GM-CSF in Patients With Pulmonary Alveolar Proteinosis

Trial of GM-CSF for Alveolar Proteinosis

Status

Terminated

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00030056

Enrollment

Registered

2002-01-31

Start date

2001-09-30

Completion date

2005-12-31

Last updated

2015-03-25

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Pulmonary Alveolar Proteinosis

Keywords

Sargramostim, Injections, Subcutaneous

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

Interventions

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

Study design

Allocation

RANDOMIZED

Primary purpose

TREATMENT

Eligibility

Sex/Gender

ALL

Age

18 Years to 70 Years

Healthy volunteers

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

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026