An interventional study of Tocilizumab in Relapsing Polychondritis, sponsored by Children's Hospital of Eastern Ontario. Completed at 1 site in Canada. Open to male participants. Per ClinicalTrials.gov, last updated 2021-03-03.
Sponsored by Children's Hospital of Eastern Ontario · Not applicable, Interventional, and Treatment
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.
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.
Children's Hospital of Eastern Ontario is the lead sponsor of 83 studies on the registry; 9 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Single arm open label study. In this arm patient will receive 8mg/kg of Tocilizumab q 2 weeks iv.
Drug: Tocilizumab
8mg/kg every 2 weeks i.v.
Also known as: Actemra
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.
Time frame: Baseline and then every 2 weeks prior to each infusion for total duration of 30 weeks
Prednisone Dose
Prednisone dose administered to patient reduction through treatment course
Time frame: 30 weeks
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.
Time frame: 30 weeks
| Milestone | Tocilizumab |
|---|---|
| Started | 1 |
| Completed | 1 |
| Not completed | 0 |
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.
| mm | Tocilizumab |
|---|---|
| Baseline | 21 |
| 2 weeks | 14 |
| 4 weeks | 12 |
| 6 weeks | 43 |
| 8 weeks | 14 |
| 10 weeks | 12 |
| 12 weeks | 8 |
| 14 weeks | 6 |
| 16 weeks | 5 |
| 18 weeks | 8 |
| 20 weeks | 0 |
| 22 weeks | 2 |
| 24 weeks | 0 |
| 26 weeks | 37 |
| 28 weeks | 19 |
| 30 weeks | 0 |
Prednisone dose administered to patient reduction through treatment course
| mg | Tocilizumab |
|---|---|
| Baseline | 25 |
| 2 weeks | 25 |
| 4 weeks | 25 |
| 6 weeks | 25 |
| 8 weeks | 25 |
| 10 weeks | 30 |
| 12 weeks | 30 |
| 14 weeks | 30 |
| 16 weeks | 30 |
| 18 weeks | 30 |
| 20 weeks | 25 |
| 22 weeks | 25 |
| 24 weeks | 25 |
| 26 weeks | 20 |
| 28 weeks | 20 |
| 30 weeks | 20 |
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.
| mm | Tocilizumab |
|---|---|
| Baseline | 53 |
| 2 weeks | 0 |
| 4 weeks | 0 |
| 6 weeks | 33 |
| 8 weeks | 12 |
| 10 weeks | 2 |
| 12 weeks | 0 |
| 14 weeks | 1 |
| 16 weeks | 1 |
| 18 weeks | 2 |
| 20 weeks | 0 |
| 22 weeks | 1 |
| 24 weeks | 0 |
| 26 weeks | 30 |
| 28 weeks | 15 |
| 30 weeks | 0 |
Collected over 30 weeks. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Tocilizumab | 0/1 (0%) | 0/1 (0%) | 0/1 (0%) |
| Age, Categorical(Participants) | Tocilizumab |
|---|---|
| <=18 years | 1 |
| Between 18 and 65 years | 0 |
| >=65 years | 0 |
| Age, Continuous(years) | Tocilizumab |
|---|---|
| Mean | 10 (10 to 10) |
| Sex: Female, Male(Participants) | Tocilizumab |
|---|---|
| Female | 0 |
| Male | 1 |
| Race and Ethnicity Not Collected(Participants) | Tocilizumab |
|---|
| Region of Enrollment(participants) | Tocilizumab |
|---|---|
| Canada | 1 |
This study is completed, as verified in Feb 2021. You cannot join it, but the record below documents what was studied.
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Polychondritis, Relapsing
Children's Hospital of Eastern Ontario