Pulmonary Alveolar ProteinosisPhase 2NCT00030056
GM-CSF in Patients With Pulmonary Alveolar Proteinosis
A Phase 2 study testing GM-CSF (granulocyte-macrophage colony-stimulating factor, sargramostim) in people with Pulmonary Alveolar Proteinosis, run by The Cleveland Clinic at 3 sites in United States.
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). Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Pulmonary Alveolar Proteinosis
- What is being tested?
- GM-CSF (granulocyte-macrophage colony-stimulating factor, sargramostim)
- How many people
- 48 participants
- Where
- 3 sitesUnited States
- Study dates
- Sep 2001to Dec 2005
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Cleveland Clinic Foundation
Cleveland, Ohio, 44195
National Jewish Medical Center
Denver, Colorado
The University of Pennsylvania Medical Center
Philadelphia, Pennsylvania
About this study
Official title: Trial of GM-CSF for Alveolar Proteinosis
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).
Read the 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
- Conditions
- Pulmonary Alveolar Proteinosis
- Being tested
- GM-CSF (granulocyte-macrophage colony-stimulating factor, sargramostim)
- Keywords
- Sargramostim, Injections, Subcutaneous
- Medical subject headings
- Pulmonary Alveolar Proteinosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GM-CSF (granulocyte-macrophage colony-stimulating factor, sargramostim)Drug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jan 2002 (estimated)
- Study startSep 2001
- Study completionDec 2005
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pulmonary Alveolar ProteinosisStudies of the same condition, open and under way.
- More trials run by The Cleveland ClinicThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Enrolment
- 48 participants (estimated)
Sponsor and investigators
- Lead sponsor
- The Cleveland Clinic
- Sponsor type
- University, hospital or other
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00030056
- Sponsor's protocol ID
- FD-R-002016
- Other IDs
- FD-R-002016-01
- Status last verified
- Jan 2002
- First submitted
- 30 Jan 2002
- Last updated
- 25 Mar 2015
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Mar 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Trial of GM-CSF for Alveolar Proteinosis
- ClinicalTrials.gov ID
- NCT00030056
- Acronym / sponsor ID
- FD-R-002016
- Conditions
- Pulmonary Alveolar Proteinosis
- Keywords
- Sargramostim, Injections, Subcutaneous
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 48 participants (estimated)
- Lead sponsor
- The Cleveland ClinicUniversity, hospital or other
- Study start
- Sep 2001
- Study completion
- Dec 2005
- First posted
- 31 Jan 2002 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (8 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GM-CSF (granulocyte-macrophage colony-stimulating factor, sargramostim) | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Mar 2015.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing GM-CSF (granulocyte-macrophage colony-stimulating factor, sargramostim) in people with Pulmonary Alveolar Proteinosis, run by The Cleveland Clinic at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 25 Mar 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early. Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Mar 2015.