Trial · NCT02001688
Phase II, Safety and Efficacy Study of Kamada-alpha-1-antitrypsin (AAT) for Inhalation"
NCT02001688, Phase 2, Completed, Kamada, Ltd.; results first posted 2020-01-18
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Phase II, Safety and Efficacy Study of Kamada-alpha-1-antitrypsin (AAT) for Inhalation". Trials, record NCT02001688. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02001688
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Phase II, Safety and Efficacy Study of Kamada-alpha-1-antitrypsin (AAT) for Inhalation"
- NCT number
- NCT02001688
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 36 participants, actual
- Lead sponsor
- Kamada, Ltd.
- Conditions
- Alpha-1 Antitrypsin Deficiency
- Interventions
- Kamada-AAT for Inhalation, 80mg, Placebo, Kamada-AAT for Inhalation, 160mg
- First posted
- 2013-12-05
- Last updated
- 2020-01-18
- Results first posted
- 2020-01-18
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in the Concentration of Antigenic Alpha-1 Antitrypsin (AAT) in the Lung Epithelial Lining Fluid (ELF) | 12 weeks from initiation of study drug | Patients underwent bronchoalveolar lavage (BAL) prior to initiation of drug and then again after 3 months. Concentration of AAT in the BAL was measured by an enzyme linked immunosorbent assay (ELISA) specific for the normal form of AAT (piM). Urea was also measured in order to determine the dilution factor of the BAL fluid and calculate the concentration of AAT in the ELF. |
| Primary | Change From Baseline to 12 Weeks in the Concentration of Functional AAT (Alpha-1 Antitrypsin) in ELF | 12 weeks from initiation of study drug | ITT population with baseline and 12 week values. Patients underwent bronchoalveolar lavage (BAL) prior to initiation of drug and then again after 3 months. Concentration of AAT in the BAL was measured by an antineutrophil elastase capacity (ANEC) assay. Urea was also measured in order to determine the dilution factor of the BAL fluid and calculate the concentration of AAT in the ELF. |
| Secondary | Change From Baseline in Levels of M Specific AAT in Plasma (PiM) | 12 weeks from initiation of study drug | Intention to treat (ITT) population with baseline and 12 week values.The median change from baseline to Week 12 in the levels of M-specific AAT (piM) in plasma was measured using a specific ELISA assay for M-specific AAT (piM) |
| Secondary | Change From Baseline in AAT-neutrophil Elastase (NE) Complexes in ELF | 12 weeks from initiation of study drug | Patients underwent a BAL procedure at baseline and 12 weeks and the fluid was analyzed to calculate the change from baseline in the concentration of complexes between AAT and neutrophil elastase in the ELF |
Source ClinicalTrials.gov
Outcomes4
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in the Concentration of Antigenic Alpha-1 Antitrypsin (AAT) in the Lung Epithelial Lining Fluid (ELF) | 12 weeks from initiation of study drug | Intention to treat (ITT) population with values available in BAL at baseline and 12 weeks | nmol | MEDIAN |
| PRIMARY | Change From Baseline to 12 Weeks in the Concentration of Functional AAT (Alpha-1 Antitrypsin) in ELF | 12 weeks from initiation of study drug | nmol | MEDIAN | |
| SECONDARY | Change From Baseline in Levels of M Specific AAT in Plasma (PiM) | 12 weeks from initiation of study drug | nM | MEDIAN | |
| SECONDARY | Change From Baseline in AAT-neutrophil Elastase (NE) Complexes in ELF | 12 weeks from initiation of study drug | nM | MEDIAN |
Source ClinicalTrials.gov
Results by group12
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in the Concentration of Antigenic Alpha-1 Antitrypsin (AAT) in the Lung Epithelial Lining Fluid (ELF) | Kamada-AAT for Inhalation, 80mg | 4551.023 | 95% Confidence Interval 2587 to 6835.6 | nmol | |
| Change From Baseline in the Concentration of Antigenic Alpha-1 Antitrypsin (AAT) in the Lung Epithelial Lining Fluid (ELF) | Kamada-AAT for Inhalation, 160mg | 13454.772 | 95% Confidence Interval 1020.7 to 70907.95 | nmol | |
