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

PHASE2CompletedINTERVENTIONAL
Enrollment

2

Participants

Timeline

Start Date

August 31, 2012

Primary Completion Date

July 31, 2013

Study Completion Date

July 31, 2014

Conditions
Hereditary Pulmonary Alveolar Proteinosis
Interventions
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.

Trial Locations (2)

45229

Cincinnati Children's Hospital Medical Center, Cincinnati

23298-0646

Virginia Commonwealth University, Richmond

All Listed Sponsors
collaborator

Virginia Commonwealth University

OTHER

collaborator

Genzyme, a Sanofi Company

INDUSTRY

lead

Children's Hospital Medical Center, Cincinnati

OTHER

NCT01511068 - Inhaled Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in Hereditary Pulmonary Alveolar Proteinosis (PAP) | Biotech Hunter | Biotech Hunter