Single Patient Use of Tocilizumab in Systemic Onset Juvenile Idiopathic Arthritis

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT00868751
• Date of registration: 24/03/2009
• Prospective Registration: No
• Primary sponsor: Tufts Medical Center
• Public title: Single Patient Use of Tocilizumab in Systemic Onset Juvenile Idiopathic Arthritis
• Scientific title: Single Patient Use of Tocilizumab for Treatment of Steroid Dependent, Active Systemic Onset Juvenile Idiopathic Arthritis
• Date of first enrolment: March 2009
• Target sample size: 1
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT00868751
• Study type: Interventional
• Study design: Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: N/A

Countries of recruitment

United States

Contacts

• Name: Marc D Natter, MD
• Affiliation: Tufts Medical Center

Key inclusion & exclusion criteria

Inclusion Criteria:

- 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

Exclusion Criteria:

- 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



Age minimum: 2 Years
Age maximum: 16 Years
Gender: All

Health Condition(s) or Problem(s) studied

Arthritis, Juvenile Rheumatoid

Still's Disease, Juvenile Onset

Intervention(s)

Biological: tocilizumab

Primary Outcome(s)

Number of Participants With at Least One Adverse Event [Time Frame: Ongoing, throughout 24 month study period]

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) [Time Frame: 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 [Time Frame: At weeks 12 and 16 of treatment versus week 0 (pretreatment)]

Secondary Outcome(s)

Measurement of Laboratory Parameters of Active Disease, Specifically C-reactive Protein, Hemoglobin, Platelets, White Blood Cell Count, Ferritin, Immunoglobulins. [Time Frame: 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. [Time Frame: At weeks 8, 12, 16 of treatment, and every 8 weeks thereafter]

Secondary ID(s)

TMC-PRHEU-TCZ-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Hoffmann-La Roche

Results

• Results available: Yes
• Date Posted: 15/05/2017
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00868751