This site became the new on June 19th. Learn more.
Show more Menu IMPORTANT: Listing of a study on this site does not reflect endorsement by the National Institutes of Health. Talk with a trusted healthcare professional before volunteering for a study. Read more... Menu IMPORTANT: Talk with a trusted healthcare professional before volunteering for a study. Read more... Menu
Give us feedback

Single Patient Study to Treat Relapsing Polychondritis With Tocilizumab

This study has been completed.
Information provided by (Responsible Party):
Johannes Roth, Children's Hospital of Eastern Ontario Identifier:
First received: December 30, 2009
Last updated: July 9, 2012
Last verified: July 2012
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 Intervention
Relapsing Polychondritis Drug: Tocilizumab

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Efficacy of Tocilizumab in a Patient With Relapsing Polychondritis

Resource links provided by NLM:

Further study details as provided by Johannes Roth, Children's Hospital of Eastern Ontario:

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

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

Enrollment: 1
Study Start Date: January 2010
Study Completion Date: January 2012
Primary Completion Date: January 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Tocilizumab Drug: Tocilizumab
8mg/kg every 2 weeks i.v.
Other Name: Actemra


Ages Eligible for Study:   Child, Adult, Senior
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: 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
  More Information

Responsible Party: Johannes Roth, Pediatric Rheumatologist, Children's Hospital of Eastern Ontario Identifier: NCT01041248     History of Changes
Other Study ID Numbers: ML 25245
Study First Received: December 30, 2009
Last Updated: July 9, 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 processed this record on September 21, 2017