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

CompletedPhase 2

Sponsor: Children's Hospital Medical Center, Cincinnati

Conditions: Hereditary Pulmonary Alveolar Proteinosis

Interventions: Leukine

Countries: United States

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 overview

Sex: ALL

Age: 8 Years to —

Healthy volunteers: No

Study type: INTERVENTIONAL

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

Locations (2)

Cincinnati, Ohio, United States
Richmond, Virginia, United States