Trial · NCT01511068
Inhaled Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in Hereditary Pulmonary Alveolar Proteinosis (PAP)
NCT01511068, Phase 2, Completed, Children's Hospital Medical Center, Cincinnati; results first posted 2020-09-01
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Inhaled Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in Hereditary Pulmonary Alveolar Proteinosis (PAP). Trials, record NCT01511068. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01511068
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Inhaled Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in Hereditary Pulmonary Alveolar Proteinosis (PAP)
- NCT number
- NCT01511068
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 2 participants, actual
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Conditions
- Hereditary Pulmonary Alveolar Proteinosis
- Interventions
- Leukine
- First posted
- 2012-01-18
- Last updated
- 2023-08-30
- Results first posted
- 2020-09-01
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change in Time (Minutes) to Discontinuation of Exercise During a Standardized Treadmill Exercise Test | Baseline, 7 months | A modified Bruce protocol stress test was used to evaluate improvement in blood oxygen saturation (SpO2). A pulse-oximeter was placed on the participant's finger with the participant at rest while sitting in a chair. Leads for the electrocardiograph were placed on the chest wall. The treadmill was started at 1.7 miles per hour (mph) and a grade of 0%. At three minute intervals, the speed increased as follows: 1.7 mph, 1.7 mph, 1.7 mph, 2.5 mph, 3.4 mph, 4.2 mph, 5.0 mph, 5.5 mph, 6.0 mph, 6.5 mph, 7.0 mph, and 7.5 mph. The participant stopped the test due to intolerable dyspnea or if the SpO2 fell below 88%. |
| Primary | Change in Minimum Pulse Oximetry During a Standardized Treadmill Exercise Test | Baseline, 7 months | A modified Bruce protocol stress test was used to evaluate improvement in blood oxygen saturation (SpO2). A pulse-oximeter was placed on the participant's finger with the participant at rest while sitting in a chair. Leads for the electrocardiograph were placed on the chest wall. The treadmill was started at 1.7 miles per hour (mph) and a grade of 0%. At three minute intervals, the speed increased as follows: 1.7 mph, 1.7 mph, 1.7 mph, 2.5 mph, 3.4 mph, 4.2 mph, 5.0 mph, 5.5 mph, 6.0 mph, 6.5 mph, 7.0 mph, and 7.5 mph. The participant stopped the test due to intolerable dyspnea or if the SpO2 fell below 88%. |
| Secondary | Change in Diffusion Capacity for Carbon Monoxide | Baseline, 7 months | Routine full pulmonary function testing, including spirometry, lung volumes, and DLCO, were performed according to American Thoracic Society guidelines. |
| Secondary | Change in Minimum Pulse Oximetry During a Standardized Exercise Protocol Oximetry | Baseline, 7 months | Standardized exercise pulse oximetry (SEPO) was used to measure SpO2 at the participant's home on a weekly basis between clinic visits. Briefly, a pulse-oximeter was placed on the finger with the participant at rest sitting in a chair. Three baseline (resting) readings were taken over a period of 1 minute to measure the SpO2 at rest. The participant then began stepping onto and off of the first step of a staircase in the home while holding onto the handrail for safety. Stepping was started by placing the bottom of one foot onto the stair followed by the other foot and then removal of one foot from the stair to the floor followed by the other foot. This procedure was repeated at a frequency of 1 cycle per second for a total of 5 minutes. The participant's parent assisted by noting the saturation data at 1-minute intervals during the test onto the weekly exercise form in the participant's Diary. The participant's saturation data continued to be recorded for 3 minutes after the test. |
| Secondary | Change in Radiographic Evidence of PAP Lung Disease | Baseline, 7 months | High resolution computed tomography (HRCT) scans were performed using an interval technique, a 1 mm slice was obtained every 20 mm. The slice series were placed so that images were obtained from the pulmonary apices to the lung bases with one of the images located at the level of the carina. The CT parameters were performed at full inspiration and required a lower dose than usual clinical CT scans; 1 mm slices at 20 mm intervals,120 kVp, 60 mAs, rotation time 0.5 second. Images were reconstructed with lung and soft tissue reconstruction kernels (B35F and B60F). The primary analysis was performed using the B60F kernel. Images were read and reported according to Radiology Department protocol. The raw data was recorded on a DVD and sent to CCHMC for centralized reading and lung attenuation analysis. |
| Secondary | Change in Quality of Life | Baseline, 7 months | The PedsQL quality of life questionnaire is a modular approach to measure health-related quality of life in healthy children and those with acute and chronic health conditions. It is self-administered and completed in less than 5 minutes. It contains 23 items divided into 4 domains: physical functioning, emotional functioning, social functioning, and school functioning. To reverse score, transform the 0-4 scale items to 0-100 as follows: 0=100, 1=75, 2=50, 3=25, 4=0. Higher scores indicate a better Health-Related Quality of Life.To create the Psychosocial Health Summary Score, the mean is computed as the sum of the items over the number of items answered in the Emotional, Social, and School Functioning Scales. The Physical Health Summary Score is the same as the Physical Functioning Scale Score. To create the Total Scale Score, the mean is computed as the sum of all the items over the number of items answered on all the Scales. |
