Mesenchymal stromal cells for treatment of drug resistant pediatric juvenile idiopathic arthritis

Status

Recruiting

Phases

Unknown

Study type

Interventional

Source

NL-OMON

Registry ID

NL-OMON21350

Enrollment

Registered

2013-09-04

Start date

2014-08-01

Completion date

Unknown

Last updated

2024-02-28

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

juvenile idiopathic arthritis

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

Eligibility

Inclusion criteria

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

Exclusion criteria: Concurrent use of biological response modifiers. Concurrent infection, febrile illness or malignancy. Pregnancy. No signed informed consent.

Design outcomes

Primary

Measure	Time frame
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%.	—

Measure

Time frame

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

Measure	Time frame
- 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.	—

Measure

Time frame

- 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.

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Contacts

Public ContactJoost Swart

UMC Utrecht, Wilhelmina Children's hospital Intern mail address KC 03.063.0 PO Box 85090

+ 31 88 7554339

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)