Trial · NCT00901511
Inhaled GM-CSF Therapy of Autoimmune PAP
NCT00901511, Phase 2, Completed, Fondazione IRCCS Policlinico San Matteo di Pavia; results first posted 2023-06-26
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Inhaled GM-CSF Therapy of Autoimmune PAP. Trials, record NCT00901511. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00901511
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Inhaled GM-CSF Therapy of Autoimmune PAP
- NCT number
- NCT00901511
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 18 participants, actual
- Lead sponsor
- Fondazione IRCCS Policlinico San Matteo di Pavia
- Conditions
- Autoimmune Pulmonary Alveolar Proteinosis
- Interventions
- Baseline WLL, Sargramostim, Rescue WLL
- First posted
- 2009-05-13
- Last updated
- 2024-07-12
- Other registries' IDs
- eudract:2008-007086-23
- Results first posted
- 2023-06-26
Source ClinicalTrials.gov
Registered outcomes14
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Time to Rescue WLL | 30 months | Time (in months) between the scheduled baseline WLL (at Study Month 0) and the first administration of an unscheduled rescue WLL (during the 30-months after Study Month 0) |
| Secondary | Number of Patients Requiring a Rescue WLL | 30 months | Number of patients requiring an unscheduled rescue WLL during the 30-months immediately following administration of the scheduled baseline WLL therapy at Study Month 0. |
| Secondary | Between-Group Difference in Mean Partial Pressure of Oxygen (PaO2) | 30 months | Primary Analysis: Between-group difference in mean PaO2 at each study visit after the scheduled baseline WLL was evaluated using repeated measures analysis of variance (ANOVA) after adjustment for baseline values, gender, age, and the number of patients at risk at each time point. Secondary analysis: Between-group difference in mean PaO2 at each visit after imputation of missing data by the last observation carried forward method. For both of the primary and secondary analyses, PaO2 is determined from lab values measured for an arterial blood gas procedure. |
| Secondary | Between-Group Difference in Mean Alveolar-arterial Difference in Oxygen Concentration (A-aDO2) | 30 months | Primary Analysis: Between-group difference in mean A-aDO2 at each study visit after the scheduled baseline WLL was evaluated using repeated measures analysis of variance (ANOVA) after adjustment for baseline values, gender, age, and the number of patients at risk at each time point. Secondary analysis: Between-group difference in mean A-aDO2 at each visit after imputation of missing data by the last-value carried forward method. For both of the primary and secondary analyses, A-aDO2 is calculated from lab values measured for an arterial blood gas, the ambient atmosphere pressure, and fraction of inspired oxygen at the time of the arterial blood gas procedure. |
| Secondary | Between-Group Difference in Mean Diffusion Capacity of the Lungs for Carbon Monoxide (DLCO) | 30 months | Primary Analysis: Between-group difference in mean DLCO at each study visit after the scheduled baseline WLL was evaluated using repeated measures analysis of variance (ANOVA) after adjustment for baseline values, gender, age, and the number of patients at risk at each time point. Secondary analysis: Between-group difference in mean DLCO at each visit after imputation of missing data by the last observation carried forward method. For both of the primary and secondary analyses, DLCO is determined from pulmonary function tests. |
| Secondary | Between-Group Difference in Mean Vital Capacity (VC) | 30 months | Primary Analysis: Between-group difference in mean VC at each study visit after the scheduled baseline WLL was evaluated using repeated measures analysis of variance (ANOVA) after adjustment for baseline values, gender, age, and the number of patients at risk at each time point. Secondary analysis: Between-group difference in mean VC at each visit after imputation of missing data by the last observation carried forward method. For both of the primary and secondary analyses, VC is determined from pulmonary function tests. |
| Secondary | Between-Group Difference in Mean Chest Computed Tomography Ground Glass Opacification (GGO) Score | 30 months | Primary Analysis: Between-group difference in median GGO Score at each study visit after the scheduled baseline WLL was evaluated using repeated measures analysis of variance (ANOVA) after adjustment for baseline values, gender, age, and the number of patients at risk at each time point. Secondary analysis: Between-group difference in median GGO Score at each visit after imputation of missing data by the last observation carried forward method. For both of the primary and secondary analyses, the degree of severity of lung disease was calculated by determining the number of segments affected. The following scale was used: Grade 1 = 1 segment affected Grade 2 = 2 - 5 segments affected Grade 3 = 6 - 9 segments affected Grade 4 = 10 - 14 segments affected Grade 5 = \> 14 affected segments |
| Secondary | Between-Group Difference in Median Serum Carcinoembryonic Antigen (CEA) Levels | 30 months | Primary Analysis: Between-group difference in median serum CEA levels at each study visit after the scheduled baseline WLL was evaluated using repeated measures analysis of variance (ANOVA) after adjustment for baseline values, gender, age, and the number of patients at risk at each time point. Secondary analysis: Between-group difference in serum CEA levels at each visit after imputation of missing data by the last observation carried forward method. For both of the primary and secondary analyses, the serum CEA levels were determined via enzyme linked immunosorbent assay (ELISA). |
