MyositisPhase 2NCT00106184
Rituximab for the Treatment of Refractory Adult and Juvenile Dermatomyositis (DM) and Adult Polymyositis (PM)
A Phase 2 study testing Rituximab in people with Myositis and Dermatomyositis, run by University of Pittsburgh at 31 sites in 4 countries.
Trial summary
Rituximab is a man-made antibody used to treat certain types of cancer. This study will determine whether rituximab is an effective treatment for adult and pediatric patients with dermatomyositis or polymyositis. Read more about this study
- Who is it for?
- Ages 5 years and older
- Conditions the study is for
- Myositis, Dermatomyositis, Polymyositis
- What is being tested?
- Rituximab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 200 participants
- Where
- 31 sitesin 4 countries
- Study dates
- Mar 2006to Aug 2010
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Adults with definite or probable dermatomyositis or polymyositis and pediatric patients five years of age and over with definite or probable juvenile dermatomyositis (JDM) by Bohan and Peter criteria. Diagnosis of JDM based on an age of onset (i.e., first symptom of myositis or dermatomyositis rash) is less 18 years of age
- 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:
- An MMT-8 (Manual Muscle Test) score that is no greater than 125/150 in conjunction with 2 other abnormal core set measures.
- OR
Show all 26Show fewer
- 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
- 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
Who cannot join
- Drug-induced myositis. Patients who have myositis or myopathic syndromes caused by taking medications known to induce myositis-like syndromes, including but not limited to statin agents, fibric acid derivatives, colchicine, and hydroxychloroquine.
- Juvenile polymyositis
Trial locations and contacts
31 sites in 4 countries.
31 sites in 4 countries
United States27 sites
University of Michigan Health System (Adult Site)
Ann Arbor, Michigan, 48109
National Institute of Health (Adult Site)
Bethesda, Maryland, 20892
National Institute of Health (Pediatric Site)
Bethesda, Maryland, 20892
University of Alabama Arthritis Intervention Program (Adult Site)
Birmingham, Alabama, 35294
Children's Hospital of Boston (Pediatric Site)
Boston, Massachusetts, 02115
Beth Israel Deaconess Medical Center (Adult Site)
Boston, Massachusetts, 02215
Cincinnati's Children's Hospital (Pediatric Site)
Cincinnati, Ohio, 45229
University of Texas Southwestern Medical Center (Adult)
Dallas, Texas, 75390-8884
Duke University Medical Center (Pediatric Site)
Durham, North Carolina, 27710
Michigan State University (Adult and Pediatric Site)
Grand Rapids, Michigan, 49546
University of Kansas Medical Center (Adult Site)
Kansas City, Kansas, 66160
North Shore Long Island Jewish Health System (Adult Site)
Lake Success, New York, 11042
Kentucky Clinic (Adult Site)
Lexington, Kentucky, 40536
Cedars-Sinai Medical Center (Adult Site)
Los Angeles, California, 90048
University of Miami School of Medicine (Adult Site)
Miami, Florida, 33136
Miami Children's Hospital (Pediatric Site)
Miami, Florida, 33155
Medical College of Wisconsin / Froedtert Memorial Luthern Hospital (Adult Site)
Milwaukee, Wisconsin, 53226
Hospital for Special Surgery (Adult Site)
New York, New York, 10021
Children's Hospital of Philadelphia (Pediatric Site)
Philadelphia, Pennsylvania, 19104
University of Pennsylvania (Adult Site)
Philadelphia, Pennsylvania, 19104
Phoenix Neurological Associates, LTD (Adult Site)
Phoenix, Arizona, 85006
Children's Hospital of Pittsburgh (Pediatric Site)
Pittsburgh, Pennsylvania, 15213
University of Pittsburgh / UPMC (Adult Site)
Pittsburgh, Pennsylvania, 15261
Mayo Clinic (Adult Site)
Rochester, Minnesota, 55905
Mayo Clinic (Pediatric Site)
Rochester, Minnesota, 55905
Stanford University (Adult Site)
Stanford, California, 94305
Stanford University (Pediatric Site)
Stanford, California, 94305
Canada2 sites
IWK Health Centre
Halifax, Nova Scotia, B3K 6R8
Hospital for Sick Children (Pediatric Site)
Toronto, Ontario, M5G 1X8
About this study
Official title: Rituximab Therapy in Refractory Adult and Juvenile Idiopathic Inflammatory Myopathy (IIM)
Rituximab is a man-made antibody used to treat certain types of cancer. This study will determine whether rituximab is an effective treatment for adult and pediatric patients with dermatomyositis or polymyositis.
