Single Patient Study to Treat Relapsing Polychondritis With Tocilizumab

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01041248
Recruitment Status : Completed
First Posted : December 31, 2009
Last Update Posted : July 10, 2012
Information provided by (Responsible Party):
Johannes Roth, Children's Hospital of Eastern Ontario

Brief Summary:
Relapsing polychondritis (RP) is a rare, immune-mediated disease associated with inflammation in cartilaginous structures and other tissues throughout the body. Prognosis can be poor, especially in cases where there is acute involvement of the laryngotracheal cartilages leading to airway destruction, which are resistant to treatments such as corticosteroids, immunosuppressive or cytotoxic drugs. The pathogenesis remains unclear although it is thought that autoimmune reactions to antigens present in cartilages, such as type II collagen and matrilin may evoke symptoms. There are no known clinical or laboratory measures that predict the expression of specific disease manifestations or the overall disease course. Two recently published case reports have shown an association with elevated serum IL-6 levels and relapsing polychondritis. In these case reports, both patients with refractory relapsing polychondritis were treated with tocilizumab, a humanized monoclonal antibody to the Interleukin 6 receptor, and achieved sustained response to the drug. This single patient trial aims to evaluate the response to Tocilizumab in an eight year old boy with relapsing polychondritis who has been shown to have elevated serum IL-6 levels and who has responded poorly to conventional therapies. The study hypothesis is that Tocilizumab will be able to control the disease in this patient.

Condition or disease Intervention/treatment Phase
Relapsing Polychondritis Drug: Tocilizumab Not Applicable

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 1 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Efficacy of Tocilizumab in a Patient With Relapsing Polychondritis
Study Start Date : January 2010
Actual Primary Completion Date : January 2012
Actual Study Completion Date : January 2012

Resource links provided by the National Library of Medicine

Drug Information available for: Tocilizumab

Arm Intervention/treatment
Experimental: Tocilizumab Drug: Tocilizumab
8mg/kg every 2 weeks i.v.
Other Name: Actemra

Primary Outcome Measures :
  1. C-reactive protein levels [ Time Frame: 3 months ]

Secondary Outcome Measures :
  1. Physician Global Assessment of Disease Activity [ Time Frame: 3 months ]
  2. Parent/Patient Global Assessment of Overall Well Being [ Time Frame: 3 months ]

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Refractory relapsing polychondritis
  • Failed glucocorticoid and methotrexate therapy

Exclusion Criteria:

  • This is an N=1 clinical trial with a known patient, therefore, exclusion criteria are non-applicable.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01041248

Canada, Ontario
Children's Hospital of Eastern Ontario
Ottawa, Ontario, Canada, K1H 8L1
Sponsors and Collaborators
Children's Hospital of Eastern Ontario
Principal Investigator: Johannes Roth, MD Children's Hospital of Eastern Ontario

Responsible Party: Johannes Roth, Pediatric Rheumatologist, Children's Hospital of Eastern Ontario Identifier: NCT01041248     History of Changes
Other Study ID Numbers: ML 25245
First Posted: December 31, 2009    Key Record Dates
Last Update Posted: July 10, 2012
Last Verified: July 2012

Keywords provided by Johannes Roth, Children's Hospital of Eastern Ontario:
Relapsing Polychondritis
Single Patient Study

Additional relevant MeSH terms:
Polychondritis, Relapsing
Cartilage Diseases
Musculoskeletal Diseases
Connective Tissue Diseases