| Secondary | Between-Group Difference in Mean Serum Krebs Von-Lungren 6 Antigen (KL-6) Levels | 30 months | Primary Analysis: Between-group difference in median serum KL-6 levels at each study visit after the scheduled baseline WLL was evaluated using repeated measures analysis of variance (ANOVA) after adjustment for baseline values, gender, age, and the number of patients at risk at each time point. Secondary analysis: Between-group difference in serum KL-6 levels at each visit after imputation of missing data by the last observation carried forward method. For both of the primary and secondary analyses, the serum KL-6 levels were determined via enzyme linked immunosorbent assay (ELISA). |
| Secondary | Between-Group Difference in Mean Serum Cytokeratin-19 Fragment (Cyfra21.1) Levels | 30 months | Primary Analysis: Between-group difference in mean serum Cyfra21.1 levels at each study visit after the scheduled baseline WLL was evaluated using repeated measures analysis of variance (ANOVA) after adjustment for baseline values, gender, age, and the number of patients at risk at each time point. Secondary analysis: Between-group difference in serum Cyfra21.1 levels at each visit after imputation of missing data by the last observation carried forward method. For both of the primary and secondary analyses, the serum Cyfra21.1 levels were determined via enzyme linked immunosorbent assay (ELISA). |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 registered outcomesOutcomes14
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Time to Rescue WLL | 30 months | Months | MEDIAN | |
| SECONDARY | Number of Patients Requiring a Rescue WLL | 30 months | participants | NUMBER | |
| SECONDARY | Between-Group Difference in Mean Partial Pressure of Oxygen (PaO2) | 30 months | mmHg | MEAN | |
| SECONDARY | Between-Group Difference in Mean Alveolar-arterial Difference in Oxygen Concentration (A-aDO2) | 30 months | mmHg | MEAN | |
| SECONDARY | Between-Group Difference in Mean Diffusion Capacity of the Lungs for Carbon Monoxide (DLCO) | 30 months | % predicted | MEAN | |
| SECONDARY | Between-Group Difference in Mean Vital Capacity (VC) | 30 months | % predicted | MEAN | |
| SECONDARY | Between-Group Difference in Mean Chest Computed Tomography Ground Glass Opacification (GGO) Score | 30 months | score on a scale | MEDIAN | |
| SECONDARY | Between-Group Difference in Median Serum Carcinoembryonic Antigen (CEA) Levels | 30 months | ng/ml | MEDIAN | |
| SECONDARY | Between-Group Difference in Mean Serum Krebs Von-Lungren 6 Antigen (KL-6) Levels | 30 months | U/ml | MEDIAN | |
| SECONDARY | Between-Group Difference in Mean Serum Cytokeratin-19 Fragment (Cyfra21.1) Levels | 30 months | ng/ml | MEAN |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 outcomesResults by group188
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Time to Rescue WLL | Control Group | 18 | Inter-Quartile Range 6 to 27 | Months | |
| Time to Rescue WLL | GM-CSF Group | 30 | Inter-Quartile Range 30 to 30 | Months | |
| Number of Patients Requiring a Rescue WLL | Control Group | 7 | participants | ||
| Number of Patients Requiring a Rescue WLL | GM-CSF Group | 1 | participants | ||
| Between-Group Difference in Mean Partial Pressure of Oxygen (PaO2) | Control Group | Pre-WLL ( Month -1) | 51 | Standard Deviation 12 | mmHg |
| Between-Group Difference in Mean Partial Pressure of Oxygen (PaO2) | GM-CSF Group | Pre-WLL ( Month -1) | 66 | Standard Deviation 14 | mmHg |
| Between-Group Difference in Mean Partial Pressure of Oxygen (PaO2) | Control Group | Baseline (Month 0) | 72 | Standard Deviation 15 | mmHg |
| Between-Group Difference in Mean Partial Pressure of Oxygen (PaO2) | GM-CSF Group | Baseline (Month 0) | 82 | Standard Deviation 11 | mmHg |
| Between-Group Difference in Mean Partial Pressure of Oxygen (PaO2) | Control Group | 1 month | 70 | Standard Deviation 14 | mmHg |
| Between-Group Difference in Mean Partial Pressure of Oxygen (PaO2) | GM-CSF Group | 1 month | 88 | Standard Deviation 8.4 | mmHg |
1 to 10 of 188Source ClinicalTrials.gov
See all 188 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Control Group | 9 | |
| Overall Study | STARTED | GM-CSF Group | 9 | |
| Overall Study | COMPLETED | Control Group | 8 | |
| Overall Study | COMPLETED | GM-CSF Group | 8 | |
| Overall Study | NOT COMPLETED | Control Group | 1 | |
| Overall Study | NOT COMPLETED | GM-CSF Group | 1 |
Source ClinicalTrials.gov
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Control Group | 0 | 9 |
| Total, all-cause mortality | GM-CSF Group | 0 | 9 |
| Total, other adverse events | Control Group | 0 | 9 |
| Total, other adverse events | GM-CSF Group | 0 | 9 |
| Total, serious adverse events | Control Group | 0 | 9 |
| Total, serious adverse events | GM-CSF Group | 0 | 9 |
Source ClinicalTrials.gov
Papers5
The papers the trial lists on ClinicalTrials.gov.
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 comparisons3
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| GM-CSF Group vs Control Group: Number of Patients Requiring a Rescue WLL | Trial comparisons |
| GM-CSF Group vs Control Group: deaths from any cause | Trial comparisons |
| GM-CSF Group vs Control Group: serious adverse events | Trial comparisons |
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/NCT00901511
GET https://conductscience.org/api/trials/v1/NCT00901511/results?page=2