to assure the safety of the therapy in CF patients.
Detailed description
In CF patients the unregulated inflammatory response overwhelms the normal protease (elastase)/antiprotease (AAT) balance, leading to the accumulation of elastase in the lung, destruction of the lung architecture, severe pulmonary dysfunction, and ultimately death.
Administration of AAT is to address the elastase/antiprotease imbalance in order to prevent destruction of the lung tissue and reduce the inflammatory dysregulation that causes pulmonary dysfunction.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 80 mg/kg AAT inhaled | Experimental | Drug: Aerosolized, human, plasma-derived Alpha-1 Antitrypsin80 mg/kg AAT inhaled |
| Placebo inhaled | Placebo comparator | Drug: Aerosolized, human, plasma-derived Alpha-1 AntitrypsinPlacebo inhaled |
Who may qualify
- Ages
- 5 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- Diagnosis of CF by clinical symptoms and positive sweat test or disease inducing mutation.
- Age >5 yrs
- Proven ability to perform reproducible PFTs
- FEV1 >25% predicted
- Steady disease state for 3 months and no decrease in lung function exceeding 10% during that period
Key exclusion criteria (22 listed)
- Severe CF with an FEV1 of <25% predicted
- History of lung transplant
- Active allergic bronchopulmonary aspergillosis (ABPA)
- Treatment with additional antibiotics (beyond standard CF treatment) for a period of 14 days before study entry (routine antibiotics permitted)
- Treatment with additional oral and/or IV steroids (beyond standard CF treatment) for a period of 14 days before study entry (screening day)
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 |
|---|---|---|
| Aerosolized, human, plasma-derived Alpha-1 Antitrypsin | Drug | 80 mg/kg AAT inhaled, Placebo inhaled |
Primary outcome
Safety and airway inflammation
Time frame: days 1,7,14,21,28,35,42,49,56,63
1 secondary outcome
Sputum microbiology, pulmonary function and serum CRP
Time frame: days 1,7,14,21,28,35,42,49,56,63
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Jun 2016.
- Individual participant dataSharing planned: No
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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.
Eitan Kerem, MD principal investigator
Hadassah Hebrew University, Medical Center, Mt. Scopus, Jerusalem
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 Aerosolized, human, plasma-derived Alpha-1 Antitrypsin in people with Cystic Fibrosis, run by Kamada, Ltd. at 1 site in Israel.
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 10 inclusion and 22 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 9 Jun 2016. 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 Israel. 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. Some participants in this study may receive a placebo. 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Safety and airway inflammation (time frame: days 1,7,14,21,28,35,42,49,56,63). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 9 Jun 2016.