|- candidate number||15358|
|- NTR Number||NTR4146|
|- ISRCTN||ISRCTN wordt niet meer aangevraagd.|
|- Date ISRCTN created|
|- date ISRCTN requested|
|- Date Registered NTR||4-sep-2013|
|- Secondary IDs||EUDRACT 2012-002067-10|
|- Public Title||Mesenchymal stromal cells for treatment of drug resistant
pediatric juvenile idiopathic arthritis|
|- Scientific Title||Mesenchymal stromal cells for treatment of drug resistant
pediatric juvenile idiopathic arthritis|
|- hypothesis||To find out if intravenous MSC is a safe treatment for children with therapy-resistant JIA|
|- Healt Condition(s) or Problem(s) studied||Juvenile idiopathic arthritis (JIA)|
|- 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
Informed consent signed.
|- Exclusion criteria||Concurrent use of biological response modifiers.|
Concurrent infection, febrile illness or malignancy.
No signed informed consent.
|- mec approval received||yes|
|- multicenter trial||no|
|- Type||Single arm|
|- planned startdate ||1-aug-2014|
|- planned closingdate||1-nov-2017|
|- Target number of participants||6|
|- Interventions||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|
|- 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.
|- Timepoints||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.
|- Trial web site||http://www.zonmw.nl/nl/projecten/project-detail/|
|- status||open: patient inclusion|
|- CONTACT FOR PUBLIC QUERIES||MD Joost Swart|
|- CONTACT for SCIENTIFIC QUERIES||MD Joost Swart|
|- Sponsor/Initiator ||University Medical Center Utrecht (UMCU)|
(Source(s) of Monetary or Material Support)
|ZON-MW, The Netherlands Organization for Health Research and Development|
|- Brief 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.|
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.
|- Main changes (audit trail)|
|- RECORD||4-sep-2013 - 16-feb-2015|