Pulmonary Alveolar ProteinosisPhase 2PAPNCT00552461
Prospective Trial of Rituximab for Primary Pulmonary Alveolar Proteinosis
A Phase 2 study testing rituximab in people with Pulmonary Alveolar Proteinosis and Primary Disease, run by East Carolina University at 1 site in United States.
Trial summary
The purpose of this study is to determine whether the use of rituximab is effective in treating pulmonary alveolar proteinosis by leading to an improvement in lung function and disease status. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Pulmonary Alveolar Proteinosis, Primary Disease
- What is being tested?
- rituximab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2007to Dec 2011
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of primary PAP (new or chronic) with presence of anti-GM-CSF antibody
- Moderately symptomatic disease with PaO2 <70 on room air and on less than 6 L/min oxygen
- Able to give written informed consent and comply with the requirements of the study
- Adequate renal and liver function
- Negative serum pregnancy test (for women of child bearing age) and on acceptable birth control during and after study completion
Who cannot join
- Severe PAP and requires in-patient care and more urgent therapy with bilateral whole lung lavage
- Treatment with any investigational agent within 4 weeks of screening
- History of severe allergic or anaphylactic reactions to humanized or murine monoclonal antibodies
- History of HIV, recurrent significant infection or recurrent bacterial infections
- Known active bacterial, viral, fungal, mycobacterial, or other infection
- Ongoing use of high dose steroids (>10mg/day) or unstable steroid dose
- Significant cardiac or pulmonary disease or blood disorder
Trial locations and contacts
1 site in 1 country.
United States1 site
East Carolina University
Greenville, North Carolina, 27834
About this study
Official title: Prospective Open-Label Trial of Rituximab for Primary Pulmonary Alveolar Proteinosis
The purpose of this study is to determine whether the use of rituximab is effective in treating pulmonary alveolar proteinosis by leading to an improvement in lung function and disease status.
Read the detailed description
The goal of this study is to conduct a prospective, open-label 6-month trial of rituximab in patients who present with symptomatic primary or idiopathic PAP. A total of 10 subjects with primary PAP will be enrolled over 12 months at East Carolina University. Patients over age 18 with a clinical diagnosis of moderate symptomatic idiopathic PAP, established by appropriate clinical history, radiographic and physiologic findings, presence of circulating anti-GM-CSF antibody, and confirmatory findings on bronchoscopy with bronchoalveolar lavage and/or open-lung biopsy will be recruited. Patients with newly diagnosed PAP or established disease may be considered for this study.
- Conditions
- Pulmonary Alveolar Proteinosis, Primary Disease
- Being tested
- rituximab
- Keywords
- Pulmonary alveolar proteinosis, Primary Pulmonary alveolar proteinosis, Rituximab, Idiopathic, Symptomatic, GM-CSF Antibody
- 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.
rituximabDrug
Also called Rituxan, MabThera
IV, 1000 mg, two weeks, 2 times
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Not described Receives: Drug: rituximab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluate symptomatic, physiologic and radiographic effects of therapy
Measured: 6-months
Secondary outcomes (1)
Evaluate the overall tolerability of therapy and the requirement for therapeutic whole-lung lavage
Measured: 6-months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Nov 2007 (estimated)
- Study startJan 2007
- Primary completionJul 2009
- Study completionDec 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Pulmonary Alveolar ProteinosisStudies of the same condition, open and under way.
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- East Carolina University
- Sponsor type
- University, hospital or other
- Collaborators
- Genentech, Inc.
- Responsible party
- Principal investigator, Malur, Anagha, East Carolina University
- Investigators
- Mani S Kavuru, MD, Principal investigator, East Carolina University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00552461
- Sponsor's protocol ID
- U2990s
- Acronym
- PAP
- Status last verified
- Sep 2023
- First submitted
- 31 Oct 2007
- Last updated
- 28 Sep 2023
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Kavuru MS, Malur A, Marshall I, Barna BP, Meziane M, Huizar I, Dalrymple H, Karnekar R, Thomassen MJ. An open-label trial of rituximab therapy in pulmonary alveolar proteinosis. Eur Respir J. 2011 Dec;38(6):1361-7. doi: 10.1183/09031936.00197710. Epub 2011 Apr 8.
