Single Patient Study to Treat Relapsing Polychondritis With Tocilizumab

Sponsor
Children's Hospital of Eastern Ontario
Study ID
NCT01041248
Status
Completed

Conditions

  • Relapsing Polychondritis

Eligibility Criteria

Sex
MALE
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

Study Details

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.

Key Dates

Start date
Jan 31, 2010
Status verified
Feb 2021
Primary completion
Jan 31, 2012
Completion
Jan 31, 2012

Study Design

Enrollment
1 participants (actual)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Tocilizumab
    Single arm open label study. In this arm patient will receive 8mg/kg of Tocilizumab q 2 weeks iv.

Primary Outcome Measure

Physician Global Assessment of Disease Activity [ Time Frame: Baseline and then every 2 weeks prior to each infusion for total duration of 30 weeks ]

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