Rituximab for the Treatment of Refractory Adult and Juvenile Dermatomyositis (DM) and Adult Polymyositis (PM)

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00106184
• Date of registration: 21/03/2005
• Prospective Registration: Yes
• Primary sponsor: University of Pittsburgh
• Public title: Rituximab for the Treatment of Refractory Adult and Juvenile Dermatomyositis (DM) and Adult Polymyositis (PM)
• Scientific title: Rituximab Therapy in Refractory Adult and Juvenile Idiopathic Inflammatory Myopathy (IIM)
• Date of first enrolment: March 2006
• Target sample size: 200
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00106184
• Study type: Interventional
• Phase: Phase 2

Countries of recruitment

Canada, Czech Republic, Sweden, United Kingdom, United States

Contacts

• Name: Ann M. Reed, MD
• Affiliation: Mayo Clinic

• Name: Chester V. Oddis, MD
• Affiliation: University of Pittsburgh

Key inclusion & exclusion criteria

Inclusion Criteria:

- 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:

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:

- 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

- 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



Age minimum: 5 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Dermatomyositis

Myositis

Polymyositis

Juvenile Dermatomyositis

Intervention(s)

Drug: Placebo

Drug: Rituximab

Primary Outcome(s)

Comparison Between the Time to Improvement Between the Two Groups of IIM (Idiopathic Inflammatory Myopathy) Patients [Time Frame: Week 44 of treatment phase]

Secondary Outcome(s)

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]

Secondary ID(s)

N01 AR042273

5R01AR061298-02

HHSN26420042273C

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Genentech, Inc.

National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

Biogen

Results

• Results available: Yes
• Date Posted: 04/03/2015
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00106184