CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 2 enrolled
Drug / intervention
Leukinedrug
Likely dose
Leukine 250 mcgfrom record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT01511068
NCT01511068Phase 2Completed
CompareCT.gov

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

Children's Hospital Medical Center, Cincinnati·interventional·Posted Jan 18, 2012·Updated Aug 30, 2023

In Brief

A Phase 2 clinical trial evaluating Leukine for Hereditary Pulmonary Alveolar Proteinosis. Completed, enrolled 2 participants across 2 sites.

Detailed 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.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsHereditary Pulmonary Alveolar Proteinosis
CountriesUnited States
CollaboratorsVirginia Commonwealth University, Genzyme, a Sanofi Company

Timeline

Phase 2CompletedFinished
201220132014201520162017201820192020202120222023202420252026
First PostedJan 18, 2012
Enrollment StartAug 1, 2012
Primary CompletionJul 1, 2013
Study CompletionJul 1, 2014
TodayJul 2, 2026
Enrollment to primary: 11 monthsPosted 14.5 years ago

Interventions

Leukinedrug

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.