GM-CSF in Patients With Pulmonary Alveolar Proteinosis

This study is currently recruiting participants.

Verified by FDA Office of Orphan Products Development, January 2002

First Received: January 30, 2002 Last Updated: June 23, 2005 History of Changes

Sponsored by:	FDA Office of Orphan Products Development
Information provided by:	FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:	NCT00030056

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 II

Study Type:	Interventional
Study Design:	Treatment, Randomized, Placebo Control, Safety/Efficacy Study
Official Title:	Trial of GM-CSF for Alveolar Proteinosis

Resource links provided by NLM:

Drug Information available for: Granulocyte-macrophage colony-stimulating factor Sargramostim

U.S. FDA Resources

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

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

FDA Office of Orphan Products Development

More Information

No publications provided

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

Keywords provided by FDA Office of Orphan Products Development:

Sargramostim
Injections, Subcutaneous

Study placed in the following topic categories:

Respiratory Tract Diseases
Lung Diseases
Pulmonary Alveolar Proteinosis

Additional relevant MeSH terms:

Respiratory Tract Diseases
Lung Diseases
Pulmonary Alveolar Proteinosis

ClinicalTrials.gov processed this record on July 02, 2009