| Secondary | Change in Dyspnea Symptom Score | Baseline, 7 months | The dyspnea visual analogue scales were used by the patient to record the level of dyspnea by a single mark on a linear scale. The dyspnea scale ranged from 0 to 10, with short of breath all the time equal to 0 and never short of breath equal to 10. A higher score indicated a better dyspnea score. |
| Secondary | Change in Serum Anti-GM-CSF Antibodies Levels | Baseline and monthly up to 7 months | Serum GM-CSF autoantibody was measured as follows: microtiter plates were incubated (4°C, overnight) with rhGM-CSF, washed in PBS and Tween-20, and incubated (room temperature (RT), 1 hour) with blocking solution. Serum samples were diluted with dilution buffer and aliquots of diluted serum or standard were pipetted into adjacent microtiter wells, incubated at RT for 40 minutes, and then washed with wash buffer. Horseradish peroxidase-conjugated secondary antibody was diluted with dilution buffer and pipetted into each well. Plates were incubated (RT, 0.5 hour) and then washed with wash buffer. Substrate solution was added to each well, plates were incubated (RT, 15 min), and color development was stopped with sulfuric acid. Absorbance at 450 nm was measured using a Benchmark® ELISA plate reader. |
| Secondary | Change in Serum Biomarkers - GM-CSF | Baseline and monthly up to 7 months | Serum GM-CSF was measured via a commercial ELISA kit from R \& D Systems. |
| Secondary | Change in Serum Biomarkers - Surfactant Protein D | Baseline and monthly up to 7 months | Surfactant protein D (SP-D) was measured via a commercial ELISA kit from Biovender. |
Source ClinicalTrials.gov
Outcomes10
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change in Time (Minutes) to Discontinuation of Exercise During a Standardized Treadmill Exercise Test | Baseline, 7 months | Percent Change | NUMBER | |
| SECONDARY | Change in Diffusion Capacity for Carbon Monoxide | Baseline, 7 months | Percent Change | NUMBER | |
| SECONDARY | Change in Minimum Pulse Oximetry During a Standardized Exercise Protocol Oximetry | Baseline, 7 months | Percent Change | NUMBER | |
| SECONDARY | Change in Radiographic Evidence of PAP Lung Disease | Baseline, 7 months | Hounsfield Units | NUMBER | |
| SECONDARY | Change in Quality of Life | Baseline, 7 months | Percent Change | NUMBER | |
| SECONDARY | Change in Dyspnea Symptom Score | Baseline, 7 months | Percent Change | NUMBER | |
| SECONDARY | Change in Serum Anti-GM-CSF Antibodies Levels | Baseline and monthly up to 7 months | micrograms/ml | NUMBER | |
| PRIMARY | Change in Minimum Pulse Oximetry During a Standardized Treadmill Exercise Test | Baseline, 7 months | Percent Change | NUMBER | |
| SECONDARY | Change in Serum Biomarkers - GM-CSF | Baseline and monthly up to 7 months | picograms/ml | NUMBER | |
| SECONDARY | Change in Serum Biomarkers - Surfactant Protein D | Baseline and monthly up to 7 months | nanograms/ml | NUMBER |
Source ClinicalTrials.gov
Results by group82
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change in Time (Minutes) to Discontinuation of Exercise During a Standardized Treadmill Exercise Test | Inhaled Leukine (rhGM-CSF) | Participant 1 | 17 | Percent Change | |
| Change in Time (Minutes) to Discontinuation of Exercise During a Standardized Treadmill Exercise Test | Inhaled Leukine (rhGM-CSF) | Participant 2 | 33 | Percent Change | |
| Change in Diffusion Capacity for Carbon Monoxide | Inhaled Leukine (rhGM-CSF) | Participant 1 | 11 | Percent Change | |
| Change in Diffusion Capacity for Carbon Monoxide | Inhaled Leukine (rhGM-CSF) | Participant 2 | 4 | Percent Change | |
| Change in Minimum Pulse Oximetry During a Standardized Exercise Protocol Oximetry | Inhaled Leukine (rhGM-CSF) | Participant 1 | 0 | Percent Change | |
| Change in Minimum Pulse Oximetry During a Standardized Exercise Protocol Oximetry | Inhaled Leukine (rhGM-CSF) | Participant 2 | 50 | Percent Change | |
| Change in Radiographic Evidence of PAP Lung Disease | Inhaled Leukine (rhGM-CSF) | Participant 1-Right Lung Baseline | -510 | Hounsfield Units | |
| Change in Radiographic Evidence of PAP Lung Disease | Inhaled Leukine (rhGM-CSF) | Participant 1- Right Lung End of Treatment | -579 | Hounsfield Units | |
| Change in Radiographic Evidence of PAP Lung Disease | Inhaled Leukine (rhGM-CSF) | Participant 1-Left Lung Baseline | -366 | Hounsfield Units | |
| Change in Radiographic Evidence of PAP Lung Disease | Inhaled Leukine (rhGM-CSF) | Participant 1-Left Lung End of Treatment | -441 | Hounsfield Units |
1 to 10 of 82Source ClinicalTrials.gov
See all 82 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Inhaled Leukine (rhGM-CSF) | 2 | |
| Overall Study | COMPLETED | Inhaled Leukine (rhGM-CSF) | 2 | |
| Overall Study | NOT COMPLETED | Inhaled Leukine (rhGM-CSF) | 0 |
Source ClinicalTrials.gov
Adverse events2
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 | Upper Respiratory Tract Infection | Respiratory, thoracic and mediastinal disorders | Inhaled Leukine (rhGM-CSF) | 2 | 2 | 2 |
| other | Noseblood | General disorders | Inhaled Leukine (rhGM-CSF) | 1 | 2 | 1 |
Source ClinicalTrials.gov
Adverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Inhaled Leukine (rhGM-CSF) | ||
| Total, other adverse events | Inhaled Leukine (rhGM-CSF) | 2 | 2 |
| Total, serious adverse events | Inhaled Leukine (rhGM-CSF) | 0 | 2 |
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.
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