Cystic FibrosisPhase 1NCT01347190
Safety and Tolerability Study of Liquid Alpha1 Proteinase Inhibitor (API) in Subjects With Cystic Fibrosis
A Phase 1 study testing CR002 Liquid API in people with Cystic Fibrosis, run by CSL Behring at 11 sites in 4 countries.
Trial summary
Study to assess safety and tolerability of a single dose of study-drug administered to Cystic Fibrosis (CF) patients. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- CR002 Liquid API
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 25 participants
- Where
- 11 sitesin 4 countries
- Study dates
- Apr 2011to Aug 2011
- Phase
- Phase 1
- 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Have a confirmed diagnosis of CF: one or more clinical findings consistent with CF and at least one of the following:
- Sweat chloride ≥ 60 mEq/L by quantitative pilocarpine iontophoresis test OR
- A genotype with two identifiable mutations consistent with CF (∆F508 homozygous or two alleles known to cause a class I, II, or III mutation)
- Have an FEV1 ≥30% of predicted normal as defined by age, gender, and height
Who cannot join
- Oxygen saturation <90%
- Changed in treatment regimen within 2 weeks prior to screening
- Antibiotics regimen change < 4 weeks before screening
- Persistent colonization with Burkholderia cepacia
- Serum IgA < 50% of lower level of normal
Trial locations and contacts
11 sites in 4 countries.
11 sites in 4 countries
Poland5 sites
Site 24
Bialystok
Site 22
Gdansk
Site 21
Poznan
Site 20
Rabka-Zdrój
Site 23
Warsaw
United Kingdom3 sites
Site 42
Papworth Hospital
Site 40
Penarth
Site 41
Southampton
About this study
Official title: A Double Blind, Randomized, Placebo Controlled, Single Dose, Phase I Study of the Safety and Tolerability of Alpha1 Proteinase Inhibitor (Human) Inhalation Solution (CR002) in Subjects With Cystic Fibrosis
Study to assess safety and tolerability of a single dose of study-drug administered to Cystic Fibrosis (CF) patients.
- Conditions
- Cystic Fibrosis
- Being tested
- CR002 Liquid API, Placebo
- Keywords
- Cystic Fibrosis
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CR002 Liquid APIBiological
A single nominal dose of CR002 (three different dose groups) will be inhaled as an aerosol mist produced by an I-neb Adaptive Aerosol Delivery (AAD) System.
PlaceboBiological
A single nominal dose of Placebo will be inhaled as an aerosol mist produced by an I-neb Adaptive Aerosol Delivery (AAD) System.
| Study group | Role | What they receive |
|---|---|---|
| Liquid API | Experimental | Not described Receives: Biological: CR002 Liquid API |
| Placebo | Placebo comparator | Not described Receives: Biological: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency of Adverse events
Measured: 44 Days (Day -21 to Day 22)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 May 2011 (estimated)
- Study startApr 2011
- Primary completionAug 2011
- Study completionAug 2011
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.
- More trials run by CSL BehringThe 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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- CSL Behring
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Program director Clinical R&D, Study director, CSL Behring
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01347190
- Sponsor's protocol ID
- CR002_1004
- Other IDs
- 2010-022671-60
- Status last verified
- Apr 2012
- First submitted
- 3 May 2011
- Last updated
- 5 Apr 2012
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Apr 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Double Blind, Randomized, Placebo Controlled, Single Dose, Phase I Study of the Safety and Tolerability of Alpha1 Proteinase Inhibitor (Human) Inhalation Solution (CR002) in Subjects With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT01347190
- Acronym / sponsor ID
- CR002_1004
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic Fibrosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- CSL BehringIndustry (pharma, biotech, devices)
- Study start
- Apr 2011
- Primary completion
- Aug 2011 (actual)
- Study completion
- Aug 2011 (actual)
- First posted
- 4 May 2011 (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.
Study to assess safety and tolerability of a single dose of study-drug administered to Cystic Fibrosis (CF) patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Liquid API | Experimental | Biological: CR002 Liquid API |
| Placebo | Placebo comparator | Biological: Placebo |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Have a confirmed diagnosis of CF: one or more clinical findings consistent with CF and at least one of the following:
- Have an FEV1 ≥30% of predicted normal as defined by age, gender, and height
Key exclusion criteria (5 listed)
- Oxygen saturation <90%
- Changed in treatment regimen within 2 weeks prior to screening
- Antibiotics regimen change < 4 weeks before screening
- Persistent colonization with Burkholderia cepacia
- Serum IgA < 50% of lower level of normal
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 |
|---|---|---|
| CR002 Liquid APIA single nominal dose of CR002 (three different dose groups) will be inhaled as an aerosol mist produced by an I-neb Adaptive Aerosol Delivery (AAD) System. | Biological | Liquid API |
| PlaceboA single nominal dose of Placebo will be inhaled as an aerosol mist produced by an I-neb Adaptive Aerosol Delivery (AAD) System. | Biological | Placebo |
Primary outcome
Frequency of Adverse events
Time frame: 44 Days (Day -21 to Day 22)
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 5 Apr 2012.
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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.
Program director Clinical R&D study director
CSL Behring
11 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing CR002 Liquid API in people with Cystic Fibrosis, run by CSL Behring at 11 sites in 4 countries.
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 4 inclusion and 5 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 5 Apr 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in Poland, United Kingdom, Bulgaria, Hungary. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Frequency of Adverse events (time frame: 44 Days (Day -21 to Day 22)). 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 5 Apr 2012.