Rituximab in IgG4-related Disease: A Phase 1-2 Trial
This study has been completed.
Information provided by (Responsible Party):
John H. Stone, MD, Massachusetts General Hospital
First received: April 22, 2012
Last updated: January 7, 2015
Last verified: January 2015
The primary objective of this study is to evaluate the safety and effectiveness of rituximab in IgG4-RD.
||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
||Rituximab in IgG4-Related Disease: A Phase 1-2 Trial
Primary Outcome Measures:
- Primary (Disease Response) [ Time Frame: Six months ] [ Designated as safety issue: No ]
Disease Response at six months is the primary endpoint in this trial. The ability to maintain disease remission off glucocorticoids is an important clinical measure in this disease.
Disease Response - Disease Response is defined at 6 months as:
- Improvement of > 2 points in the IgG4-RD RI over baseline
- No glucocorticoid or other immunosuppressive drug use between months 4 and 6
- No disease flares, as assessed by the IgG4-RD Responder Index.
| Study Start Date:
| Study Completion Date:
| Primary Completion Date:
||January 2014 (Final data collection date for primary outcome measure)
Rituximab 1000 mg IV times two doses, separated by approximately 15 days.
Other Name: Rituxan
This two-center trial will enroll at total of 30 patients with IgG4-RD. The two participating sites are the Massachusetts General Hospital (Boston, MA) and the Mayo Clinic (Rochester, MN). All patients will receive rituximab 1 gram intravenously times two doses, separated by approximately 15 days. The primary efficacy outcome - disease remission and successful completion of the glucocorticoid taper - will be assessed at six months. Patients will be followed on the protocol for an additional six months after measurement of the primary outcome.
|Ages Eligible for Study:
||18 Years and older
|Genders Eligible for Study:
|Accepts Healthy Volunteers:
Patients will be included in the trial based on the following disease-specific criteria:
All patients with pathologic diagnoses will have their specimens reviewed by pathology investigators.
• Organ involvement in a pattern consistent with IgG4-RD. This must include dysfunction of one of the following organs: pancreas (autoimmune pancreatitis); salivary glands (chronic sclerosing sialadenitis); lacrimal glands; orbital pseudotumor; kidneys; lungs; lymph nodes; meninges; aorta (including aortitis/periaortitis and/or retroperitoneal fibrosis); thyroid gland (Riedel's thyroiditis). If a patient is enrolled with a clinical diagnosis alone, the diagnosis must be accompanied by both an imaging finding compatible with IgG4-RD and a 1.5-fold elevation in the serum IgG4 concentration.
Patients will be excluded from the study based on the following criteria:
Disease-Specific Concerns: Excessive fibrosis within organs, such that a disease response to rituximab would not be expected.
General Medical Concerns:
- Pregnancy (a negative serum pregnancy test should be performed for all women of childbearing potential within 7 days of treatment), or lactating.
- Inability to comply with study and/or follow-up procedures.
- History of HIV.
- Presence of active infection.
- New York Heart Association Classification III or IV heart disease (See Appendix D).
- Concomitant malignancies or previous malignancies within the last five years, with the exception of adequately treated basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix.
- At the Investigator's discretion, receipt of a live vaccine within 4 weeks prior to randomization.
- Positive hepatitis B or C serology is considered a potential exclusion criterion. Hepatitis B screening should include hepatitis B antibody and surface antigen for a patient with no risk factors. For patients with risk factors or previous history of hepatitis B, add core antibodies and e-antigen.
- Allergies: History of severe allergic reactions to human or chimeric monoclonal antibodies or murine protein.
- Uncontrolled disease: They show evidence of other uncontrolled disease, including drug and alcohol abuse, which that could interfere with participation in the trial according to the protocol.
- History of anti-human anti-chimeric antibody formation.
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: NCT01584388
Massachusetts General Hospital
||John H Stone, MD, MPH
||Massachusetts General Hospital (Rheumatology Unit)
||Arezou Khosroshahi, MD
||Massachusetts General Hospital (Rheumatology Unit)
No publications provided
||John H. Stone, MD, Director, Clinical Rheumatology, Massachusetts General Hospital
History of Changes
|Other Study ID Numbers:
|Study First Received:
||April 22, 2012
||January 7, 2015
||United States: Food and Drug Administration
Keywords provided by Massachusetts General Hospital:
Type 1 autoimmune pancreatitis
IgG4-related sclerosing cholangitis
Chronic sclerosing sialadenitis
IgG4-related tubulointerstitial nephritis
Additional relevant MeSH terms:
ClinicalTrials.gov processed this record on March 01, 2015
Digestive System Diseases
Salivary Gland Diseases
Physiological Effects of Drugs