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

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ClinicalTrials.gov Identifier: NCT01511068
Recruitment Status : Completed
First Posted : January 18, 2012
Last Update Posted : June 4, 2014
Sponsor:
Collaborators:
Virginia Commonwealth University
Genzyme, a Sanofi Company
Information provided by (Responsible Party):
Children's Hospital Medical Center, Cincinnati

December 12, 2011
January 18, 2012
June 4, 2014
August 2012
July 2013   (Final data collection date for primary outcome measure)
Time for the oxygen saturation to fall below 88% [ Time Frame: 6 months ]
Same as current
Complete list of historical versions of study NCT01511068 on ClinicalTrials.gov Archive Site
  • Improvement in pulmonary function [ Time Frame: 6 months ]
  • Improvement in exercise tolerance [ Time Frame: 6 months ]
  • Improvement in CT scan [ Time Frame: 6 months ]
  • Improvement in Quality of Life [ Time Frame: 6 months ]
  • Improvement in dyspnea and fatigue [ Time Frame: 6 months ]
  • Biomarkers will be assayed to detect changes from baseline values: surfactant protein-D (SP-D), KL-6, CEA), GM-CSF, GM-CSF autoantibodies, macrophage colony stimulating factor (M-CSF), and macrophage inflammatory protein -1 alpha MIP-1alpha. [ Time Frame: 6 months ]
Same as current
Not Provided
Not Provided
 
Inhaled Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in Hereditary Pulmonary Alveolar Proteinosis (PAP)
Inhaled Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in Hereditary Pulmonary Alveolar Proteinosis (PAP)
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.
Not Provided
Interventional
Phase 2
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Hereditary Pulmonary Alveolar Proteinosis
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.
Other Name: GM-CSF [Leukine (Sargramostim)]
Experimental: inhaled recombinant
inhaled recombinant human GM-CSF in individuals with hereditary Pulmonary Alveolar Proteinosis (PAP) due to partial dysfunction of the GM-CSF receptor
Intervention: Drug: Leukine
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
2
Same as current
February 2014
July 2013   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • 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 Criteria:

  • 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
Sexes Eligible for Study: All
8 Years and older   (Child, Adult, Older Adult)
No
Contact information is only displayed when the study is recruiting subjects
United States
 
 
NCT01511068
2011-0959_CCHMC_IRB
Yes
Not Provided
Not Provided
Children's Hospital Medical Center, Cincinnati
Children's Hospital Medical Center, Cincinnati
  • Virginia Commonwealth University
  • Genzyme, a Sanofi Company
Principal Investigator: Bruce Trapnell, MD Children's Hospital Medical Center, Cincinnati
Principal Investigator: Bruce Rubin, MD, FRCPC Virginia Commonwealth University
Children's Hospital Medical Center, Cincinnati
January 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP