Prospective Trial of Rituximab for Primary Pulmonary Alveolar Proteinosis (PAP)
Primary Purpose
Pulmonary Alveolar Proteinosis, Primary Disease
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
rituximab
Sponsored by
About this trial
This is an interventional treatment trial for Pulmonary Alveolar Proteinosis focused on measuring Pulmonary alveolar proteinosis, Primary Pulmonary alveolar proteinosis, Rituximab, Idiopathic, Symptomatic, GM-CSF Antibody
Eligibility Criteria
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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
Sites / Locations
- East Carolina University
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
1
Arm Description
Outcomes
Primary Outcome Measures
Evaluate symptomatic, physiologic and radiographic effects of therapy
Secondary Outcome Measures
Evaluate the overall tolerability of therapy and the requirement for therapeutic whole-lung lavage
Full Information
NCT ID
NCT00552461
First Posted
October 31, 2007
Last Updated
September 26, 2023
Sponsor
East Carolina University
Collaborators
Genentech, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT00552461
Brief Title
Prospective Trial of Rituximab for Primary Pulmonary Alveolar Proteinosis
Acronym
PAP
Official Title
Prospective Open-Label Trial of Rituximab for Primary Pulmonary Alveolar Proteinosis
Study Type
Interventional
2. Study Status
Record Verification Date
September 2023
Overall Recruitment Status
Completed
Study Start Date
January 2007 (undefined)
Primary Completion Date
July 2009 (Actual)
Study Completion Date
December 2011 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
East Carolina University
Collaborators
Genentech, Inc.
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief 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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pulmonary Alveolar Proteinosis, Primary Disease
Keywords
Pulmonary alveolar proteinosis, Primary Pulmonary alveolar proteinosis, Rituximab, Idiopathic, Symptomatic, GM-CSF Antibody
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Actual)
8. Arms, Groups, and Interventions
Arm Title
1
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
rituximab
Other Intervention Name(s)
Rituxan, MabThera
Intervention Description
IV, 1000 mg, two weeks, 2 times
Primary Outcome Measure Information:
Title
Evaluate symptomatic, physiologic and radiographic effects of therapy
Time Frame
6-months
Secondary Outcome Measure Information:
Title
Evaluate the overall tolerability of therapy and the requirement for therapeutic whole-lung lavage
Time Frame
6-months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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
Exclusion Criteria:
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Mani S Kavuru, MD
Organizational Affiliation
East Carolina University
Official's Role
Principal Investigator
Facility Information:
Facility Name
East Carolina University
City
Greenville
State/Province
North Carolina
ZIP/Postal Code
27834
Country
United States
12. IPD Sharing Statement
Citations:
PubMed Identifier
21478218
Citation
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.
Results Reference
derived
Links:
URL
http://www.ecu.edu
Description
East Carolina University Division of Pulmonary
Learn more about this trial
Prospective Trial of Rituximab for Primary Pulmonary Alveolar Proteinosis
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