Rituximab for the Treatment of Refractory Adult and Juvenile Dermatomyositis (DM) and Adult Polymyositis (PM)
NCT ID: NCT00106184
Last Updated: 2015-03-04
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
200 participants
INTERVENTIONAL
2006-03-31
2010-08-31
Brief Summary
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Study hypotheses: 1) The time to improvement in Group A patients (receiving rituximab first) will occur significantly earlier than in Group B patients (receiving rituximab later). 2) The proportion of patients improved at Week 8 of the treatment phase will be significantly greater in Group A than in Group B.
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Detailed Description
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A patient's participation in this study will last approximately 45 weeks. At screening, participants will have a physical exam, muscle strength assessment, an electrocardiogram, and blood and urine collection; they will also be asked to complete several questionnaires. All participants will receive 2 infusions of rituximab and 2 infusions of placebo. Participants will be randomly assigned to one of two groups. Group A will receive rituximab at Weeks 0 and 1 and placebo at Weeks 8 and 9. Group B will receive placebo at Weeks 0 and 1 and rituximab at Weeks 8 and 9. Each infusion will be given on an outpatient basis over a minimum of approximately 5 hours' time.
There will be a total of 14 study visits. All participants will visit the outpatient clinic at selected time points for muscle strength testing, a physical exam, disease activity measurements, and blood collection. During the study, participants will be monitored closely for improvement or worsening of their disease and for serious drug related side effects. Some participants will be asked if they are willing to undergo 2 muscle biopsy procedures, 1 prior to receiving study medication and 1 after receiving study medication, to determine the effects of rituximab on muscle tissue.
If a participant is unable to locate a near-by clinical center, the adult and pediatric centers at the National Institute of Health located in Bethesda, Maryland have funds available to assist with travel costs.
NIH SUB-STUDY: "Rituximab to Treat Dermatomyositis and Polymyositis"
* This study is currently recruiting patients.
* Sponsored by: National Institute of Environmental Health Sciences (NIEHS)
* Information provided by: National Institutes of Health Clinical Center (CC)
* Expected Total Enrollment: 30
* Study start: October 2006
* Location and Contact Information: Patient Recruitment and Public Liaison Office;(800) 411-1222; [email protected]; Phone: 1-866-411-1010
The NIH sub-study will take advantage of the multi-center core RIM trial to identify changes in gene expression patterns in muscle, skin, and peripheral blood and the imaging features and immunopathology of muscle, skin, and peripheral cells before (week 0) and after (week 16) therapy. These changes will also be correlated with the large number of clinical, laboratory, and research variables already planned to be collected in the core RIM Study. Furthermore, knowing specifically which gene expression patterns are altered in resistant patients before rituximab, and which are changed after rituximab therapy - in conjunction with flow cytometry of peripheral cells and immunopathology of the tissues - will help in understanding more about the pathogenesis of myositis and the possible contribution of B lymphocytes and their subsets.
Patients with dermatomyositis and polymyositis who meet the inclusion/exclusion criteria for the core RIM trial may be eligible for this sub-study. The following procedures will be conducted in addition to the core RIM trial procedures during the 13 clinic visits over a period of 44 weeks:
* Weeks 0, 16: Muscle and skin biopsy (adult only). Small samples of muscle and skin tissue will be surgically removed for examination under a microscope.
* Weeks 0, 8, 16, 44: Skin evaluation and photography. The effect of the disease on the skin will be thoroughly evaluated and photographs of any rashes and of the skin around the nails will be taken.
* Weeks 0, 8, 16, 44: Magnetic resonance imaging (MRI). All participants will have MRI scans of the skin and of the muscle in the legs. Adults will also have an MRI to examine blood flow in the muscle.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
TRIPLE
Study Groups
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Adult Study Group 1
Refractory adult polymyositis patients who will receive rituximab at Weeks 0 and 1 followed by placebo at Weeks 8 and 9 (Treatment Group A)
Rituximab
Treatment Group A - intravenous rituximab 750mg/m2 BSA (Body Surface Area) up to a maximum dose of 1 gram at Weeks 0 and 1 Group B - intravenous rituximab 750mg/m2 BSA (Body Surface Area) up to a maximum does of 1 gram at Weeks 8 and 9
Placebo
Treatment Group A: placebo infusion at Weeks 8 and 9
Treatment Group B: placebo infusion at Weeks 0 and 1
Adult Study Group 2
Refractory adult polymyositis patients who will receive placebo at Weeks 0 and 1 followed by rituximab at Weeks 8 and 9 (Treatment Group B)
Rituximab
Treatment Group A - intravenous rituximab 750mg/m2 BSA (Body Surface Area) up to a maximum dose of 1 gram at Weeks 0 and 1 Group B - intravenous rituximab 750mg/m2 BSA (Body Surface Area) up to a maximum does of 1 gram at Weeks 8 and 9
Placebo
Treatment Group A: placebo infusion at Weeks 8 and 9
Treatment Group B: placebo infusion at Weeks 0 and 1
Adult Study Group 3
Adult dermatomyositis patients who will receive rituximab at Weeks 0 and 1 followed by placebo at Weeks 8 and 9 (Treatment Group A)
Rituximab
Treatment Group A - intravenous rituximab 750mg/m2 BSA (Body Surface Area) up to a maximum dose of 1 gram at Weeks 0 and 1 Group B - intravenous rituximab 750mg/m2 BSA (Body Surface Area) up to a maximum does of 1 gram at Weeks 8 and 9
Placebo
Treatment Group A: placebo infusion at Weeks 8 and 9
Treatment Group B: placebo infusion at Weeks 0 and 1
Adult Study Group 4
Adult dermatomyositis patients who will receive placebo at Weeks 0 and 1 followed by rituximab at Weeks 8 and 9 (Treatment Group B)
Rituximab
Treatment Group A - intravenous rituximab 750mg/m2 BSA (Body Surface Area) up to a maximum dose of 1 gram at Weeks 0 and 1 Group B - intravenous rituximab 750mg/m2 BSA (Body Surface Area) up to a maximum does of 1 gram at Weeks 8 and 9
Placebo
Treatment Group A: placebo infusion at Weeks 8 and 9
Treatment Group B: placebo infusion at Weeks 0 and 1
JDM Study Group 1
Refractory juvenile dermatomyositis patients who will receive rituximab at Weeks 0 and 1 followed by placebo at Weeks 8 and 9 (Treatment Group A)
Rituximab
Treatment Group A - intravenous rituximab 750mg/m2 BSA (Body Surface Area) up to a maximum dose of 1 gram at Weeks 0 and 1 Group B - intravenous rituximab 750mg/m2 BSA (Body Surface Area) up to a maximum does of 1 gram at Weeks 8 and 9
Placebo
Treatment Group A: placebo infusion at Weeks 8 and 9
Treatment Group B: placebo infusion at Weeks 0 and 1
JDM Study Group 2
Refractory juvenile dermatomyositis patients who will receive placebo at Weeks 0 and 1 followed by rituximab at Weeks 8 and 9 (Treatment Group B)
Rituximab
Treatment Group A - intravenous rituximab 750mg/m2 BSA (Body Surface Area) up to a maximum dose of 1 gram at Weeks 0 and 1 Group B - intravenous rituximab 750mg/m2 BSA (Body Surface Area) up to a maximum does of 1 gram at Weeks 8 and 9
Placebo
Treatment Group A: placebo infusion at Weeks 8 and 9
Treatment Group B: placebo infusion at Weeks 0 and 1
Interventions
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Rituximab
Treatment Group A - intravenous rituximab 750mg/m2 BSA (Body Surface Area) up to a maximum dose of 1 gram at Weeks 0 and 1 Group B - intravenous rituximab 750mg/m2 BSA (Body Surface Area) up to a maximum does of 1 gram at Weeks 8 and 9
Placebo
Treatment Group A: placebo infusion at Weeks 8 and 9
Treatment Group B: placebo infusion at Weeks 0 and 1
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Refractory myositis, defined by intolerance to or inadequate response to corticosteroids plus an adequate regime of at least one other immunosuppressive agent. Intolerance is defined as side effects that require discontinuation of the medication or an underlying condition that precludes further use of the medication.
* Baseline manual muscle testing which is based on a maximum MMT-8 (Manual Muscle Test) score of 150:Adult subjects with dermatomyositis (DM) or polymyositis (PM) must have a score that is no greater than 125/150 in conjunction with 2 other abnormal core set measures.
Subjects with a diagnosis of Juvenile Dermatomyositis (JDM) must meet either of the following criteria:
1. An MMT-8 (Manual Muscle Test) score that is no greater than 125/150 in conjunction with 2 other abnormal core set measures.
OR
2. If MMT (Manual Muscle Test) score is greater than 125/150 the patient MUST meet at least 3 abnormal core set measures.
* Background therapy with at least 1 non-corticosteroid immunosuppressive agent at a stable dose for at least 6 weeks prior to screening
* Able and willing to complete self-report questionnaires. Parents of pediatric participants will be required to complete the questionnaires on behalf of their children.
* Willing to use acceptable forms of contraception for the duration of the study for patients of reproductive potential.
* Parent willing to provide informed consent, if applicable
* Willing to forgo immunization with a live vaccine for the duration of the study
Exclusion Criteria
* Juvenile polymyositis
* Inclusion body myositis
* Cancer-associated myositis, defined as the diagnosis of myositis within 2 years of the diagnosis of cancer. Patients with basal or squamous cell skin cancer or carcinoma in situ of the cervix are not excluded, if it has been at least 5 years since excision.
