Trial Outcomes & Findings for Single Patient Use of Tocilizumab in Systemic Onset Juvenile Idiopathic Arthritis (NCT NCT00868751)

NCT ID: NCT00868751

Last Updated: 2017-05-15

Results Overview

Recruitment status

TERMINATED

Study phase

NA

Target enrollment

1 participants

Primary outcome timeframe

At week 12 of treatment versus week 0 (pretreatment)

Results posted on

2017-05-15

Participant Flow

Participant milestones

Participant milestones
Measure	Tocilizumab Single arm study - treatment only tocilizumab: Initial therapy: Tocilizumab dosed by body weight (8mg/kg based on body weight ≥ 30kg) given by intravenous infusion every two weeks for 12 weeks. Extension of therapy: Continuation of treatment with tocilizumab at 8mg/kg by body weight given by intravenous infusion every 2 weeks based upon achievement of Primary Objective by week 12, OR continuation of treatment with escalation of tocilizumab dose to 12mg/kg by body weight, given by intravenous infusion every two weeks, for failure to achieve ACR JIA30 at 12 weeks or ACR JIA50 response at any time after week 16.
Overall Study STARTED	1
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Single Patient Use of Tocilizumab in Systemic Onset Juvenile Idiopathic Arthritis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Tocilizumab n=1 Participants Single arm study - treatment only tocilizumab: Initial therapy: Tocilizumab dosed by body weight (8mg/kg based on body weight ≥ 30kg) given by intravenous infusion every two weeks for 12 weeks. Extension of therapy: Continuation of treatment with tocilizumab at 8mg/kg by body weight given by intravenous infusion every 2 weeks based upon achievement of Primary Objective by week 12, OR continuation of treatment with escalation of tocilizumab dose to 12mg/kg by body weight, given by intravenous infusion every two weeks, for failure to achieve ACR JIA30 at 12 weeks or ACR JIA50 response at any time after week 16.
Age, Categorical <=18 years	0 Participants n=99 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=99 Participants
Age, Categorical >=65 years	0 Participants n=99 Participants
Sex: Female, Male Female	0 Participants n=99 Participants
Sex: Female, Male Male	1 Participants n=99 Participants
Region of Enrollment United States	1 participants n=99 Participants

PRIMARY outcome

Timeframe: At week 12 of treatment versus week 0 (pretreatment)

Population: No data are available because data were not collected

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: At weeks 12 and 16 of treatment versus week 0 (pretreatment)

Population: No data are available because data were not collected

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: Ongoing, throughout 24 month study period

Population: No data are available because data were not collected

To evaluate the safety of tocilizumab administration in this subject

Outcome measures

Outcome measures
Measure	Tocilizumab n=1 Participants Single arm study - treatment only tocilizumab: Initial therapy: Tocilizumab dosed by body weight (8mg/kg based on body weight ≥ 30kg) given by intravenous infusion every two weeks for 12 weeks. Extension of therapy: Continuation of treatment with tocilizumab at 8mg/kg by body weight given by intravenous infusion every 2 weeks based upon achievement of Primary Objective by week 12, OR continuation of treatment with escalation of tocilizumab dose to 12mg/kg by body weight, given by intravenous infusion every two weeks, for failure to achieve ACR JIA30 at 12 weeks or ACR JIA50 response at any time after week 16.
Number of Participants With at Least One Adverse Event	1 Participants

SECONDARY outcome

Timeframe: At weeks 8, 12, and 16 of treatment, and every 8-12 weeks thereafter

Population: No data are available because data were not collected

To assess normalization of laboratory parameters of active disease, specifically C-reactive protein, hemoglobin, platelets, white blood cell

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: At weeks 8, 12, 16 of treatment, and every 8 weeks thereafter

Population: No data are available because data were not collected

To assess sustained clinical response to tocilizumab, including active joint count, joints with limited range of motion, and absence of fever

Outcome measures

Outcome data not reported

Adverse Events

Tocilizumab

Serious events: 1 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Tocilizumab n=1 participants at risk Single arm study - treatment only tocilizumab: Initial therapy: Tocilizumab dosed by body weight (8mg/kg based on body weight ≥ 30kg) given by intravenous infusion every two weeks for 12 weeks. Extension of therapy: Continuation of treatment with tocilizumab at 8mg/kg by body weight given by intravenous infusion every 2 weeks based upon achievement of Primary Objective by week 12, OR continuation of treatment with escalation of tocilizumab dose to 12mg/kg by body weight, given by intravenous infusion every two weeks, for failure to achieve ACR JIA30 at 12 weeks or ACR JIA50 response at any time after week 16.
Nervous system disorders Hyperintensities of unclear etiology on brain MRI. Follow up revealed no progression.	100.0% 1/1

Other adverse events

Adverse event data not reported

Additional Information

Research Administration

Tufts Medical Center

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place