Relapsing PolychondritisNCT01041248
Single Patient Study to Treat Relapsing Polychondritis With Tocilizumab
A study testing Tocilizumab in people with Relapsing Polychondritis, run by Children's Hospital of Eastern Ontario at 1 site in Canada.
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… Read more about this study
- Who is it for?
- Any age, men only
- Conditions the study is for
- Relapsing Polychondritis
- What is being tested?
- Tocilizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteCanada
- Study dates
- Jan 2010to Jan 2012
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Refractory relapsing polychondritis
- Failed glucocorticoid and methotrexate therapy
Who cannot join
- This is an N=1 clinical trial with a known patient, therefore, exclusion criteria are non-applicable.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Efficacy of Tocilizumab in a Patient With Relapsing Polychondritis
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.
Read the 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
- Being tested
- Tocilizumab
- Keywords
- Relapsing Polychondritis, Single Patient Study, Tocilizumab
- Medical subject headings
- Polychondritis, Relapsing
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TocilizumabDrug
Also called Actemra
8mg/kg every 2 weeks i.v.
| Study group | Role | What they receive |
|---|---|---|
| Tocilizumab | Experimental | Single arm open label study. In this arm patient will receive 8mg/kg of Tocilizumab q 2 weeks iv. Receives: Drug: Tocilizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline and then every 2 weeks prior to each infusion for total duration of 30 weeks
Secondary outcomes (2)
Prednisone Dose
Prednisone dose administered to patient reduction through treatment course
Measured: 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.
Measured: 30 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Dec 2009 (estimated)
- Study startJan 2010
- Primary completionJan 2012
- Study completionJan 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Relapsing PolychondritisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital of Eastern Ontario
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Johannes Roth, Children's Hospital of Eastern Ontario
- Investigators
- Johannes Roth, MD, Principal investigator, Children's Hospital of Eastern Ontario
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01041248
- Sponsor's protocol ID
- ML 25245
- Status last verified
- Feb 2021
- First submitted
- 30 Dec 2009
- Last updated
- 3 Mar 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
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.
PubMed 19106169
Study at a glance
From the registry record, last updated by the sponsor on 3 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy of Tocilizumab in a Patient With Relapsing Polychondritis
- ClinicalTrials.gov ID
- NCT01041248
- Acronym / sponsor ID
- ML 25245
- Conditions
- Relapsing Polychondritis
- Keywords
- Relapsing Polychondritis, Single Patient Study, Tocilizumab
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Children's Hospital of Eastern OntarioUniversity, hospital or other
- Study start
- Jan 2010
- Primary completion
- Jan 2012 (actual)
- Study completion
- Jan 2012 (actual)
- First posted
- 31 Dec 2009 (estimated)
- Results
- Posted 3 Mar 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Tocilizumab | Experimental | Drug: TocilizumabSingle arm open label study. In this arm patient will receive 8mg/kg of Tocilizumab q 2 weeks iv. |
Who may qualify
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Refractory relapsing polychondritis
- Failed glucocorticoid and methotrexate therapy
Key exclusion criteria (1 listed)
- This is an N=1 clinical trial with a known patient, therefore, exclusion criteria are non-applicable.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TocilizumabAlso: Actemra8mg/kg every 2 weeks i.v. | Drug | Tocilizumab |
Primary outcome
Physician Global Assessment of Disease Activity
Time frame: Baseline and then every 2 weeks prior to each infusion for total duration of 30 weeks
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.
2 secondary outcomes
Prednisone Dose
Time frame: 30 weeks
Parent/Patient Global Assessment of Overall Well Being
Time frame: 30 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Mar 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 19106169Kawai 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Johannes Roth, MD principal investigator
Children's Hospital of Eastern Ontario
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Tocilizumab in people with Relapsing Polychondritis, run by Children's Hospital of Eastern Ontario at 1 site in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 3 Mar 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Physician Global Assessment of Disease Activity (time frame: Baseline and then every 2 weeks prior to each infusion for total duration of 30 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01041248, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Mar 2021.