Evaluation of a Single Dose of Inhaled Sargramostim in Patients With Autoimmune Pulmonary Alveolar Proteinosis
Autoimmune Pulmonary Alveolar Proteinosis
About this trial
This is an interventional basic science trial for Autoimmune Pulmonary Alveolar Proteinosis
Eligibility Criteria
Inclusion Criteria:
Male or female
Age ≥ 18 years and ≤ 80 years
Able to understand and willing to sign a written informed consent document
Able and willing to use hand held nebulizer
Able and willing to adhere to study visit schedule and study procedures
Diagnosis of autoimmune PAP determined by:
- History of a diagnosis of PAP with or without supporting lung histology or BAL/cytology
and
- Abnormal serum GM-CSF autoantibody test (GMAb ELISA Test)
and
- Chest CT findings compatible with a diagnosis of autoimmune PAP
Evidence of impaired GM-CSF signaling demonstrated by an abnormal STAT5 phosphorylation index (STAT5-PI) test measured in heparinized whole blood at the time screening
A-aDO2 ≥ 15 mm Hg
Exclusion Criteria:
Diagnosis of any other PAP-causing disease
Autoimmune PAP complicated by:
- Severe disease at screening/enrollment (A-aD02<50)
- Clinically significant pulmonary fibrosis
History of any clinically significant:
- Other lung disease
- Cardiovascular disease
- Disease requiring use of systemic steroids in past year
- Coagulopathy or other hematologic disease
- Active / serious lung or systemic infection
- Persistent or unexplained fever >101oF within 2 months of study
- Use of any immunosuppressive medication within 3-6 months of screening
- Women who are pregnant or plan to become pregnant
- History of active tobacco/e-cig/marijuana use
- Concomitant or recent use of specific medicines
Sites / Locations
- University of California, Los Angeles
- Cincinnati Children's Hospital Medical Center
Arms of the Study
Arm 1
Experimental
Sargramostim
Participants will receive a single administration of inhaled sargramostim (either 125 mcg or 250 mcg dose)