Prospective Trial of Rituximab for Primary Pulmonary Alveolar Proteinosis (PAP)

• ClinicalTrials.gov Identifier: NCT00552461
• Recruitment Status: Unknown
• First Posted: November 2, 2007
• Last Update Posted: May 9, 2008
• Sponsor: East Carolina University
• Collaborator: Genentech, Inc.
• Information provided by: East Carolina University

Tracking Information

• First Submitted Date: October 31, 2007
• First Posted Date: November 2, 2007
• Last Update Posted Date: May 9, 2008
• Study Start Date: January 2007
• Estimated Primary Completion Date: July 2009 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: October 31, 2007): Evaluate symptomatic, physiologic and radiographic effects of therapy [ Time Frame: 6-months ]
• Original Primary Outcome Measures: Same as current
• Current Secondary Outcome Measures (submitted: October 31, 2007): Evaluate the overall tolerability of therapy and the requirement for therapeutic whole-lung lavage [ Time Frame: 6-months ]
• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Prospective Trial of Rituximab for Primary Pulmonary Alveolar Proteinosis
• Official Title: Prospective Open-Label Trial of Rituximab for Primary Pulmonary Alveolar Proteinosis
• 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.
• Study Type: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: Non-Randomized; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment

Condition

• Pulmonary Alveolar Proteinosis
• Primary Disease

Intervention

Drug: rituximab

IV, 1000 mg, two weeks, 2 times

Other Names:

• Rituxan
• MabThera

Study Arms

Experimental: 1

Intervention: Drug: rituximab

• 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.

Recruitment Information

• Recruitment Status: Unknown status
• Estimated Enrollment (submitted: October 31, 2007): 10
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: December 2009
• Estimated Primary Completion Date: July 2009 (Final data collection date for primary outcome measure)

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT00552461
• Other Study ID Numbers: U2990s
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: Mani S. Kavuru, MD, Prinicipal Investigator, Professor and Division Chief Pulmonary & CCM, East Carolina University
• Original Responsible Party: Not Provided
• Current Study Sponsor: East Carolina University
• Original Study Sponsor: Same as current
• Collaborators: Genentech, Inc.

Investigators

• Principal Investigator: Mani S Kavuru, MD; East Carolina University

• PRS Account: East Carolina University
• Verification Date: May 2008