drugset / Trial / NCT01511068
Inhaled Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in Hereditary Pulmonary Alveolar Proteinosis (PAP)
Phase 2
Completed
2 enrolled
Children's Hospital Medical Center, Cincinnati
Genzyme, a Sanofi Company · collabVirginia Commonwealth University · collab
NaSingle-groupOpen-labelTreatment
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.
Timeline
- Start
- 2012-08
- Primary completion
- 2013-07
- Completion
- 2014-07
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Leukine | Protein / enzyme biologic | 250 ug | Inhaled |
| Subject | Leukine | Protein / enzyme biologic | 500 ug | Inhaled |