Trial Outcomes & Findings for Single Patient Study to Treat Relapsing Polychondritis With Tocilizumab (NCT NCT01041248)

NCT ID: NCT01041248

Last Updated: 2021-03-03

Results Overview

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.

• Recruitment status: COMPLETED
• Study phase: NA
• Target enrollment: 1 participants
• Primary outcome timeframe: Baseline and then every 2 weeks prior to each infusion for total duration of 30 weeks
• Results posted on: 2021-03-03

Participant Flow

Participant milestones

Measure	Tocilizumab Tocilizumab: 8mg/kg every 2 weeks i.v.
Overall Study STARTED	1
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Measure	Tocilizumab n=1 Participants Tocilizumab: 8mg/kg every 2 weeks i.v.
Age, Categorical <=18 years	1 Participants n=1 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=1 Participants
Age, Categorical >=65 years	0 Participants n=1 Participants
Age, Continuous	10 years n=1 Participants
Sex: Female, Male Female	0 Participants n=1 Participants
Sex: Female, Male Male	1 Participants n=1 Participants
Region of Enrollment Canada	1 participants n=1 Participants

PRIMARY outcome

Timeframe: Baseline and then every 2 weeks prior to each infusion for total duration of 30 weeks

Population: a known patient for whom this trial was designed was recruited into this open label single arm study

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.

Outcome measures

Measure	Tocilizumab n=1 Participants Tocilizumab: 8mg/kg every 2 weeks i.v.
Physician Global Assessment of Disease Activity Baseline	21 mm
Physician Global Assessment of Disease Activity 2 weeks	14 mm
Physician Global Assessment of Disease Activity 4 weeks	12 mm
Physician Global Assessment of Disease Activity 6 weeks	43 mm
Physician Global Assessment of Disease Activity 8 weeks	14 mm
Physician Global Assessment of Disease Activity 10 weeks	12 mm
Physician Global Assessment of Disease Activity 12 weeks	8 mm
Physician Global Assessment of Disease Activity 14 weeks	6 mm
Physician Global Assessment of Disease Activity 16 weeks	5 mm
Physician Global Assessment of Disease Activity 18 weeks	8 mm
Physician Global Assessment of Disease Activity 20 weeks	0 mm
Physician Global Assessment of Disease Activity 22 weeks	2 mm
Physician Global Assessment of Disease Activity 24 weeks	0 mm
Physician Global Assessment of Disease Activity 26 weeks	37 mm
Physician Global Assessment of Disease Activity 28 weeks	19 mm
Physician Global Assessment of Disease Activity 30 weeks	0 mm

SECONDARY outcome

Timeframe: 30 weeks

Population: single arm open label study for 1 patient with relapsing polychondritis

Prednisone dose administered to patient reduction through treatment course

Outcome measures

Measure	Tocilizumab n=1 Participants Tocilizumab: 8mg/kg every 2 weeks i.v.
Prednisone Dose 20 weeks	25 mg
Prednisone Dose Baseline	25 mg
Prednisone Dose 2 weeks	25 mg
Prednisone Dose 4 weeks	25 mg
Prednisone Dose 6 weeks	25 mg
Prednisone Dose 8 weeks	25 mg
Prednisone Dose 10 weeks	30 mg
Prednisone Dose 12 weeks	30 mg
Prednisone Dose 14 weeks	30 mg
Prednisone Dose 16 weeks	30 mg
Prednisone Dose 18 weeks	30 mg
Prednisone Dose 22 weeks	25 mg
Prednisone Dose 24 weeks	25 mg
Prednisone Dose 26 weeks	20 mg
Prednisone Dose 28 weeks	20 mg
Prednisone Dose 30 weeks	20 mg

SECONDARY outcome

Timeframe: 30 weeks

Population: Patient receiving Tocilizumab

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.

Outcome measures

Measure	Tocilizumab n=1 Participants Tocilizumab: 8mg/kg every 2 weeks i.v.
Parent/Patient Global Assessment of Overall Well Being Baseline	53 mm
Parent/Patient Global Assessment of Overall Well Being 2 weeks	0 mm
Parent/Patient Global Assessment of Overall Well Being 4 weeks	0 mm
Parent/Patient Global Assessment of Overall Well Being 6 weeks	33 mm
Parent/Patient Global Assessment of Overall Well Being 8 weeks	12 mm
Parent/Patient Global Assessment of Overall Well Being 10 weeks	2 mm
Parent/Patient Global Assessment of Overall Well Being 12 weeks	0 mm
Parent/Patient Global Assessment of Overall Well Being 14 weeks	1 mm
Parent/Patient Global Assessment of Overall Well Being 16 weeks	1 mm
Parent/Patient Global Assessment of Overall Well Being 18 weeks	2 mm
Parent/Patient Global Assessment of Overall Well Being 20 weeks	0 mm
Parent/Patient Global Assessment of Overall Well Being 22 weeks	1 mm
Parent/Patient Global Assessment of Overall Well Being 24 weeks	0 mm
Parent/Patient Global Assessment of Overall Well Being 26 weeks	30 mm
Parent/Patient Global Assessment of Overall Well Being 28 weeks	15 mm
Parent/Patient Global Assessment of Overall Well Being 30 weeks	0 mm

Adverse Events

Tocilizumab

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr Johannes Roth

Childrens Hospital of Eastern Ontario

Phone: 6137377600

Email: jroth@cheo.on.ca

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place