Clinical trial NCT01041248: Single Patient Study to Treat Relapsing Polychondritis With Tocilizumab
Official title: Efficacy of Tocilizumab in a Patient With Relapsing Polychondritis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Relapsing Polychondritis
Keywords: Relapsing Polychondritis
Single Patient Study
Tocilizumab
Lead sponsor: Children's Hospital of Eastern Ontario
Sponsor class: OTHER
First posted: 2009-12-31
Enrollment: 1
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Detailed description: In this N = 1 study a single known patient with relapsing polychondritis who has failed methotrexate, various anti TNF medications, anti IL1 medication and prolongued glucocorticosteroids will be recruited to receive Tocilizumab 8 mg /kg q 2 weeks iv.

The objective is to assess efficacy of tociliuzmab in combination with stable ongoing therapy. Our patient received tocilizumab 8 mg/kg over 1 hour by intravenous infusion every 2 weeks throughout the course of the study. To assess tocilizumab efficacy, the primary objective is the change in physician global assessment on a 100-mm horizontal visual analogue scale (VAS) of disease activity.

The secondary objectives were the change in parent global assessment of disease activity on a 100 mm VAS and the glucocorticoid dose in mg per day. Frequency of adverse events was also measured at baseline and after each biweekly tocilizumab infusion.

Eligibility 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.

Primary outcomes:
measure: Physician Global Assessment of Disease Activity
timeFrame: Baseline and then every 2 weeks prior to each infusion for total duration of 30 weeks
Secondary outcomes:
measure: Prednisone Dose
timeFrame: 30 weeks
measure: Parent/Patient Global Assessment of Overall Well Being
timeFrame: 30 weeks
Other outcomes:
Locations:
city: Ottawa
country: Canada
facility: Children's Hospital of Eastern Ontario
state: Ontario
Study officials:
affiliation: Children's Hospital of Eastern Ontario
name: Johannes Roth, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Kawai M, Hagihara K, Hirano T, Shima Y, Kuwahara Y, Arimitsu J, Narazaki M, Ogata A, Kawase I, Kishimoto T, Tanaka T. Sustained response to tocilizumab, anti-interleukin-6 receptor antibody, in two patients with refractory relapsing polychondritis. Rheumatology (Oxford). 2009 Mar;48(3):318-9. doi: 10.1093/rheumatology/ken468. Epub 2008 Dec 23. No abstract available.