No registrations found.
ID
Source
Brief title
Health condition
juvenile idiopathic arthritis
Sponsors and support
Intervention
Outcome measures
Primary outcome
Total number of adverse events in the 3 months prior to MSC infusion and the number of adverse events 3 months after MSC infusion.
To offer an effective alternative for that category of JIA patients that is therapy-resistant.
For this effectiveness the ACR Pediatric 70 criteria should be achieved.
The ACR Pedi 70 criteria are defined as improvement of >= 70% in at least 3 of 6 core response variables used to assess disease activity with no more than 1 variable worsening by >= 30%.
Secondary outcome
- The ACR Pediatric 30 criteria should be met.
The ACR Pedi 30 criteria are defined as improvement of ≥ 30% in at least 3 of 6 core response variables used to assess disease activity with no more than 1 variable worsening by ≥ 30%.
Core response variables are:
1. Physician global assessment of overall disease activity
2. Parent or patient global assessment of overall well-being
3. Functional ability (CHAQ)
4. Number of joints with active arthritis
5. Number of joints with limited range of motion
6. Index of inflammation: ESR or CRP
- Radiological (MRI) improvement of most active large joint.
- Improvement in laboratory parameters or biomarkers.
Background summary
The main objective is to offer a safe alternative for that category of JIA patients that is therapy-resistant. We hypothesize that intravenous administration of MSC in therapy refractory JIA patients is safe and has the potential to have clinical relevant effect as measured by the ACR Pedi 30.
Aims:
1. Total number of adverse events in the 3 months prior to MSC infusion and the number of adverse events 3 months after MSC infusion.
2. The ACR Pediatric 30 criteria should be met
3. Radiological (MRI) improvement of most active large joint.
4. Improvement in laboratory parameters or biomarkers.
Study objective
To find out if intravenous MSC is a safe treatment for children with therapy-resistant JIA
Study design
52 weeks after the (first) MSC injection the primary outcome will be measured and the third MRI will be made.
Also all data and materials for the secondary outcomes are collected within this time frame.
This will also be the end of the study for the individual.
Intervention
1 to maximal 3 iv MSC infusions.3 MRI scans, 4 extra visits to the hospital and 3 extra venapunctures when compared to standard treatment in the typical JIA patient
Intern mail address KC 03.063.0
PO Box 85090
Joost Swart
Utrecht 3500 AB
The Netherlands
+ 31 88 7554339
Intern mail address KC 03.063.0
PO Box 85090
Joost Swart
Utrecht 3500 AB
The Netherlands
+ 31 88 7554339
Inclusion criteria
Inclusion criteria: In order to be eligible to participate in this study, a subject must meet all of the following criteria:
Patients (4-18 years of age) diagnosed with juvenile idiopathic arthritis according to the ILAR-criteria with active arthritis resistant to intra-articular steroids and systemic use of methotrexate and for whom no on-label indication exists for (not yet used) biologicals.
The patient is followed for adverse events via the
Pharmachild database.
Informed consent signed.
Exclusion criteria
Concurrent use of biological response modifiers.
Concurrent infection, febrile illness or malignancy.
Pregnancy.
No signed informed consent.
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| NTR-new | NL3923 |
| NTR-old | NTR4146 |
| Other | 2012-002067-10 : EUDRACT |
| ISRCTN | ISRCTN wordt niet meer aangevraagd. |