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

NCT01511068 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 2

Last updated 2023-08-30

Study results available
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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

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.

Sponsors & Collaborators

  • Virginia Commonwealth University

    collaborator OTHER

  • Genzyme, a Sanofi Company

    collaborator INDUSTRY

  • Children's Hospital Medical Center, Cincinnati

    lead OTHER

Principal Investigators

  • Bruce Trapnell, MD · Children's Hospital Medical Center, Cincinnati

  • Bruce Rubin, MD, FRCPC · Virginia Commonwealth University

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
8 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-08-31
Primary Completion
2013-07-31
Completion
2014-07-31

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01511068 on ClinicalTrials.gov