Single Patient Study to Treat Relapsing Polychondritis With Tocilizumab

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Johannes Roth, Children's Hospital of Eastern Ontario
ClinicalTrials.gov Identifier:
NCT01041248
First received: December 30, 2009
Last updated: July 9, 2012
Last verified: July 2012
  Purpose

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: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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 Children's Hospital of Eastern Ontario:

Primary Outcome Measures:
  • C-reactive protein levels [ Time Frame: 3 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Physician Global Assessment of Disease Activity [ Time Frame: 3 months ] [ Designated as safety issue: No ]
  • Parent/Patient Global Assessment of Overall Well Being [ Time Frame: 3 months ] [ Designated as safety issue: No ]

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

  Eligibility

Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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 ClinicalTrials.gov identifier: NCT01041248

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

Publications:
Responsible Party: Johannes Roth, Pediatric Rheumatologist, Children's Hospital of Eastern Ontario
ClinicalTrials.gov Identifier: NCT01041248     History of Changes
Other Study ID Numbers: ML 25245
Study First Received: December 30, 2009
Last Updated: July 9, 2012
Health Authority: Canada: Health Canada

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

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

ClinicalTrials.gov processed this record on September 22, 2014