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Effects of a T Cell-Depleting Monoclonal Antibody, Alemtuzumab, in Patients With Inclusion Body Myositis: A Pilot Clinicopathological Study

Phase 2
25 Years
80 Years
Not Enrolling
Myositis, Inclusion Body

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Trial Information

Effects of a T Cell-Depleting Monoclonal Antibody, Alemtuzumab, in Patients With Inclusion Body Myositis: A Pilot Clinicopathological Study

Sporadic Inclusion-Body Myositis (s-IBM) is the most common muscle disease in patients above
the age of 50 years. It is an inflammatory myopathy mediated by sensitized, cytotoxic CD8+
T cells that clonally expand in situ and invade MHC-I-expressing muscle fibers. The antigen
recognized by the T cells is unknown. The disease is progressive, resists the currently
available immunotherapies and leads to wheelchair confinement. Applying therapeutic
strategies with agents that deplete T cells clones and investigating the antigenic
specificity of the endomysial T cells is expected to enhance our understanding of the cause
of s-IBM and lead to clinical improvement. The present study is designed to: a) test in a
pilot study the safety, T cell depletion of the endomysial T cells and clinical efficacy of
the monoclonal antibody Alemtuzumab in 20 patients with s-IBM followed for 12 months by
serial quantitative assessment of muscle strength; b) explore the spectrum of the antigens
recognized by the T cells extracted from the muscle biopsy specimens by searching for immune
dominant peptides using positional scanning synthetic combinational peptide libraries,
before and after therapy; and c) determine the reciprocal relationship between clinical
response and endomysial inflammatory mediators before and after treatment. It is
anticipated that the study may lead to identification of putative antigens that trigger the
disease, clarify the significance of the inflammation and amyloid deposits in muscle fiber
injury and provide a novel therapy for s-IBM patients.

Inclusion Criteria

- Only patients who are currently enrolled in protocol 02-N-0121 Study of Immune
Dysregulation in Patients with Sporadic Inclusion Body Myositis (s-IBM) will be
considered for enrollment in protocol 04-N-0133.


Patients with confirmed diagnosis of s-IBM willing to undergo therapy with Alemtuzumab.

Willingness and legal ability to give and sign informed study consent.

Willingness to travel to the Clinical Center for planned studies and treatment as required
by protocol.

Men and women of reproductive potential must agree to use an acceptable method of birth
control during and for six months after completion of treatment.

Availability of tissue for testing. This will include muscle and peripheral blood
lymphocytes isolated through routine lymphocytapheresis or phlebotomy.


Immunosuppressive drug therapy at the time of or 6 months prior to enrollment.
Specifically, candidates may not be taking Prednisone, cyclosporine, tacrolimus,
azathioprine, mycophenolate mofetil, anti-lymphocyte agents, cyclophosphamide
methotrexate, or other agents whose concomitant use may enhance the toxicity of

Any medical or personal difficulties that precludes serial follow-up visits.

Any active malignancy.

Significant coagulopathy or requirement for anticoagulation therapy that would
contraindicate protocol.

Platelet count less than 100,000/mm(3).

Hemoglobin less than 9.0 mg/dl.

Any known immunodeficiency syndrome included HIV infection.

Any history of cardiac insufficiency, major vascular disease, or unstable coronary artery

Any history of systemic or pulmonary edema.

Any history of previous treatment with monoclonal antibodies or sensitivity to the study
drug (Alemtuzumab), pre-medication regimen, or prophylactic agents.

History of chronic hypotension (SBP less than 100 mmHg).

Any medical condition that would likely increase the risk of side effects of the study
drug or confound the interpretation of the data including active infections.

Pregnancy and active nursing (breast feeding).

History of uncontrolled thyroid disease or a history of autoimmune thyroiditis.

Type of Study:


Study Design:

Primary Purpose: Treatment

Outcome Measure:

Change in the muscle strength at 6 months by 15%.


United States: Federal Government

Study ID:




Start Date:

March 2004

Completion Date:

March 2007

Related Keywords:

  • Myositis, Inclusion Body
  • Inflammation
  • Myositis
  • Humanized Monoclonal Antibody
  • Cytotoxic T Cells
  • Endomysial T Cells
  • Alemtuzumab
  • Monoclonal Antibodies
  • IBM
  • Anti-CD 52
  • Inclusion Body Myositis
  • Myositis
  • Myositis, Inclusion Body



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