GM-CSF in Patients With Pulmonary Alveolar Proteinosis
Recruitment status was Recruiting
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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 |
| Estimated Enrollment: | 48 |
| Study Start Date: | September 2001 |
| Estimated Study Completion Date: | September 2004 |
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 |
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| Contact: Mani S. Kavuru, M.D. | 216-445-6972 | kavurum@ccf.org |
| 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 | |
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 May 16, 2013