Cystic FibrosisPhase 2NCT01684410
Safety and Tolerability Trial of Inhaled Alpha1-Proteinase Inhibitor (Human), Hydrophobic Chromatography Process (Alpha-1 HC) in Subjects With Cystic Fibrosis
A Phase 2 study testing Alpha-1 HC 100 mg and Alpha-1 HC 200 mg in people with Cystic Fibrosis, run by Grifols Therapeutics LLC at 6 sites in United States.
Trial summary
This was a randomized, double-blind, placebo-controlled, dose escalation study to assess the safety and tolerability of 100 mg and 200 mg of inhaled Alpha-1 HC administered once a day for three weeks in subjects aged 18 years and older with cystic fibrosis (CF). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Alpha-1 HC 100 mg and Alpha-1 HC 200 mg
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 30 participants
- Where
- 6 sitesUnited States
- Study dates
- Aug 2012to Oct 2013
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age 18 years or older.
- Documentation of CF diagnosis.
- Have a pre-bronchodilator FEV1 ≥ 40% of predicted at Visit 1 and have a Visit 2 pre-investigational product FEV1 that is ≥ 40% of predicted and within ± 15% of the Visit 1 result.
- Deemed by the Investigator to be a suitable candidate for serial collection of expectorated sputum.
Who cannot join
- Had a pulmonary exacerbation during the 4 weeks before screening (Visit 1) which required the initiation of new antibiotic treatment
- Have a pulmonary exacerbation during the screening period (between Visit 1 and Visit 2) which requires the initiation of new antibiotic treatment
- FEV1 < 0.59 liters at the screening visit
- Respiratory insufficiency with continuous supplemental oxygen therapy, or carbon dioxide retention
- Elevated aspartate transaminase (AST) or alanine aminotransferase (ALT) that is ≥ 3 times the upper limit of normal for age and gender
- Smoking during the past 6 months
Show all 18Show fewer
- Lung surgery during the past 2 years
- Positive culture for Burkholderia cepacia or mycobacterium during the past two years.
- Active allergic bronchopulmonary aspergillosis
- Pre-treatment sputum collection at Visit 1 or Visit 2 (Randomization) characterized by problems such as inadequate sputum volume or quality.
- Known selective Immunoglobulin A (IgA) deficiency with known antibody against IgA (anti-IgA antibody).
- History of anaphylaxis or severe systemic response to any plasma-derived alpha1-proteinase inhibitor preparation or other blood product(s), or to polysorbates.
- Use of chronic oral steroids during the study. Note: Inhaled corticosteroids that had been administered for at least 4 weeks prior to Visit 1 were permissible during the study.
- Use of chronic, high dose ibuprofen therapy within 3 weeks of screening and at anytime during the study.
- Chronic maintenance therapy with systemic antibiotics within 3 weeks of screening and through last dose of investigational product.
- Use of leukotriene synthesis inhibitor (zileuton) or leukotriene receptor antagonists (montelukast, zafirlukast) within 3 weeks of screening and at anytime during the study.
- Use of roflumilast within 3 weeks of screening and at any time during the study.
- Initiation of a new chronic medication or dosage change of a chronic medication for treatment of cystic fibrosis (example: Kalydeco™ [ivacaftor]) within 3 weeks of screening (Visit 1).
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
The University of Alabama at Birmingham
Birmingham, Alabama, 35233
Children's Hospital Boston
Boston, Massachusetts, 02115
UNC at Chapel Hill
Chapel Hill, North Carolina, 27599
Medical University of South Carolina
Charleston, South Carolina, 29425
Rainbow Babies and Children's Hospital
Cleveland, Ohio, 44106
National Jewish Hospital
Denver, Colorado, 80206
About this study
Official title: A Three Week Dose Escalation, Randomized, Double-Blind, Placebo-Controlled Trial to Assess the Safety and Tolerability of 100 mg or 200 mg of Inhaled Alpha-1 HC, Once a Day in Subjects With Cystic Fibrosis.
This was a randomized, double-blind, placebo-controlled, dose escalation study to assess the safety and tolerability of 100 mg and 200 mg of inhaled Alpha-1 HC administered once a day for three weeks in subjects aged 18 years and older with cystic fibrosis (CF). The treatment duration in this study was intended to provide multi-dose safety information prior to proceeding to longer durations of exposure.
- Conditions
- Cystic Fibrosis
- Being tested
- Alpha-1 HC 100 mg, Placebo, Alpha-1 HC 200 mg
- Keywords
- Cystic Fibrosis, Alpha1-proteinase inhibitor, Alpha1-antitrypsin
- Medical subject headings
- Cystic Fibrosis, alpha 1-Antitrypsin Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Alpha-1 HC 100 mgBiological
Also called Alpha1-proteinase inhibitor, alpha1-antitrypsin
Alpha-1 HC is a sterile, liquid preparation of purified alpha1-proteinase inhibitor prepared from pooled human plasma. Alpha-1 HC 100 mg inhaled once daily for 21 days for a total of 21 inhaled treatments.
PlaceboBiological
Phosphate Buffer Saline with Polysorbate (placebo) composed of the same elements listed for Alpha-1 HC, minus the 50 mg/mL of Alpha-1 HC. Placebo inhaled once daily for 21 days for a total of 21 inhaled treatments.
