Single Patient Study to Treat Relapsing Polychondritis With Tocilizumab

Relapsing Polychondritis Clinical Trial

Official title:

Efficacy of Tocilizumab in a Patient With Relapsing Polychondritis

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01041248
• Other study ID #: ML 25245
• Status: Completed
• Phase: N/A
• Start date: January 2010
• Est. completion date: January 2012

Study information

• Verified date: February 2021
• Source: Children's Hospital of Eastern Ontario
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 1
• Est. completion date: January 2012
• Est. primary completion date: January 2012
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Relapsing Polychondritis

Intervention

Drug:
• Tocilizumab
8mg/kg every 2 weeks i.v.

Locations

Country	Name	City	State
Canada	Children's Hospital of Eastern Ontario	Ottawa	Ontario

Sponsors (1)

Lead Sponsor	Collaborator
Children's Hospital of Eastern Ontario

Country where clinical trial is conducted

Canada, 

References & Publications (1)

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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Physician Global Assessment of Disease Activity	Physician global assessment of disease activity was assessed on a 100 mm Visual Analogue Scale where 0 would be no disease activity and 100 would be the maximum disease activity. Higher values therefore indicate higher disease activity and therefore a worse outcome. Change of this outcome measure over time was documented.	Baseline and then every 2 weeks prior to each infusion for total duration of 30 weeks
Secondary	Prednisone Dose	Prednisone dose administered to patient reduction through treatment course	30 weeks
Secondary	Parent/Patient Global Assessment of Overall Well Being	A 100 mm visual analogue scale was used for the assessment of the parent/patient globale well being, maximum value is 100 and minimum value is 0 with lower values being better well being and therefore improved outcome and higher values worse well being and therefore worse outcome. Changes in this score over time are being assessed with this measure.	30 weeks