* Myositis in overlap with another connective tissue disease that may preclude the accurate assessment of a treatment response
* Live viral vaccine within 4 weeks prior to study entry
* Any joint disease or other musculoskeletal condition that may interfere with muscle strength testing
* Known hypersensitivity to mouse proteins
* Any concomitant or life-threatening non-myositis illness that, in the opinion of the investigator, may interfere with the study
* Known or suspected history of drug or alcohol abuse within the last 6 months prior to study entry, as determined by medical record or patient interview
* Anticipated poor compliance with study requirements
* Participation in another clinical trial within 30 days prior to screening
* Any history or evidence of any severe illness or other condition that, in the opinion of the investigator, may interfere with the study
* Previously received rituximab
* Evidence of prior infection with hepatitis B or hepatitis C virus
* Initiation of an exercise program within 4 weeks of screening OR initiation of an exercise program during the study
* Consumed any creatine-containing, over-the-counter products in the form of dietary supplements 30 days prior to screening visit and for the duration of the study
5 Years
ALL
No
Sponsors
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National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
NIH
Genentech, Inc.
INDUSTRY
Biogen
INDUSTRY
University of Pittsburgh
OTHER
Responsible Party
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Chester Oddis
MD, Professor of Medicine, University of Pittsburgh, Division of Rheumatology and Clinical Immunology
Principal Investigators
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Chester V. Oddis, MD
Role: PRINCIPAL_INVESTIGATOR
University of Pittsburgh
Ann M. Reed, MD
Role: PRINCIPAL_INVESTIGATOR
Mayo Clinic
Locations
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University of Alabama Arthritis Intervention Program (Adult Site)
Birmingham, Alabama, United States
Phoenix Neurological Associates, LTD (Adult Site)
Phoenix, Arizona, United States
Cedars-Sinai Medical Center (Adult Site)
Los Angeles, California, United States
Stanford University (Adult Site)
Stanford, California, United States
Stanford University (Pediatric Site)
Stanford, California, United States
University of Miami School of Medicine (Adult Site)
Miami, Florida, United States
Miami Children's Hospital (Pediatric Site)
Miami, Florida, United States
University of Kansas Medical Center (Adult Site)
Kansas City, Kansas, United States
Kentucky Clinic (Adult Site)
Lexington, Kentucky, United States
National Institute of Health (Adult Site)
Bethesda, Maryland, United States
National Institute of Health (Pediatric Site)
Bethesda, Maryland, United States
Children's Hospital of Boston (Pediatric Site)
Boston, Massachusetts, United States
Beth Israel Deaconess Medical Center (Adult Site)
Boston, Massachusetts, United States
University of Michigan Health System (Adult Site)
Ann Arbor, Michigan, United States
Michigan State University (Adult and Pediatric Site)
Grand Rapids, Michigan, United States
Mayo Clinic (Adult Site)
Rochester, Minnesota, United States
Mayo Clinic (Pediatric Site)
Rochester, Minnesota, United States
North Shore Long Island Jewish Health System (Adult Site)
Lake Success, New York, United States
Hospital for Special Surgery (Adult Site)
New York, New York, United States
Duke University Medical Center (Pediatric Site)
Durham, North Carolina, United States
Cincinnati's Children's Hospital (Pediatric Site)
Cincinnati, Ohio, United States
Children's Hospital of Philadelphia (Pediatric Site)
Philadelphia, Pennsylvania, United States
University of Pennsylvania (Adult Site)
Philadelphia, Pennsylvania, United States
Children's Hospital of Pittsburgh (Pediatric Site)
Pittsburgh, Pennsylvania, United States
University of Pittsburgh / UPMC (Adult Site)
Pittsburgh, Pennsylvania, United States
University of Texas Southwestern Medical Center (Adult)
Dallas, Texas, United States
Medical College of Wisconsin / Froedtert Memorial Luthern Hospital (Adult Site)
Milwaukee, Wisconsin, United States
IWK Health Centre
Halifax, Nova Scotia, Canada
Hospital for Sick Children (Pediatric Site)
Toronto, Ontario, Canada
Institute of Rheumatology
Prague, , Czechia
Karolinska Institute
Stockholm, , Sweden
Countries
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References
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Feldman B, Wang E, Willan A, Szalai JP. The randomized placebo-phase design for clinical trials. J Clin Epidemiol. 2001 Jun;54(6):550-7. doi: 10.1016/s0895-4356(00)00357-7.
Levine TD. Rituximab in the treatment of dermatomyositis: an open-label pilot study. Arthritis Rheum. 2005 Feb;52(2):601-7. doi: 10.1002/art.20849.
Oddis CV, Reed AM, Aggarwal R, Rider LG, Ascherman DP, Levesque MC, Barohn RJ, Feldman BM, Harris-Love MO, Koontz DC, Fertig N, Kelley SS, Pryber SL, Miller FW, Rockette HE; RIM Study Group. Rituximab in the treatment of refractory adult and juvenile dermatomyositis and adult polymyositis: a randomized, placebo-phase trial. Arthritis Rheum. 2013 Feb;65(2):314-24. doi: 10.1002/art.37754.
Related Links
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Click here for the Myositis Association Web site
Click here for the NIH Clinical Center (sub-study)
Other Identifiers
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HHSN26420042273C
Identifier Type: -
Identifier Source: secondary_id
N01 AR042273
Identifier Type: -
Identifier Source: org_study_id
NCT00393237
Identifier Type: -
Identifier Source: nct_alias
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