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

The recruitment status of this study is unknown because the information has not been verified recently.
Verified May 2008 by East Carolina University.
Recruitment status was  Recruiting
Sponsor:
Collaborator:
Genentech, Inc.
Information provided by:
East Carolina University
ClinicalTrials.gov Identifier:
NCT00552461
First received: October 31, 2007
Last updated: May 6, 2008
Last verified: May 2008
  Purpose

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.


Condition Intervention Phase
Pulmonary Alveolar Proteinosis
Primary Disease
Drug: rituximab
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Prospective Open-Label Trial of Rituximab for Primary Pulmonary Alveolar Proteinosis

Resource links provided by NLM:


Further study details as provided by East Carolina University:

Primary Outcome Measures:
  • Evaluate symptomatic, physiologic and radiographic effects of therapy [ Time Frame: 6-months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Evaluate the overall tolerability of therapy and the requirement for therapeutic whole-lung lavage [ Time Frame: 6-months ] [ Designated as safety issue: No ]

Estimated Enrollment: 10
Study Start Date: January 2007
Estimated Study Completion Date: December 2009
Estimated Primary Completion Date: July 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1 Drug: rituximab
IV, 1000 mg, two weeks, 2 times
Other Names:
  • Rituxan
  • MabThera

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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00552461

Contacts
Contact: Irene Marshall, PA-C 252-744-5888 marshalli@ecu.edu

Locations
United States, North Carolina
East Carolina University Recruiting
Greenville, North Carolina, United States, 27834
Contact: Mani S Kavuru, MD    252-744-4653    kavurum@ecu.edu   
Contact: Irene Marshall, PA-C    252-744-5888    marshalli@ecu.edu   
Principal Investigator: Mani S Kavuru, MD         
Sponsors and Collaborators
East Carolina University
Genentech, Inc.
Investigators
Principal Investigator: Mani S Kavuru, MD East Carolina University
  More Information

Additional Information:
No publications provided by East Carolina University

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Mani S. Kavuru, MD, Prinicipal Investigator, Professor and Division Chief Pulmonary & CCM, East Carolina University
ClinicalTrials.gov Identifier: NCT00552461     History of Changes
Other Study ID Numbers: U2990s
Study First Received: October 31, 2007
Last Updated: May 6, 2008
Health Authority: United States: Institutional Review Board

Keywords provided by East Carolina University:
Pulmonary alveolar proteinosis
Primary Pulmonary alveolar proteinosis
Rituximab
Idiopathic
Symptomatic
GM-CSF Antibody

Additional relevant MeSH terms:
Pulmonary Alveolar Proteinosis
Lung Diseases
Respiratory Tract Diseases
Rituximab
Antineoplastic Agents
Antirheumatic Agents
Immunologic Factors
Pharmacologic Actions
Physiological Effects of Drugs
Therapeutic Uses

ClinicalTrials.gov processed this record on October 30, 2014