GM-CSF in Patients With Pulmonary Alveolar Proteinosis

NCT ID: NCT00030056

Last Updated: 2015-03-25

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 48 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2001-09-30
• Study Completion Date: 2005-12-31

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).

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

Conditions

Pulmonary Alveolar Proteinosis

Study Design

• Allocation Method: RANDOMIZED
• Primary Study Purpose: TREATMENT

Interventions

GM-CSF (granulocyte-macrophage colony-stimulating factor, sargramostim)

Intervention Type: DRUG

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

The Cleveland Clinic

OTHER

Sponsor Role: lead

Locations

National Jewish Medical Center

Denver, Colorado, United States

Cleveland Clinic Foundation

Cleveland, Ohio, United States

The University of Pennsylvania Medical Center

Philadelphia, Pennsylvania, United States

Countries

United States

Other Identifiers

FD-R-002016-01

Identifier Type: -

Identifier Source: secondary_id

FD-R-002016

Identifier Type: -

Identifier Source: org_study_id