Single Patient Study to Treat Relapsing Polychondritis With Tocilizumab
Status:
Completed
Trial end date:
2012-01-01
Target enrollment:
Participant gender:
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.