Alpha1-antitrypsin DeficiencyPhase 1/2NCT00157092
Study of the Effect of Aerosolized, Recombinant Alpha 1-Antitrypsin on Epithelial Lining Fluid Analytes in Subjects With Alpha 1-Antitrypsin Deficiency
A Phase 1/2 study testing Aerosolized, Recombinant Alpha 1-Antitrypsin in people with Alpha1-antitrypsin Deficiency, run by Baxalta now part of Shire at 1 site in United States.
Trial summary
The study was a Phase 1B/2A, uncontrolled, open-label, single-center study in individuals with congenital AAT (alpha 1-antitrypsin) deficiency. A baseline bronchoscopy with bronchoalveolar lavage (BAL) was performed 3 to a maximum of 4 weeks prior to the first administration of study drug. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Alpha1-antitrypsin Deficiency
- What is being tested?
- Aerosolized, Recombinant Alpha 1-Antitrypsin
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Mar 2004to 14 Oct 2004
- Phase
- Phase 1/2
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Signed Informed Consent
- Male or female 18 years of age or older
- Documented, endogenous plasma AAT levels < 11 µM (< 80 mg/dL), either obtained from the medical history or, if not documented, plasma AAT measured after 28 day washout of any prior replacement therapy
- Forced expiratory volume at 1 second (FEV1) that is >= 50% of predicted, measured 30 minutes after a short-acting inhaled bronchodilator
- Arterial oxygen percent saturation (SaO2, measured using room air) within the normal limits for the individual study site
- For subjects receiving an inhaled corticosteroid, inhaled or oral β-2 agonist (e.g., albuterol via metered dose inhaler [MDI]) or inhaled anticholinergic bronchodilator (e.g., ipratropium bromide), or oral PDE (phosphodiesterase) inhibitor, treatment on a stable dose for at least 14 days prior to enrollment
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- For any female of childbearing potential, a negative urine test for pregnancy within 3 days prior to enrollment and agreement to employ adequate birth control measures for the duration of the study
- No clinically significant abnormalities detected on a 12-lead electrocardiogram (ECG) performed at the screening visit
- Laboratory results obtained at the screening visit, meeting the following criteria:
- Serum aspartate transaminase (AST) and alanine transaminase (ALT) <= 2 times upper limit of normal range (ULN)
- Serum total bilirubin <= 2 times ULN
- < 2+ proteinuria on urine dipstick
- Serum creatinine <= 1.5 times ULN
- Absolute neutrophil count >= 1500 cells/mm3
- Hemoglobin >= 10.0 g/dL
- Platelet count >= 100,000/mm3
Who cannot join
- Clinically significant pulmonary impairment, other than emphysema and/or chronic bronchitis
- Moderate to severe bronchiectasis
- Clinically significant cardiac, hemostatic, or neurologic impairment, or other significant medical condition that, in the opinion of the investigator, would affect subject safety or compliance
- Psychiatric or cognitive disturbance or illness, or recreational drug/alcohol use that, in the opinion of the investigator, would affect subject safety or compliance
- Acute exacerbation of emphysema within 28 days prior to the screening visit
- Pregnancy or lactation
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- Known history of allergy to yeast products
- Medical history precluding the use of epinephrine or other rescue medication for treatment of anaphylaxis
- Prior history of adverse reactions to the local anesthetic, sedative, BAL procedure, or pre-medication employed at the study center
- Use of oral or parenteral glucocorticosteroids, or alpha 1-antitrypsin replacement therapy within 28 days prior to baseline BAL, or any use planned during the study. However, the subject may enroll provided that a) consent is given to undergo a 28-day washout of the replacement or steroid therapy, and b) no study procedures are done until the washout is completed.
- Use of another investigational drug or investigational device within 28 days prior to baseline BAL
- Any upper or lower respiratory infection within 28 days prior to baseline BAL
- Having received a lung or liver transplant
Trial locations and contacts
1 site in 1 country.
United States1 site
Shands Hospital at the University of Florida
Gainesville, Florida, 32610
About this study
Official title: A Phase 1B/2A Study to Evaluate the Effect of Aerosolized, Recombinant Alpha 1-Antitrypsin on Epithelial Lining Fluid Analytes in Subjects With Alpha 1-Antitrypsin Deficiency
The study was a Phase 1B/2A, uncontrolled, open-label, single-center study in individuals with congenital AAT (alpha 1-antitrypsin) deficiency. A baseline bronchoscopy with bronchoalveolar lavage (BAL) was performed 3 to a maximum of 4 weeks prior to the first administration of study drug. Fifteen eligible subjects were randomized to receive 1 of 3 dosing regimens of rAAT (100 mg daily, 100 mg twice daily, or 200 mg daily) administered via nebulization for 7 consecutive days. A post-treatment nadir BAL was obtained on study Day 8 (12 hours after last dose for subjects who receive drug therapy twice daily and 24 hours after the last dose for subjects who receive study product daily). BALs were conducted in the same lung lobe/segment. Follow-up visits took place on Day 15 and Day 36.
