GM-CSF in Patients With Pulmonary Alveolar Proteinosis

Pulmonary Alveolar Proteinosis Clinical Trial

Official title:

Trial of GM-CSF for Alveolar Proteinosis

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT00030056
• Other study ID #: FD-R-002016
• Secondary ID: FD-R-002016-01
• Status: Terminated
• Phase: Phase 2
• First received: January 30, 2002
• Last updated: March 24, 2015
• Start date: September 2001
• Est. completion date: December 2005

Study information

• Verified date: January 2002
• Source: FDA Office of Orphan Products Development
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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).

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

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 48
• Est. completion date: December 2005
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 70 Years

Eligibility

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

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Pulmonary Alveolar Proteinosis

Intervention

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

Locations

Country	Name	City	State
United States	Cleveland Clinic Foundation	Cleveland	Ohio
United States	National Jewish Medical Center	Denver	Colorado
United States	The University of Pennsylvania Medical Center	Philadelphia	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
The Cleveland Clinic

Country where clinical trial is conducted

United States,