PubMed 21478218
Study at a glance
From the registry record, last updated by the sponsor on 28 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Open-Label Trial of Rituximab for Primary Pulmonary Alveolar Proteinosis
- ClinicalTrials.gov ID
- NCT00552461
- Acronym / sponsor ID
- PAP / U2990s
- Conditions
- Pulmonary Alveolar Proteinosis, Primary Disease
- Keywords
- Pulmonary alveolar proteinosis, Primary Pulmonary alveolar proteinosis, Rituximab, Idiopathic, Symptomatic, GM-CSF Antibody
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- East Carolina UniversityUniversity, hospital or other
- Collaborators
- Genentech, Inc.
- Study start
- Jan 2007
- Primary completion
- Jul 2009 (actual)
- Study completion
- Dec 2011 (actual)
- First posted
- 2 Nov 2007 (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.
The purpose of this study is to determine whether the use of rituximab is effective in treating pulmonary alveolar proteinosis by leading to an improvement in lung function and disease status.
Detailed description
The goal of this study is to conduct a prospective, open-label 6-month trial of rituximab in patients who present with symptomatic primary or idiopathic PAP. A total of 10 subjects with primary PAP will be enrolled over 12 months at East Carolina University. Patients over age 18 with a clinical diagnosis of moderate symptomatic idiopathic PAP, established by appropriate clinical history, radiographic and physiologic findings, presence of circulating anti-GM-CSF antibody, and confirmatory findings on bronchoscopy with bronchoalveolar lavage and/or open-lung biopsy will be recruited. Patients with newly diagnosed PAP or established disease may be considered for this study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Drug: rituximab |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Diagnosis of primary PAP (new or chronic) with presence of anti-GM-CSF antibody
- Moderately symptomatic disease with PaO2 <70 on room air and on less than 6 L/min oxygen
- Able to give written informed consent and comply with the requirements of the study
- Adequate renal and liver function
- Negative serum pregnancy test (for women of child bearing age) and on acceptable birth control during and after study completion
Key exclusion criteria (7 listed)
- Severe PAP and requires in-patient care and more urgent therapy with bilateral whole lung lavage
- Treatment with any investigational agent within 4 weeks of screening
- History of severe allergic or anaphylactic reactions to humanized or murine monoclonal antibodies
- History of HIV, recurrent significant infection or recurrent bacterial infections
- Known active bacterial, viral, fungal, mycobacterial, or other infection
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 |
|---|---|---|
| rituximabAlso: Rituxan, MabTheraIV, 1000 mg, two weeks, 2 times | Drug | 1 |
Primary outcome
Evaluate symptomatic, physiologic and radiographic effects of therapy
Time frame: 6-months
1 secondary outcome
Evaluate the overall tolerability of therapy and the requirement for therapeutic whole-lung lavage
Time frame: 6-months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Sep 2023.
- Derived from this study: PubMed 21478218Kavuru MS, Malur A, Marshall I, Barna BP, Meziane M, Huizar I, Dalrymple H, Karnekar R, Thomassen MJ. An open-label trial of rituximab therapy in pulmonary alveolar proteinosis. Eur Respir J. 2011 Dec;38(6):1361-7. doi: 10.1183/09031936.00197710. Epub 2011 Apr 8.
- East Carolina University Division of PulmonaryLink provided by the sponsor.
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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.
Mani S Kavuru, MD principal investigator
East Carolina University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing rituximab in people with Pulmonary Alveolar Proteinosis and Primary Disease, run by East Carolina University at 1 site 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 7 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: finished, as of the sponsor's last update on 28 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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.
What does the primary outcome measure?
Evaluate symptomatic, physiologic and radiographic effects of therapy (time frame: 6-months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00552461, which also lists 1 linked publication.
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 28 Sep 2023.