- Conditions
- Alpha1-antitrypsin Deficiency
- Being tested
- Aerosolized, Recombinant Alpha 1-Antitrypsin
- Medical subject headings
- alpha 1-Antitrypsin Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Aerosolized, Recombinant Alpha 1-AntitrypsinDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
Alpha 1-antitrypsin levels assessed from epithelial lining fluid (ELF) samples
Measured: Baseline (minimum of 3 to a maximum of 4 weeks prior to the first administration of study product).
Alpha 1-antitrypsin levels assessed from epithelial lining fluid (ELF) samples
Measured: Day 8 after the first administration of study product
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Sep 2005 (estimated)
- Study start1 Mar 2004
- Primary completion14 Oct 2004
- Study completion14 Oct 2004
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alpha1-antitrypsin DeficiencyStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baxalta now part of Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Arriva Pharmaceuticals, Inc.
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00157092
- Sponsor's protocol ID
- 410302
- Status last verified
- Apr 2021
- First submitted
- 8 Sep 2005
- Last updated
- 5 May 2021
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1B/2A Study to Evaluate the Effect of Aerosolized, Recombinant Alpha 1-Antitrypsin on Epithelial Lining Fluid Analytes in Subjects With Alpha 1-Antitrypsin Deficiency
- ClinicalTrials.gov ID
- NCT00157092
- Acronym / sponsor ID
- 410302
- Conditions
- Alpha1-antitrypsin Deficiency
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Baxalta now part of ShireIndustry (pharma, biotech, devices)
- Collaborators
- Arriva Pharmaceuticals, Inc.
- Study start
- 1 Mar 2004 (actual)
- Primary completion
- 14 Oct 2004 (actual)
- Study completion
- 14 Oct 2004 (actual)
- First posted
- 12 Sep 2005 (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 study was a Phase 1B/2A, uncontrolled, open-label, single-center study in individuals with congenital AAT (alpha 1-antitrypsin) deficiency. A baseline bronchoscopy with bronchoalveolar lavage (BAL) was performed 3 to a maximum of 4 weeks prior to the first administration of study drug. Fifteen eligible subjects were randomized to receive 1 of 3 dosing regimens of rAAT (100 mg daily, 100 mg twice daily, or 200 mg daily) administered via nebulization for 7 consecutive days. A post-treatment nadir BAL was obtained on study Day 8 (12 hours after last dose for subjects who receive drug therapy twice daily and 24 hours after the last dose for subjects who receive study product daily). BALs were conducted in the same lung lobe/segment. Follow-up visits took place on Day 15 and Day 36.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- Signed Informed Consent
- Male or female 18 years of age or older
- Documented, endogenous plasma AAT levels < 11 µM (< 80 mg/dL), either obtained from the medical history or, if not documented, plasma AAT measured after 28 day washout of any prior replacement therapy
- Forced expiratory volume at 1 second (FEV1) that is >= 50% of predicted, measured 30 minutes after a short-acting inhaled bronchodilator
- Arterial oxygen percent saturation (SaO2, measured using room air) within the normal limits for the individual study site
Key exclusion criteria (13 listed)
- Clinically significant pulmonary impairment, other than emphysema and/or chronic bronchitis
- Moderate to severe bronchiectasis
- Clinically significant cardiac, hemostatic, or neurologic impairment, or other significant medical condition that, in the opinion of the investigator, would affect subject safety or compliance
- Psychiatric or cognitive disturbance or illness, or recreational drug/alcohol use that, in the opinion of the investigator, would affect subject safety or compliance
- Acute exacerbation of emphysema within 28 days prior to the screening visit
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, Recombinant Alpha 1-Antitrypsin | Drug | – |
Primary outcome
Alpha 1-antitrypsin levels assessed from epithelial lining fluid (ELF) samples
Time frame: Baseline (minimum of 3 to a maximum of 4 weeks prior to the first administration of study product).
Alpha 1-antitrypsin levels assessed from epithelial lining fluid (ELF) samples
Time frame: Day 8 after the first administration of study product
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 May 2021.
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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.
Study Director study director
Takeda
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Aerosolized, Recombinant Alpha 1-Antitrypsin in people with Alpha1-antitrypsin Deficiency, run by Baxalta now part of Shire at 1 site 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 16 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 5 May 2021. 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 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined 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.
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?
Alpha 1-antitrypsin levels assessed from epithelial lining fluid (ELF) samples (time frame: Baseline (minimum of 3 to a maximum of 4 weeks prior to the first administration of study product).). The study lists 2 primary outcomes in all. 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 May 2021.