Phase 2
N=2
Inhaled Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in Hereditary Pulmonary Alveolar Proteinosis (PAP)
Hereditary Pulmonary Alveolar Proteinosis
Bottom Line
View on ClinicalTrials.gov: NCT01511068 ↗Enrolled (actual)
2
Serious AEs
0.0%
Results posted
Sep 2020
Primary outcome: Primary: Change in Time (Minutes) to Discontinuation of Exercise During a Standardized Treadmill Exercise Test — 17; 33 Percent Change
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Leukine (Drug)
- Age
- Pediatric, Adult, Older Adult · 8+ yrs
- Sex
- All
- Sponsor
- Children's Hospital Medical Center, Cincinnati
- Primary completion
- Jul 2013
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Time (Minutes) to Discontinuation of Exercise During a Standardized Treadmill Exercise Test |
17; 33 | — |
| PRIMARY Change in Minimum Pulse Oximetry During a Standardized Treadmill Exercise Test |
0; -2.1 | — |
| SECONDARY Change in Diffusion Capacity for Carbon Monoxide |
11; 4 | — |
| SECONDARY Change in Minimum Pulse Oximetry During a Standardized Exercise Protocol Oximetry |
0; 50 | — |
| SECONDARY Change in Radiographic Evidence of PAP Lung Disease |
-510; -579; -366; -441; -593; -675 | — |
| SECONDARY Change in Quality of Life |
10.7; 75; -5.2; 19.6; 3.1; 11.7 | — |
| SECONDARY Change in Dyspnea Symptom Score |
71; 11 | — |
| SECONDARY Change in Serum Anti-GM-CSF Antibodies Levels |
0.67; 0.36; 0.32; 1.85; 19.7; 71.8 | — |
| SECONDARY Change in Serum Biomarkers - GM-CSF |
43.75; 39.4; 43.2; 88.5; 11.5; 7.8 | — |
| SECONDARY Change in Serum Biomarkers - Surfactant Protein D |
796.2; 619; 696.4; 589; 697.2; 536.9 | — |
Summary
The purpose of this study is to evaluate the therapeutic efficacy of inhaled recombinant human GM-CSF in individuals with hereditary Pulmonary Alveolar Proteinosis (PAP) due to partial dysfunction of the GM-CSF receptor.
Eligibility Criteria
Inclusion Criteria
- A diagnosis of PAP caused by bi-allelic mutations in CSF2RA or CSF2RB associated with impaired GM-CSF-R-alpha or GM-CSF-R-beta function, respectively, resulting in reduced but non-zero GM-CSF signaling
- Able and willing to give written informed consent / assent as necessary
- Clinically stable
Exclusion Criteria
- Confirmed diagnosis of a disorder of surfactant production caused by bi-allelic mutations in ABCA3, SFTPB, or SFTPC
- Confirmed diagnosis of autoimmune PAP caused by a high level of GM-CSF autoantibody
- Confirmed diagnosis of secondary PAP caused by an underlying clinical disorder known to be associated with the development of PAP, e.g., inhalation of silica or titanium; myelodysplasia and others
- Treatment with any investigational agent in the 3 months prior to enrollment
- History of severe allergic or anaphylactic reactions to GM-CSF or other yeast-derived products
- History of asthma or other reactive airways disease
- Known active, viral, fungal, mycobacterial, or other infection
- A serious medical condition which, in the opinion of the investigator or data and safety monitoring committee, would make the patient unsuitable for the study
Data sourced from ClinicalTrials.gov (NCT01511068). 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.