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GM-CSF in Patients With Pulmonary Alveolar Proteinosis

Primary Purpose

Pulmonary Alveolar Proteinosis

Status
Terminated
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
GM-CSF (granulocyte-macrophage colony-stimulating factor, sargramostim)
Sponsored by
The Cleveland Clinic
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Pulmonary Alveolar Proteinosis focused on measuring Sargramostim, Injections, Subcutaneous

Eligibility Criteria

18 Years - 70 Years (Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

  • National Jewish Medical Center
  • Cleveland Clinic Foundation
  • The University of Pennsylvania Medical Center

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

First Posted
January 30, 2002
Last Updated
March 24, 2015
Sponsor
The Cleveland Clinic
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1. Study Identification

Unique Protocol Identification Number
NCT00030056
Brief Title
GM-CSF in Patients With Pulmonary Alveolar Proteinosis
Official Title
Trial of GM-CSF for Alveolar Proteinosis
Study Type
Interventional

2. Study Status

Record Verification Date
January 2002
Overall Recruitment Status
Terminated
Study Start Date
September 2001 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
December 2005 (undefined)

3. Sponsor/Collaborators

Name of the Sponsor
The Cleveland Clinic

4. Oversight

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pulmonary Alveolar Proteinosis
Keywords
Sargramostim, Injections, Subcutaneous

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Allocation
Randomized
Enrollment
48 (Anticipated)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
GM-CSF (granulocyte-macrophage colony-stimulating factor, sargramostim)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
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
Facility Information:
Facility Name
National Jewish Medical Center
City
Denver
State/Province
Colorado
Country
United States
Facility Name
Cleveland Clinic Foundation
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Facility Name
The University of Pennsylvania Medical Center
City
Philadelphia
State/Province
Pennsylvania
Country
United States

12. IPD Sharing Statement

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GM-CSF in Patients With Pulmonary Alveolar Proteinosis

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