NCT01511068

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 2 (actual)
• Sponsor: Children's Hospital Medical Center, Cincinnati · Academic / Other
• Sex: All
• Age: 8 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Hereditary Pulmonary Alveolar Proteinosis

Interventions

Type	Name	Description
DRUG	Leukine	Participants will receive inhaled rhGM-CSF (Sargramostim, Leukine) at the dose of 250 mcg one time per week for 12 weeks. Following an interim safety evaluation, participants may be entered into a second 12 week treatment period where participants will receive either 250 mcg or 500 mcg once weekly. At the end of any treatment period, participants will be followed for 12 additional weeks in the absence of inhaled rhGM-CSF to evaluate safety and efficacy.

Timeline

• Start date: 2012-08-01
• Primary completion: 2013-07-01
• Completion: 2014-07-01
• First posted: 2012-01-18
• Last updated: 2023-08-30
• Results posted: 2020-09-01

Locations

2 sites across 1 country: United States