Alpha-1 HC 200 mgBiological
Also called Alpha1-proteinase inhibitor, alpha1-antitrypsin
Alpha-1 HC is a sterile, liquid preparation of purified alpha1-proteinase inhibitor prepared from pooled human plasma. Alpha-1 HC 200 mg inhaled once daily for 21 days for a total of 21 inhaled treatments.
| Study group | Role | What they receive |
|---|---|---|
| Alpha-1 HC 100 mg | Experimental | 100 mg of aerosolized Alpha-1 HC inhaled daily via nebulizer for 3 weeks. Receives: Biological: Alpha-1 HC 100 mg |
| Alpha-1 HC 200 mg | Experimental | 200 mg of aerosolized Alpha-1 HC inhaled daily via nebulizer for 3 weeks. Receives: Biological: Alpha-1 HC 200 mg |
| Placebo | Placebo comparator | Placebo inhaled daily via nebulizer for 3 weeks. Placebo (phosphate buffer saline with polysorbate). Receives: Biological: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events
adverse event frequency
Measured: 3 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Sep 2012 (estimated)
- Study startAug 2012
- Primary completionOct 2013
- Study completionOct 2013
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.
- 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
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Grifols Therapeutics LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01684410
- Sponsor's protocol ID
- T6005-201
- Status last verified
- Jan 2016
- First submitted
- 6 Sep 2012
- Last updated
- 8 Feb 2016
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 8 Feb 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Three Week Dose Escalation, Randomized, Double-Blind, Placebo-Controlled Trial to Assess the Safety and Tolerability of 100 mg or 200 mg of Inhaled Alpha-1 HC, Once a Day in Subjects With Cystic Fibrosis.
- ClinicalTrials.gov ID
- NCT01684410
- Acronym / sponsor ID
- T6005-201
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic Fibrosis, Alpha1-proteinase inhibitor, Alpha1-antitrypsin
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Grifols Therapeutics LLCIndustry (pharma, biotech, devices)
- Study start
- Aug 2012
- Primary completion
- Oct 2013 (actual)
- Study completion
- Oct 2013 (actual)
- First posted
- 13 Sep 2012 (estimated)
- Results
- Posted 8 Feb 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This was a randomized, double-blind, placebo-controlled, dose escalation study to assess the safety and tolerability of 100 mg and 200 mg of inhaled Alpha-1 HC administered once a day for three weeks in subjects aged 18 years and older with cystic fibrosis (CF). The treatment duration in this study was intended to provide multi-dose safety information prior to proceeding to longer durations of exposure.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Alpha-1 HC 100 mg | Experimental | Biological: Alpha-1 HC 100 mg100 mg of aerosolized Alpha-1 HC inhaled daily via nebulizer for 3 weeks. |
| Alpha-1 HC 200 mg | Experimental | Biological: Alpha-1 HC 200 mg200 mg of aerosolized Alpha-1 HC inhaled daily via nebulizer for 3 weeks. |
| Placebo | Placebo comparator | Biological: PlaceboPlacebo inhaled daily via nebulizer for 3 weeks. Placebo (phosphate buffer saline with polysorbate). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Age 18 years or older.
- Documentation of CF diagnosis.
- Have a pre-bronchodilator FEV1 ≥ 40% of predicted at Visit 1 and have a Visit 2 pre-investigational product FEV1 that is ≥ 40% of predicted and within ± 15% of the Visit 1 result.
- Deemed by the Investigator to be a suitable candidate for serial collection of expectorated sputum.
Key exclusion criteria (18 listed)
- Had a pulmonary exacerbation during the 4 weeks before screening (Visit 1) which required the initiation of new antibiotic treatment
- Have a pulmonary exacerbation during the screening period (between Visit 1 and Visit 2) which requires the initiation of new antibiotic treatment
- FEV1 < 0.59 liters at the screening visit
- Respiratory insufficiency with continuous supplemental oxygen therapy, or carbon dioxide retention
- Elevated aspartate transaminase (AST) or alanine aminotransferase (ALT) that is ≥ 3 times the upper limit of normal for age and gender
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 |
|---|---|---|
| Alpha-1 HC 100 mgAlso: Alpha1-proteinase inhibitor, alpha1-antitrypsinAlpha-1 HC is a sterile, liquid preparation of purified alpha1-proteinase inhibitor prepared from pooled human plasma. Alpha-1 HC 100 mg inhaled once daily for 21 days for a total of 21 inhaled treatments. | Biological | Alpha-1 HC 100 mg |
| PlaceboPhosphate Buffer Saline with Polysorbate (placebo) composed of the same elements listed for Alpha-1 HC, minus the 50 mg/mL of Alpha-1 HC. Placebo inhaled once daily for 21 days for a total of 21 inhaled treatments. | Biological | Placebo |
| Alpha-1 HC 200 mgAlso: Alpha1-proteinase inhibitor, alpha1-antitrypsinAlpha-1 HC is a sterile, liquid preparation of purified alpha1-proteinase inhibitor prepared from pooled human plasma. Alpha-1 HC 200 mg inhaled once daily for 21 days for a total of 21 inhaled treatments. | Biological | Alpha-1 HC 200 mg |
Primary outcome
Adverse Events
Time frame: 3 weeks
adverse event frequency
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Feb 2016.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
The record lists no contact. Use the official record to reach the sponsor.
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Alpha-1 HC 100 mg and Alpha-1 HC 200 mg in people with Cystic Fibrosis, run by Grifols Therapeutics LLC at 6 sites in United States.
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 18 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 8 Feb 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States. 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?
Yes. Results are posted in the registry (first posted 8 Feb 2016). 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?
Adverse Events (time frame: 3 weeks). 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 8 Feb 2016.