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.
Read the detailed description
Rituximab is a chimeric, murine-human, genetically engineered monoclonal antibody directed against the CD20 (cluster of differentiation antigen 20) antigen found on the surface of B-lymphocytes and is known to deplete B cells when administered intravenously. It is approved to treat non-Hodgkin's lymphoma. Rituximab has been used for autoimmune diseases such as systemic lupus erythematosus (SLE), rheumatoid arthritis (RA), and immune-mediated hematologic disorders. It has also been studied and used in small numbers of patients with myositis. This study will evaluate the efficacy of rituximab in treating refractory adult and pediatric patients with dermatomyositis and adult polymyositis.
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; prpl@mail.cc.nih.gov; 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
- Myositis, Dermatomyositis, Polymyositis, Juvenile Dermatomyositis
- Being tested
- Rituximab, Placebo
- Keywords
- rituximab, myositis, dermatomyositis, polymyositis, refractory, Juvenile Dermatomyositis
- Medical subject headings
- Myositis, Dermatomyositis, Polymyositis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RituximabDrug
Also called Rituxan
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
PlaceboDrug
Treatment Group A: placebo infusion at Weeks 8 and 9
Treatment Group B: placebo infusion at Weeks 0 and 1
| Study group | Role | What they receive |
|---|---|---|
| Adult Study Group 1 | Experimental | Refractory adult polymyositis patients who will receive rituximab at Weeks 0 and 1 followed by placebo at Weeks 8 and 9 (Treatment Group A) Receives: Drug: Rituximab; Drug: Placebo |
| Adult Study Group 2 | Experimental | Refractory adult polymyositis patients who will receive placebo at Weeks 0 and 1 followed by rituximab at Weeks 8 and 9 (Treatment Group B) Receives: Drug: Rituximab; Drug: Placebo |
| Adult Study Group 3 | Experimental | Adult dermatomyositis patients who will receive rituximab at Weeks 0 and 1 followed by placebo at Weeks 8 and 9 (Treatment Group A) Receives: Drug: Rituximab; Drug: Placebo |
| Adult Study Group 4 | Experimental | Adult dermatomyositis patients who will receive placebo at Weeks 0 and 1 followed by rituximab at Weeks 8 and 9 (Treatment Group B) Receives: Drug: Rituximab; Drug: Placebo |
| JDM Study Group 1 | Experimental | Refractory juvenile dermatomyositis patients who will receive rituximab at Weeks 0 and 1 followed by placebo at Weeks 8 and 9 (Treatment Group A) Receives: Drug: Rituximab; Drug: Placebo |
| JDM Study Group 2 | Experimental | Refractory juvenile dermatomyositis patients who will receive placebo at Weeks 0 and 1 followed by rituximab at Weeks 8 and 9 (Treatment Group B) Receives: Drug: Rituximab; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Comparison Between the Time to Improvement Between the Two Groups of IIM (Idiopathic Inflammatory Myopathy) Patients
The Definition of Improvement for both adult and pediatric patients will be: 3 of any of the 6 core set measures improved by ≥ 20%, with no more than 2 of the core set measures worsening by ≥25% (worsening measure cannot include the MMT) at two consecutive visits. Of note, the MMT could not be one of the worsening measures.
Core Set Measures Included:
- Manual Muscle Testing (MMT)- Muscle Strength
- Physician Global Disease Activity VAS Score
- Health Assessment Questionnaire Index Score - Physical Function
- Patient Global Assessment of Disease Activity VAS score
- Extramuscular Activity - Myositis Disease Activity Assessment Tool
- 2 or more elevated muscle enzymes (Aldolase, CK, AST, ALT, and LDH)
Measured: Week 44 of treatment phase
Secondary outcomes (2)
Response Rates (Proportion of Improved Patients) Between Groups A (Rituximab Wks 0 and 1) and B (Rituximab Wks 8 and 9) at Week 8
The Definition of Improvement for both adult and pediatric patients will be: 3 of any of the 6 core set measures improved by ≥ 20%, with no more than 2 of the core set measures worsening by ≥25% (worsening measure cannot include the MMT) at two consecutive visits. Of note, the MMT could not be one of the worsening measures.
