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GM-CSF in Patients With Pulmonary Alveolar Proteinosis

The recruitment status of this study is unknown because the information has not been verified recently.
Verified January 2002 by FDA Office of Orphan Products Development.
Recruitment status was  Recruiting
Sponsor:
Information provided by:
FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:
NCT00030056
First received: January 30, 2002
Last updated: June 23, 2005
Last verified: January 2002
  Purpose

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

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Primary Purpose: Treatment
Official Title: Trial of GM-CSF for Alveolar Proteinosis

Resource links provided by NLM:


Further study details as provided by FDA Office of Orphan Products Development:

Estimated Enrollment: 48
Study Start Date: September 2001
Estimated Study Completion Date: September 2004
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.

  Eligibility

Ages Eligible for Study:   18 Years to 70 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00030056

Contacts
Contact: Mani S. Kavuru, M.D. 216-445-6972 kavurum@ccf.org

Locations
United States, Colorado
National Jewish Medical Center Recruiting
Denver, Colorado, United States
United States, Ohio
Cleveland Clinic Foundation Recruiting
Cleveland, Ohio, United States, 44195
United States, Pennsylvania
The University of Pennsylvania Medical Center Recruiting
Philadelphia, Pennsylvania, United States
Sponsors and Collaborators
  More Information

No publications provided

ClinicalTrials.gov Identifier: NCT00030056     History of Changes
Other Study ID Numbers: FD-R-002016, FD-R-002016-01
Study First Received: January 30, 2002
Last Updated: June 23, 2005
Health Authority: United States: Food and Drug Administration

Keywords provided by FDA Office of Orphan Products Development:
Sargramostim
Injections, Subcutaneous

Additional relevant MeSH terms:
Pulmonary Alveolar Proteinosis
Lung Diseases
Respiratory Tract Diseases

ClinicalTrials.gov processed this record on November 25, 2014