Single Patient Use of Tocilizumab in Systemic Onset Juvenile Idiopathic Arthritis

NCT00868751 | Terminated Results

• 1 other identifier: TMC-PRHEU-TCZ-01
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to see if tocilizumab is safe and effective for treating systemic onset Juvenile Idiopathic Arthritis (soJIA). Another purpose is to see if tocilizumab helps reduce the amount of steroids (prednisone) needed to control symptoms of soJIA.

Conditions

Arthritis, Juvenile Rheumatoid; Still's Disease, Juvenile Onset

Keywords

Antibodies, Monoclonal; Interleukin 6

Outcome Measures

Primary Outcomes (3)

• 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 (2)

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

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

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

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

Study Arms (1)

Tocilizumab

EXPERIMENTAL

Single arm study - treatment only

Biological: tocilizumab

Interventions

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.

Also known as: Actemra, RoACTEMRA, MRA

Tocilizumab

Eligibility Criteria

• Age: 2 Years - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Systemic Juvenile Idiopathic Arthritis according to ILAR criteria (2001)
• Duration of disease ≥ 6 months since onset
• Presence of active disease as determined by the presence of at least 5 active joints, OR at least 2 active joints if receiving prednisone at a dose \> 0.2 mg/kg/day or \> 10 mg/day (whichever is less)
• Incomplete prior response to methotrexate treatment for at least 3 months at a minimum dose of 15 mg/M2/week, or intolerance to methotrexate
• Discontinued treatment with other biologics prior to first tocilizumab infusion, for approximately two pharmacokinetic half-lives as per specific biologic (e.g. 48 hours for anakinra, 7 days for etanercept)
• Not receiving corticosteroids, OR taking oral corticosteroids and the dose has remained stable for 1 week prior to the first tocilizumab infusion at ≤ 2 mg/kg/day prednisone or prednisolone and no more than 80 mg/day

You may not qualify if:

• Concomitant administration of biologic therapies
• Serum creatinine \>1.5 ULN (upper limits normal)
• AST or ALT \> 1.5 ULN
• Total bilirubin \> 1.3 mg/dL
• Platelet count \< LLN (lower limits normal)
• Hemoglobin \< 6.0 g/dL
• WBC count \< 5,000/mm3
• Neutrophil count \< 2,000/ mm3
• Fibrinogen \< LLN

Sponsors & Collaborators

• Tufts Medical Center: lead
• Hoffmann-La Roche: collaborator

Study Sites (1)

Tufts Medical Center/Floating Hospital for Children

Boston, Massachusetts, 02111, United States

Location

Related Publications (1)

• 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.

  PMID: 18358927 BACKGROUND

Related Links

• A Study of Tocilizumab in Patients With Active Systemic Juvenile Idiopathic Arthritis

MeSH Terms

Conditions

Arthritis, Juvenile

Interventions

tocilizumab

Condition Hierarchy (Ancestors)

Arthritis; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases

Results Point of Contact

• Title: Research Administration
• Organization: Tufts Medical Center

mcoughlin@tuftsmedicalcenter.org

Study Officials

• Marc D Natter, MD

  Tufts Medical Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 24, 2009
• First Posted: March 25, 2009
• Study Start: March 1, 2009
• Primary Completion: November 1, 2009
• Study Completion: June 1, 2010
• Last Updated: May 15, 2017
• Results First Posted: May 15, 2017

Record last verified: 2017-05

Locations

US (1)