E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
Severe sarcoidosis; a lung disease |
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E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To determine whether serum concentration guided dosing of infliximab is not inferior to standard dosing based on bodyweight in patients with severe sarcoidosis in terms of FVC change from baseline at week 26. |
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E.2.2 | Secondary objectives of the trial |
To compare both treatment strategies in terms of FVC change from baseline at week 50, DLCO change from baseline at week 26 and 50.
To compare both treatment strategies in terms of X-Thorax, HRCT and PET activity change from baseline at week 26 and 50.
To compare both treatment strategies in terms of sIL2R, ACE concentration change from baseline at week 26 and 50.
To compare both treatment strategies in terms of changes in 6MWT, quality of life and fatigue at several time points.
To compare both treatment strategies in terms of safety e.g. development of infections and infusion reactions.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
patients diagnosed with sarcoidosis being treated with infliximab or with an indication for infliximab
capability of giving informed consent
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E.4 | Principal exclusion criteria |
• vaccination with live viral or bacterial vaccines within the previous 3 months, or with the last dose within the previous 3 months • active or untreated latent tuberculosis (by mantoux-Elispot/TBC-IGRA) • serious infections within the last 2 months • serious right ventricular heart failure or cor polmunale • Active hepatitis B history of allergic reactions to monocolonal antibodies or their fragments
oppotunistic infections with the last 6 months
HIV
transplantation
known malignancy
pregnancy or breastfeeding
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E.5 End points |
E.5.1 | Primary end point(s) |
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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) |
Symptoms, CRP-, ACE-, sIL2R-concentrations at week 0, 14, 26 and 50. Lung function i.e. FVC and DLCO at week 0, 26 and 50.
Imaging: X-Thorax, HRCT and PET scanning at week 0, 26 and 50.
Endpoints in terms of Quality of Life are measurements obtained with EuroQol 5D and SF36 questionnaires at week 0, 14, 26 and 50.
Endpoints in terms of fatigue are measurements obtained with Checklist Individual Strength (CIS) at week 0, 14, 26 and 50.
Safety endpoints such as infusion reactions, infections.
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Depends on endpoint; at several of the following timepoints i.e. 0, 14, 26 and 50 weeks |
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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 | Yes |
E.6.4 | Safety | No |
E.6.5 | Efficacy | Yes |
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) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
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 | Yes |
E.8.2.3.1 | Comparator description |
infliximab dosing based on bodyweight is compared to concentration guided dosing |
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E.8.2.4 | Number of treatment arms in the trial | 2 |
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 | No |
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
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 4 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |