Phase 2
N=18
Inhaled GM-CSF Therapy of Autoimmune PAP
Autoimmune Pulmonary Alveolar Proteinosis
Bottom Line
View on ClinicalTrials.gov: NCT00901511 ↗Enrolled (actual)
18
Serious AEs
0.0%
Results posted
Jun 2023
Primary outcome: Primary: Time to Rescue WLL — 18; 30 Months — p=0.0078
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Baseline WLL (Procedure); Sargramostim (Drug); Rescue WLL (Procedure)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Fondazione IRCCS Policlinico San Matteo di Pavia
- Primary completion
- Jun 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Time to Rescue WLL |
18; 30 | 0.0078 sig |
| SECONDARY Number of Patients Requiring a Rescue WLL |
7; 1 | 0.0152 sig |
| SECONDARY Between-Group Difference in Mean Partial Pressure of Oxygen (PaO2) |
51; 66; 72; 82; 70; 88 | <0.0001 sig |
| SECONDARY Between-Group Difference in Mean Alveolar-arterial Difference in Oxygen Concentration (A-aDO2) |
55; 43; 33; 26; 37; 20 | <0.0001 sig |
| SECONDARY Between-Group Difference in Mean Diffusion Capacity of the Lungs for Carbon Monoxide (DLCO) |
38; 46; 50; 55; 55; 68 | 0.0220 sig |
| SECONDARY Between-Group Difference in Mean Vital Capacity (VC) |
60; 64; 74; 76; 75; 76 | 0.5320 |
| SECONDARY Between-Group Difference in Mean Chest Computed Tomography Ground Glass Opacification (GGO) Score |
5; 5; 4; 3; 4; 3 | 0.0530 |
| SECONDARY Between-Group Difference in Median Serum Carcinoembryonic Antigen (CEA) Levels |
18; 6; 8; 5; 6; 3 | 0.0010 sig |
| SECONDARY Between-Group Difference in Mean Serum Krebs Von-Lungren 6 Antigen (KL-6) Levels |
9023; 5758; 10556; 5538; 8744; 2401 | 0.0300 sig |
| SECONDARY Between-Group Difference in Mean Serum Cytokeratin-19 Fragment (Cyfra21.1) Levels |
27; 13; 10; 6; 11; 5 | 0.220 |
| SECONDARY Between-Group Difference in Median Serum GM-CSF Autoantibody (GMAb) Levels |
23; 32; 20; 28; 25; 30 | 0.3865 |
| SECONDARY Between-Group Difference in Mean White Blood Cell (WBC) Counts |
7.7; 6.2; 7.5; 8.7; 6.8; 6.4 | 0.0582 |
| SECONDARY Between-Group Difference in Mean Platelet Counts |
289; 236; 290; 269; 266; 248 | 0.1864 |
| SECONDARY Between-Group Difference in Mean 36-Item Short Form Survey (SF-36) General Health Score |
44; 39; 77; 85; 79; 93 | 0.1490 |
Summary
This is a prospective, randomized, open-label, long-term, phase 2 study of inhaled granulocyte/macrophage-colony stimulating factor following whole lung lavage therapy in patients with autoimmune pulmonary alveolar proteinosis.
Eligibility Criteria
Inclusion Criteria
- Female of male 18 years of age or older
- Diagnosis of autoimmune PAP
- Able and willing to provide written informed consent
- Eligible for whole lung lavage determined as the presence of persistent or progressive respiratory failure (PaO2 at rest 5 percentage points during standard exercise
Exclusion Criteria
- Diagnosis with secondary PAP, congenital PAP, or hereditary PAP
- Contraindication to whole lung lavage
- Contraindication to administration of inhaled GM-CSF
- Chronic lung disease associated with already existing respiratory failure, such as emphysema or pulmonary fibrosis, chronic heart failure, ischemic heart disease, active pulmonary embolism, progressive cancer, and other severe metabolic conditions
Data sourced from ClinicalTrials.gov (NCT00901511). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.