| Change From Baseline in the Concentration of Antigenic Alpha-1 Antitrypsin (AAT) in the Lung Epithelial Lining Fluid (ELF) | Placebo | -27.15 | 95% Confidence Interval -103.6 to 196.7 | nmol | |
| Change From Baseline to 12 Weeks in the Concentration of Functional AAT (Alpha-1 Antitrypsin) in ELF | Kamada-AAT for Inhalation, 80mg | 2766.343 | 95% Confidence Interval 840.9 to 3800.3 | nmol | |
| Change From Baseline to 12 Weeks in the Concentration of Functional AAT (Alpha-1 Antitrypsin) in ELF | Kamada-AAT for Inhalation, 160mg | 3557.563 | 95% Confidence Interval 1582.8 to 10673.2 | nmol | |
| Change From Baseline to 12 Weeks in the Concentration of Functional AAT (Alpha-1 Antitrypsin) in ELF | Placebo | 5.831 | 95% Confidence Interval -1032 to 884.3 | nmol | |
| Change From Baseline in Levels of M Specific AAT in Plasma (PiM) | Kamada-AAT for Inhalation, 80mg | 113.17 | 95% Confidence Interval 56.3 to 158.1 | nM | |
| Change From Baseline in Levels of M Specific AAT in Plasma (PiM) | Kamada-AAT for Inhalation, 160mg | 90.73 | 95% Confidence Interval 37.3 to 369.7 | nM | |
| Change From Baseline in Levels of M Specific AAT in Plasma (PiM) | Placebo | -4.02 | 95% Confidence Interval -9.8 to 4.9 | nM | |
| Change From Baseline in AAT-neutrophil Elastase (NE) Complexes in ELF | Kamada-AAT for Inhalation, 80mg | 38.71 | 95% Confidence Interval 8.4 to 66.8 | nM |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Kamada-AAT for Inhalation, 80mg | 12 | |
| Overall Study | STARTED | Kamada-AAT for Inhalation, 160mg | 12 | |
| Overall Study | STARTED | Placebo | 12 | |
| Overall Study | COMPLETED | Kamada-AAT for Inhalation, 80mg | 12 | |
| Overall Study | COMPLETED | Kamada-AAT for Inhalation, 160mg | 11 | |
| Overall Study | COMPLETED | Placebo | 11 | |
| Overall Study | NOT COMPLETED | Kamada-AAT for Inhalation, 80mg | 0 | |
| Overall Study | NOT COMPLETED | Kamada-AAT for Inhalation, 160mg | 1 | |
| Overall Study | NOT COMPLETED | Placebo | 1 |
Source ClinicalTrials.gov
Adverse events150
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Gastrooesophageal reflux disease | Gastrointestinal disorders | Kamada-AAT for Inhalation, 80mg | 0 | 12 | 0 |
| other | Gastrooesophageal reflux disease | Gastrointestinal disorders | Kamada-AAT for Inhalation, 160mg | 2 | 12 | 2 |
| other | Gastrooesophageal reflux disease | Gastrointestinal disorders | Placebo | 0 | 12 | 0 |
| other | Lip swelling | Gastrointestinal disorders | Kamada-AAT for Inhalation, 80mg | 0 | 12 | 0 |
| other | Lip swelling | Gastrointestinal disorders | Kamada-AAT for Inhalation, 160mg | 0 | 12 | 0 |
| other | Lip swelling | Gastrointestinal disorders | Placebo | 1 | 12 | 1 |
| other | Nausea | Gastrointestinal disorders | Kamada-AAT for Inhalation, 80mg | 0 | 12 | 0 |
| other | Nausea | Gastrointestinal disorders | Kamada-AAT for Inhalation, 160mg | 1 | 12 | 1 |
| other | Nausea | Gastrointestinal disorders | Placebo | 0 | 12 | 0 |
| other | Chest pain | General disorders | Kamada-AAT for Inhalation, 80mg | 0 | 12 | 0 |
1 to 10 of 150Source ClinicalTrials.gov
See all 150 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Kamada-AAT for Inhalation, 80mg | 0 | 12 |
| Total, all-cause mortality | Kamada-AAT for Inhalation, 160mg | 0 | 12 |
| Total, all-cause mortality | Placebo | 0 | 12 |
| Total, other adverse events | Kamada-AAT for Inhalation, 80mg | 10 | 12 |
| Total, other adverse events | Kamada-AAT for Inhalation, 160mg | 8 | 12 |
| Total, other adverse events | Placebo | 7 | 12 |
| Total, serious adverse events | Kamada-AAT for Inhalation, 80mg | 0 | 12 |
| Total, serious adverse events | Kamada-AAT for Inhalation, 160mg | 1 | 12 |
| Total, serious adverse events | Placebo | 0 | 12 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02001688
GET https://conductscience.org/api/trials/v1/NCT02001688/events?page=2