Single Patient Study to Treat Relapsing Polychondritis With Tocilizumab

NCT ID: NCT01041248

Last Updated: 2021-03-03

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 1 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2010-01-31
• Study Completion Date: 2012-01-31

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.

Conditions

Relapsing Polychondritis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Tocilizumab

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

Group Type: EXPERIMENTAL

Tocilizumab

Intervention Type: DRUG

8mg/kg every 2 weeks i.v.

Interventions

Tocilizumab

8mg/kg every 2 weeks i.v.

Intervention Type: DRUG

Other Intervention Names

Actemra

Eligibility Criteria

Inclusion Criteria

• Refractory relapsing polychondritis
• Failed glucocorticoid and methotrexate therapy

• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Children's Hospital of Eastern Ontario

OTHER

Sponsor Role: lead

Responsible Party

Johannes Roth

Pediatric Rheumatologist

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Johannes Roth, MD

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital of Eastern Ontario

Locations

Children's Hospital of Eastern Ontario

Ottawa, Ontario, Canada

Countries

Canada

References

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.

Reference Type: BACKGROUND

PMID: 19106169 (View on PubMed)

Other Identifiers

ML 25245

Identifier Type: -

Identifier Source: org_study_id