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GM-CSF in Patients With Pulmonary Alveolar Proteinosis

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ClinicalTrials.gov Identifier: NCT00030056
Recruitment Status : Terminated
First Posted : January 31, 2002
Last Update Posted : March 25, 2015
Information provided by:
FDA Office of Orphan Products Development

Brief Summary:
This is a study to determine the efficacy and safety of granulocyte-macrophage colony-stimulating factor (GM-CSF, sargramostim) administered subcutaneously to patients with pulmonary alveolar proteinosis (PAP).

Condition or disease Intervention/treatment Phase
Pulmonary Alveolar Proteinosis Drug: GM-CSF (granulocyte-macrophage colony-stimulating factor, sargramostim) Phase 2

Detailed Description:

PAP is a rare lung disease characterized by accumulation of surfactant phospholipids and proteins within the lungs. There is no specific pharmacologic therapy for PAP and the current practice of lung lavage under general anesthesia is invasive and has limitations. Although it is unknown if the anti GM-CSF antibody is related to the disease pathogenesis, observations suggest a role for GM-CSF in lung homeostasis as well as in the pathogenesis of PAP.

Patients will receive subcutaneous GM-CSF or placebo once a day and will be followed on an outpatient basis at 2 weeks, and 1, 2, 3, 4, 5 and 6 months after initiation of therapy. Clinical response will determine dosing schedule and will be evaluated by symptom scores, gas exchange data, and chest radiographs.

Completion date provided represents the completion date of the grant per OOPD records

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 48 participants
Allocation: Randomized
Primary Purpose: Treatment
Official Title: Trial of GM-CSF for Alveolar Proteinosis
Study Start Date : September 2001
Study Completion Date : December 2005

Resource links provided by the National Library of Medicine

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion criteria:

  • Diagnosis of primary or idiopathic PAP
  • Creatinine no greater than 2 mg/dL
  • Bilirubin no greater than 2 mg/dL
  • Liver enzymes no greater than 3 times normal
  • Women must use an effective method of contraception, be post menopausal, or be surgically sterilized

Exclusion criteria:

  • Active respiratory infection
  • Active cardiovascular disease (e.g., cardiogenic pulmonary edema)
  • Underlying myeloproliferative disorder or leukemia
  • Other secondary cause of PAP (e.g., occupational exposure to silica or HIV with PCP)
  • At increased risk of side effects from GM-CSF therapy (i.e., rheumatoid arthritis, immune thrombocytopenia, or autoimmune thyroiditis)
  • Previous therapy with GM-CSF
  • Pregnant or nursing
  • Significant renal or liver disease

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00030056

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United States, Colorado
National Jewish Medical Center
Denver, Colorado, United States
United States, Ohio
Cleveland Clinic Foundation
Cleveland, Ohio, United States, 44195
United States, Pennsylvania
The University of Pennsylvania Medical Center
Philadelphia, Pennsylvania, United States
Sponsors and Collaborators
The Cleveland Clinic
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ClinicalTrials.gov Identifier: NCT00030056    
Other Study ID Numbers: FD-R-002016
First Posted: January 31, 2002    Key Record Dates
Last Update Posted: March 25, 2015
Last Verified: January 2002
Keywords provided by FDA Office of Orphan Products Development:
Injections, Subcutaneous
Additional relevant MeSH terms:
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Pulmonary Alveolar Proteinosis
Lung Diseases
Respiratory Tract Diseases
Immunologic Factors
Physiological Effects of Drugs
Antineoplastic Agents