Core Set Measures Included:
- Manual Muscle Testing (MMT)- Muscle Strength
- Physician Global Disease Activity VAS Score
- Health Assessment Questionnaire Index Score - Physical Function
- Patient Global Assessment of Disease Activity VAS score
- Extramuscular Activity - Myositis Disease Activity Assessment Tool
- 2 or more elevated muscle enzymes (Aldolase, CK, AST, ALT, and LDH)
Measured: Week 8 of the treatment phase
20% Improvement in Manual Muscle Testing (MMT) Over Baseline on Two Consecutive Time Points (Muscle is the Primary Organ of Involvement, and MMT is the One Objective Measurement of the Definition of Improvement [DOI])
Number of participants with a 20% improvement in MMT over baseline on two consecutive time points.
Measured: Week 44 of treatment phase
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Mar 2005 (estimated)
- Study startMar 2006
- Primary completionFeb 2010
- Study completionAug 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for MyositisStudies of the same condition, open and under way.
- More trials run by University of PittsburghThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 200 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Pittsburgh
- Sponsor type
- University, hospital or other
- Collaborators
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), Genentech, Inc., Biogen
- Responsible party
- Principal investigator, Chester Oddis, University of Pittsburgh
- Investigators
- Chester V. Oddis, MD, Principal investigator, University of PittsburghAnn M. Reed, MD, Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00106184
- Sponsor's protocol ID
- N01 AR042273
- Other IDs
- 5R01AR061298-02, HHSN26420042273C
- Status last verified
- Mar 2015
- First submitted
- 21 Mar 2005
- Last updated
- 4 Mar 2015
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
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.
PubMed 11377114Levine 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.
PubMed 15692974Oddis 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.
PubMed 23124935
Study at a glance
From the registry record, last updated by the sponsor on 4 Mar 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Rituximab Therapy in Refractory Adult and Juvenile Idiopathic Inflammatory Myopathy (IIM)
- ClinicalTrials.gov ID
- NCT00106184
- Acronym / sponsor ID
- N01 AR042273
- Conditions
- Myositis, Dermatomyositis, Polymyositis, Juvenile Dermatomyositis
- Keywords
- rituximab, myositis, dermatomyositis, polymyositis, refractory, Juvenile Dermatomyositis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 200 participants (actual)
- Lead sponsor
- University of PittsburghUniversity, hospital or other
- Collaborators
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), Genentech, Inc., Biogen
- Study start
- Mar 2006
- Primary completion
- Feb 2010 (actual)
- Study completion
- Aug 2010 (actual)
- First posted
- 22 Mar 2005 (estimated)
- Results
- Posted 4 Mar 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Rituximab is a man-made antibody used to treat certain types of cancer. This study will determine whether rituximab is an effective treatment for adult and pediatric patients with dermatomyositis or polymyositis.
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.
Detailed description
Rituximab is a chimeric, murine-human, genetically engineered monoclonal antibody directed against the CD20 (cluster of differentiation antigen 20) antigen found on the surface of B-lymphocytes and is known to deplete B cells when administered intravenously. It is approved to treat non-Hodgkin's lymphoma. Rituximab has been used for autoimmune diseases such as systemic lupus erythematosus (SLE), rheumatoid arthritis (RA), and immune-mediated hematologic disorders. It has also been studied and used in small numbers of patients with myositis. This study will evaluate the efficacy of rituximab in treating refractory adult and pediatric patients with dermatomyositis and adult polymyositis.
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; prpl@mail.cc.nih.gov; 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| Adult Study Group 1 | Experimental | Drug: Rituximab, Drug: PlaceboRefractory adult polymyositis patients who will receive rituximab at Weeks 0 and 1 followed by placebo at Weeks 8 and 9 (Treatment Group A) |
| Adult Study Group 2 | Experimental | Drug: Rituximab, Drug: PlaceboRefractory adult polymyositis patients who will receive placebo at Weeks 0 and 1 followed by rituximab at Weeks 8 and 9 (Treatment Group B) |
| Adult Study Group 3 | Experimental | Drug: Rituximab, Drug: PlaceboAdult dermatomyositis patients who will receive rituximab at Weeks 0 and 1 followed by placebo at Weeks 8 and 9 (Treatment Group A) |
| Adult Study Group 4 | Experimental | Drug: Rituximab, Drug: PlaceboAdult dermatomyositis patients who will receive placebo at Weeks 0 and 1 followed by rituximab at Weeks 8 and 9 (Treatment Group B) |
| JDM Study Group 1 | Experimental | Drug: Rituximab, Drug: PlaceboRefractory juvenile dermatomyositis patients who will receive rituximab at Weeks 0 and 1 followed by placebo at Weeks 8 and 9 (Treatment Group A) |
| JDM Study Group 2 | Experimental | Drug: Rituximab, Drug: PlaceboRefractory juvenile dermatomyositis patients who will receive placebo at Weeks 0 and 1 followed by rituximab at Weeks 8 and 9 (Treatment Group B) |
Who may qualify
- Ages
- 5 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (26 listed)
- Adults with definite or probable dermatomyositis or polymyositis and pediatric patients five years of age and over with definite or probable juvenile dermatomyositis (JDM) by Bohan and Peter criteria. Diagnosis of JDM based on an age of onset (i.e., first symptom of myositis or dermatomyositis rash) is less 18 years of age
- 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:
- An MMT-8 (Manual Muscle Test) score that is no greater than 125/150 in conjunction with 2 other abnormal core set measures.
Key exclusion criteria (2 listed)
- Drug-induced myositis. Patients who have myositis or myopathic syndromes caused by taking medications known to induce myositis-like syndromes, including but not limited to statin agents, fibric acid derivatives, colchicine, and hydroxychloroquine.
- Juvenile polymyositis
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RituximabAlso: RituxanTreatment 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 | Drug | Adult Study Group 1, Adult Study Group 2, Adult Study Group 3, Adult Study Group 4, JDM Study Group 1, JDM Study Group 2 |
| PlaceboTreatment Group A: placebo infusion at Weeks 8 and 9 Treatment Group B: placebo infusion at Weeks 0 and 1 | Drug | Adult Study Group 1, Adult Study Group 2, Adult Study Group 3, Adult Study Group 4, JDM Study Group 1, JDM Study Group 2 |
Primary outcome
Comparison Between the Time to Improvement Between the Two Groups of IIM (Idiopathic Inflammatory Myopathy) Patients
Time frame: Week 44 of treatment phase
The Definition of Improvement for both adult and pediatric patients will be: 3 of any of the 6 core set measures improved by ≥ 20%, with no more than 2 of the core set measures worsening by ≥25% (worsening measure cannot include the MMT) at two consecutive visits. Of note, the MMT could not be one of the worsening measures. Core Set Measures Included: 1. Manual Muscle Testing (MMT)- Muscle Strength 2. Physician Global Disease Activity VAS Score 3. Health Assessment Questionnaire Index Score - Physical Function 4. Patient Global Assessment of Disease Activity VAS score 5. Extramuscular Activity - Myositis Disease Activity Assessment Tool 6. 2 or more elevated muscle enzymes (Aldolase, CK, AST, ALT, and LDH)
2 secondary outcomes
Response Rates (Proportion of Improved Patients) Between Groups A (Rituximab Wks 0 and 1) and B (Rituximab Wks 8 and 9) at Week 8
Time frame: Week 8 of the treatment phase
20% Improvement in Manual Muscle Testing (MMT) Over Baseline on Two Consecutive Time Points (Muscle is the Primary Organ of Involvement, and MMT is the One Objective Measurement of the Definition of Improvement [DOI])
Time frame: Week 44 of treatment phase
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Mar 2015.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 11377114Feldman 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.
- Background: PubMed 15692974Levine 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.
- Results publication: PubMed 23124935Oddis 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.
- Click here for the Myositis Association Web siteLink provided by the sponsor.
- Click here for the NIH Clinical Center (sub-study)Link provided by the sponsor.
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Dermatomyositis, Myositis
RecruitingPhase 1
A Study to Investigate the Safety and Preliminary Efficacy of ALLO-329, an Allogeneic CAR T-cell Therapy, in Adults With Autoimmune Disease
Systemic Lupus Erythematosus (With and Without Nephritis), Idiopathic Inflammatory Myopathy
RecruitingPhase 1/2
A Study of AZD0120 in Autoimmune Diseases
Systemic Sclerosis, Idiopathic Inflammatory Myopathies
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Chester V. Oddis, MD principal investigator
University of Pittsburgh
Ann M. Reed, MD principal investigator
Mayo Clinic
31 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Rituximab in people with Myositis and Dermatomyositis, run by University of Pittsburgh at 31 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 26 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Mar 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 31 sites in United States, Canada, Czechia, Sweden. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 4 Mar 2015). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Comparison Between the Time to Improvement Between the Two Groups of IIM (Idiopathic Inflammatory Myopathy) Patients (time frame: Week 44 of treatment phase). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00106184, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Mar 2015; AzTrials last refreshed its copy on 8 Nov 2026.