Status:
COMPLETED
Inhaled Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in Hereditary Pulmonary Alveolar Proteinosis (PAP)
Lead Sponsor:
Children's Hospital Medical Center, Cincinnati
Collaborating Sponsors:
Virginia Commonwealth University
Genzyme, a Sanofi Company
Conditions:
Hereditary Pulmonary Alveolar Proteinosis
Eligibility:
All Genders
8+ years
Phase:
PHASE2
Brief 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 ...
Eligibility Criteria
Inclusion
- A diagnosis of PAP caused by bi-allelic mutations in CSF2RA or CSF2RB associated with impaired GM-CSF-R-alpha or GM-CSF-R-beta function, respectively, resulting in reduced but non-zero GM-CSF signaling
- Able and willing to give written informed consent / assent as necessary
- Clinically stable
Exclusion
- Confirmed diagnosis of a disorder of surfactant production caused by bi-allelic mutations in ABCA3, SFTPB, or SFTPC
- Confirmed diagnosis of autoimmune PAP caused by a high level of GM-CSF autoantibody
- Confirmed diagnosis of secondary PAP caused by an underlying clinical disorder known to be associated with the development of PAP, e.g., inhalation of silica or titanium; myelodysplasia and others
- Treatment with any investigational agent in the 3 months prior to enrollment
- History of severe allergic or anaphylactic reactions to GM-CSF or other yeast-derived products
- History of asthma or other reactive airways disease
- Known active, viral, fungal, mycobacterial, or other infection
- A serious medical condition which, in the opinion of the investigator or data and safety monitoring committee, would make the patient unsuitable for the study
Key Trial Info
Start Date :
August 1 2012
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 1 2014
Estimated Enrollment :
2 Patients enrolled
Trial Details
Trial ID
NCT01511068
Start Date
August 1 2012
End Date
July 1 2014
Last Update
August 30 2023
Active Locations (2)
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1
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
2
Virginia Commonwealth University
Richmond, Virginia, United States, 23298-0646