To find out if intravenous MSC is a safe treatment for children with therapy-resistant JIA
ID
Bron
Verkorte titel
Aandoening
juvenile idiopathic arthritis
Ondersteuning
Onderzoeksproduct en/of interventie
Uitkomstmaten
Primaire uitkomstmaten
Total number of adverse events in the 3 months prior to MSC infusion and the number of adverse events 3 months after MSC infusion.<br>
To offer an effective alternative for that category of JIA patients that is therapy-resistant.<br>
For this effectiveness the ACR Pediatric 70 criteria should be achieved.<br>
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%.
Achtergrond van het onderzoek
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.
Doel van het onderzoek
To find out if intravenous MSC is a safe treatment for children with therapy-resistant JIA
Onderzoeksopzet
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.
Onderzoeksproduct en/of interventie
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
Publiek
Intern mail address KC 03.063.0
PO Box 85090
Joost Swart
Utrecht 3500 AB
The Netherlands
+ 31 88 7554339
Wetenschappelijk
Intern mail address KC 03.063.0
PO Box 85090
Joost Swart
Utrecht 3500 AB
The Netherlands
+ 31 88 7554339
Belangrijkste voorwaarden om deel te mogen nemen (Inclusiecriteria)
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.
Belangrijkste redenen om niet deel te kunnen nemen (Exclusiecriteria)
Concurrent use of biological response modifiers.
Concurrent infection, febrile illness or malignancy.
Pregnancy.
No signed informed consent.
Opzet
Deelname
Opgevolgd door onderstaande (mogelijk meer actuele) registratie
Geen registraties gevonden.
Andere (mogelijk minder actuele) registraties in dit register
Geen registraties gevonden.
In overige registers
| Register | ID |
|---|---|
| NTR-new | NL3923 |
| NTR-old | NTR4146 |
| Ander register | 2012-002067-10 : EUDRACT |
| ISRCTN | ISRCTN wordt niet meer aangevraagd. |