E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Juvenile Idiopathic Arthritis (JIA) |
juveniele idiopathische artritis (JIA) |
|
E.1.1.1 | Medical condition in easily understood language |
juvenile arthritis |
jeugdreuma |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
NAM, well known as a dietary supplement, has also been extensively studied in humans in a variety of diseases in both children and adults. However, the bioavailability of NAM in patients with JIA at the side of inflammation, and therefore it’s potential as a therapeutic agent, is yet unknown. The primary objective of this study is therefore to assess the penetration of orally ingested NAM in the synovial fluid.
In this phase II trial essential preliminary information will be gained on the peak NAM levels in the synovial fluid. |
NAM is algemeen bekend als voedingssupplement maar is daarnaast ook uitgebreid onderzocht als therapie in verschillende ziektebeelden in kinderen en volwassenen. Echter, de beschikbaarheid van NAM in patiënten met JIA op de plek van inflammatie en daarmee de potentiele rol van NAM in de behandeling van JIA is nog onbekend. Het hoofddoel van deze studie is dan ook de penetratie van oraal ingenomen NAM in het synoviaal vocht te onderzoeken.
In deze fase II trial zal essentiële informatie mbt tot de maximale NAM spiegels in het synoviaal vocht verkregen worden. |
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Patients with a diagnosis of oligo-articular or poly-articular JIA with active disease in 1 or multiple joints and an indication for intra-articular corticosteroid injection. - Age of 16 years or older and under treatment of the pediatric rheumatology department of the WKZ/UMC Utrecht. |
- Patiënten met de diagnose oligo-articulaire of poly-articulaire JIA met actieve ziekte in 1 of meerdere gewrichten en een indicatie voor intra-articulaire corticosteroïd injectie. - Leeftijd van 16 jaar of ouder en onder behandeling bij de afdeling kinderreumatologie van het WKZ/UMC Utrecht |
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E.4 | Principal exclusion criteria |
- No informed consent possible by patient - Inability to take oral medication - Participation in other interventional trials |
- Geen informed consent mogelijk - Onvermogen tot inname van orale medicatie - Deelname in andere interventie studies |
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E.5 End points |
E.5.1 | Primary end point(s) |
Penetration of nicotinamide in the synovial fluid. Adequate penetration is defined as a nicotinamide level in the synovial fluid of at least 10% of the peak plasma level. |
Penetratie van nicotinamide in het synoviaal vocht. Adequate penetratie is gedefinieerd als het bereiken van een nicotinamide spiegel in het synoviaal vocht die minimaal 10% is van de piekspiegel in plasma. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | No |
E.6.5 | Efficacy | No |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | Yes |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | Yes |
E.7.1.3.1 | Other trial type description |
First time in adults with JIA, extensively studied in adults with other diseases |
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E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
LVLS |
Laatste bezoek van laatste participant |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |