Inhaled Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in Hereditary Pulmonary Alveolar Proteinosis (PAP)

• Status: Completed
• Trial end date: 2014-07-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Children's Hospital Medical Center, Cincinnati
• Collaborators: Genzyme, a Sanofi Company; Virginia Commonwealth University
• Treatments: Sargramostim

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