Single Patient Study to Treat Relapsing Polychondritis With Tocilizumab

NCT01041248 | Completed Results

• 1 other identifier: ML 25245
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

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.

Conditions

Relapsing Polychondritis

Keywords

Relapsing Polychondritis; Single Patient Study; Tocilizumab

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (2)

• Prednisone Dose

  30 weeks

• Parent/Patient Global Assessment of Overall Well Being

  30 weeks

Study Arms (1)

Tocilizumab

EXPERIMENTAL

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

Drug: Tocilizumab

Interventions

Tocilizumab DRUG

8mg/kg every 2 weeks i.v.

Also known as: Actemra

Tocilizumab

Eligibility Criteria

• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Refractory relapsing polychondritis
• Failed glucocorticoid and methotrexate therapy

Sponsors & Collaborators

• Children's Hospital of Eastern Ontario: lead

Study Sites (1)

Children's Hospital of Eastern Ontario

Ottawa, Ontario, K1H 8L1, Canada

Location

Related 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. No abstract available.

  PMID: 19106169 BACKGROUND

MeSH Terms

Conditions

Polychondritis, Relapsing

Interventions

tocilizumab

Condition Hierarchy (Ancestors)

Cartilage Diseases; Musculoskeletal Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases

Results Point of Contact

• Title: Dr Johannes Roth
• Organization: Childrens Hospital of Eastern Ontario

jroth@cheo.on.ca

6137377600

Study Officials

• Johannes Roth, MD

  Children's Hospital of Eastern Ontario

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Pediatric Rheumatologist

Study Record Dates

• First Submitted: December 30, 2009
• First Posted: December 31, 2009
• Study Start: January 1, 2010
• Primary Completion: January 1, 2012
• Study Completion: January 1, 2012
• Last Updated: March 3, 2021
• Results First Posted: March 3, 2021

Record last verified: 2021-02

Locations

CA (1)