Biomarker-guided treatment-and-stop-strategy for short acting IL-1 blockade in patients with systemic Juvenile Idiopathic Arthritis.

• Register: EUCTR
• Last refreshed on: 24 October 2016
• Main ID: EUCTR2015-004393-16-NL
• Date of registration: 08/07/2016
• Prospective Registration: Yes
• Primary sponsor: University Medical Center Utrecht
• Public title: Biomarker-guided treatment-and-stop-strategy for short acting IL-1 blockade in patients with systemic Juvenile Idiopathic Arthritis.
• Scientific title: Biomarker-guided treatment-and-stop-strategy for recombinant IL-1receptor antagonist (anakinra) in patients with systemic Juvenile Idiopathic Arthritis. - ESTIS trial: Early Stop of targeted Treatment in children with Systemic JIA
• Date of first enrolment: 04/10/2016
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2015-004393-16
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: yes Other trial design description: open-label stop trial If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): yes

Countries of recruitment

Netherlands

Contacts

• Name: BO&O, Bureau Onderzoek & onderwijs
• Affiliation: University Medical Center Utrecht

• Name: BO&O, Bureau Onderzoek & onderwijs
• Affiliation: University Medical Center Utrecht

Key inclusion & exclusion criteria

Inclusion criteria:
1. Children and adolescents diagnosed with sJIA;

2. Both male and female patients, aged 8 months - 16 years (anakinra is approved in children aged 8 months and older who suffer from CAPS, and as per definition, JIA has an onset before the age of 16);

3. Patients treated with anakinra as first line therapy who showed an initial response to anakinra (no fever on day 7);
4. Parents or legal guardian (and the subject when age is appropriate) who are willing to sign the consent/assent forms.

Are the trial subjects under 18? yes
Number of subjects for this age range: 55
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1. An onset of Macrophage Activation Syndrome (MAS) simultaneously with sJIA or after the diagnosis of sJIA will lead to exclusion of a (potential) subject from participation in this study;
2. Previous steroid treatment in the 3 months before diagnosis.



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Immune System Diseases [C20]

systemic Juvenile Idiopathic Arthritis

Intervention(s)

Trade Name: kineret® or anakinra
Product Name: anakinra / kineret
Product Code: not applicable
Pharmaceutical Form: Solution for injection in pre-filled syringe
INN or Proposed INN: ANAKINRA
CAS Number: 143090-92-0
Current Sponsor code: Anakinra
Other descriptive name: rIL-1RA
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 150-

Primary Outcome(s)

Main Objective: to develop a biomarker guided treatment and stop strategy for rIL-1RA in systemic Juvenile Idiopathic Arthritis

Secondary Objective: - the total number of disease flares during or after tapering and stop of therapy in the first year
- the number of patients with remission off medication at time point 1 and 2 years
- the total number of injections of anakinra per patient in the first year
- the number of patients needing to switch treatment because of treatment failure during the first year
- the number of (serious) adverse events in the first year.

Timepoint(s) of evaluation of this end point: Time point 1 year after the start of anakinra (rIL-1RA)

Primary end point(s): The number of patients with ‘clinically inactive disease’ without medication at time point 1 year after the start of anakinra (rIL-1RA)

Secondary Outcome(s)

Secondary end point(s): * the total number of disease flares during or after tapering and stop of therapy (rIL-1RA) in the first year;
* the number of patients with remission off medication at time point 1 and 2 years;
* the total number of injections of anakinra per patient;
* the number of patients needing to switch treatment because of treatment failure during the first year
* the number of (serious) adverse events in the first year.

Timepoint(s) of evaluation of this end point: Time point 1 and 2 years after the start of anakinra (rIL-1RA)

Secondary ID(s)

NL55231.041.15

Source(s) of Monetary Support

Swedish Orphan Biovitrium AB

Netherlands Organisation for Health Research and Develpment ZonMW, Rational Pharmacotherapy program