Single Patient Use of Tocilizumab in Systemic Onset Juven... | NCT00868751

Name	Class
Hoffmann-La Roche	INDUSTRY

Single Patient Use of Tocilizumab in Systemic Onset Juven... | NCT00868751 | Trialant

Arms/Interventions Module

Label	Type	Description	Intervention Names
Tocilizumab	Experimental	Single arm study - treatment only	Biological: tocilizumab

Interventions

Name	Type	Description	Arm Group Labels	Other Names
tocilizumab	Biological	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.

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Efficacy of Tocilizumab as Defined by Presence of an Equal to or Greater Than 30% Improvement in JIA Core Set (i.e. ACR JIA30 Response)		At week 12 of treatment versus week 0 (pretreatment)
Efficacy of Tocilizumab as Defined by Reduction of Oral Prednisone Dose by at Least 20%, or to Less Than 0.5mg/kg/Day, Whichever is of Lesser Daily Dose, While Maintaining an ACR JIA30 Response		At weeks 12 and 16 of treatment versus week 0 (pretreatment)
Number of Participants With at Least One Adverse Event	To evaluate the safety of tocilizumab administration in this subject	Ongoing, throughout 24 month study period

Secondary Outcomes

Measure	Description	Time Frame
Measurement of Laboratory Parameters of Active Disease, Specifically C-reactive Protein, Hemoglobin, Platelets, White Blood Cell Count, Ferritin, Immunoglobulins.	To assess normalization of laboratory parameters of active disease, specifically C-reactive protein, hemoglobin, platelets, white blood cell	At weeks 8, 12, and 16 of treatment, and every 8-12 weeks thereafter

Contacts/Locations Module

Name	Affiliation	Role
Marc D Natter, MD	Tufts Medical Center	Principal Investigator

Facility	Status	City	State	ZIP	Country	Contacts
Tufts Medical Center/Floating Hospital for Children		Boston	Massachusetts	02111	United States

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
18358927	Background	Yokota S, Imagawa T, Mori M, Miyamae T, Aihara Y, Takei S, Iwata N, Umebayashi H, Murata T, Miyoshi M, Tomiita M, Nishimoto N, Kishimoto T. Efficacy and safety of tocilizumab in patients with systemic-onset juvenile idiopathic arthritis: a randomised, double-blind, placebo-controlled, withdrawal phase III trial. Lancet. 2008 Mar 22;371(9617):998-1006. doi: 10.1016/S0140-6736(08)60454-7.

Label	URL
A Study of Tocilizumab in Patients With Active Systemic Juvenile Idiopathic Arthritis	View source

Arm/Group Information

ID	Title	Description
FG000	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.

Baseline Characteristics Module

Arm/Group Information

ID	Title	Description
BG000	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.

Units	Counts
Participants	BG000

Baseline Measures

Title	Description	Population Description	Parameter Type	Dispersion Type	Unit of Measure	Calculate Percentage	Denominator Units Selected	Denominators	Classes
Age, Categorical			Count of Participants

Outcome Measures Module

Outcome Measures

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

Primary

Efficacy of Tocilizumab as Defined by Presence of an Equal to or Greater Than 30% Improvement in JIA Core Set (i.e. ACR JIA30 Response)

No data are available because data were not collected

Posted

At week 12 of treatment versus week 0 (pretreatment)

ID	Title	Description
OG000	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.

Adverse Events Module

Arm/Groups

ID	Title	Description	Deaths (Affected)	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	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.

Serious Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Notes	Statistical Information
Hyperintensities of unclear etiology on brain MRI. Follow up revealed no progression.	Nervous system disorders

More Info Module

Certain Agreements

Title	Organization	Phone	Extension	Email
Research Administration	Tufts Medical Center			mcoughlin@tuftsmedicalcenter.org

Condition Browse Module

ID	Term
D001171	Arthritis, Juvenile

ID	Term
D001168	Arthritis
D007592	Joint Diseases
D009140	Musculoskeletal Diseases
D012216	Rheumatic Diseases

ID	Term
C502936	tocilizumab

Primary

Efficacy of Tocilizumab as Defined by Reduction of Oral Prednisone Dose by at Least 20%, or to Less Than 0.5mg/kg/Day, Whichever is of Lesser Daily Dose, While Maintaining an ACR JIA30 Response

No data are available because data were not collected

Posted

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

ID	Title	Description
OG000	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.

Units	Counts
Participants	OG0000

Primary

Number of Participants With at Least One Adverse Event

To evaluate the safety of tocilizumab administration in this subject

No data are available because data were not collected

Posted

Count of Participants

Participants

Ongoing, throughout 24 month study period

ID	Title	Description
OG000	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.

Units	Counts
Participants	OG0001

Title

Denominators

Categories

Title	Measurements
	OG0001

Secondary

Measurement of Laboratory Parameters of Active Disease, Specifically C-reactive Protein, Hemoglobin, Platelets, White Blood Cell Count, Ferritin, Immunoglobulins.

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

No data are available because data were not collected

Posted

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

ID	Title	Description
OG000	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.

Units	Counts
Participants	OG0000

Secondary

Measurement of Sustained Clinical Response to Tocilizumab, Including Active Joint Count, Joints With Limited Range of Motion, and Absence of Fever or Rash.

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

No data are available because data were not collected

Posted

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

ID	Title	Description
OG000	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.

Units	Counts
Participants	OG0000