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 |
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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 |
Determining the GC-sparing effect of RTX by assessing the proportion of PMR patients with GC-free remission and GC cumulative dose after 20 weeks |
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E.2.2 | Secondary objectives of the trial |
Assessing the effect of 1* 1000 mg RTX on disease activity in PMR patients by determining the change of ESR and CRP, PMR-AS, inner core domain set as proposed by the OMERACT, SF-36, EQ5D-5L, HAQ-DI from baseline to 20 weeks;
Assessing biomarkers such as B-cells, BAFF, IL-6, T-cells and anti-ferritin antibodies and RTX antibodies;
Determining the frequency and types of GC-related adverse events during the study by using the GTI
Determining the frequency and types of GC- and RTX-related adverse events during the study
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
·PMR according to the ACR/EULAR 2012 PMR core classification criteria
·Signed written informed consent
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E.4 | Principal exclusion criteria |
·Not being able to speak, read or write Dutch
·PMR diagnosed >4 weeks before inclusion in the study
·Exposure to GC or other immunosuppressant treatments in the past 3 months
·Known concomitant GCA or other rheumatic diseases such as RA, spondylarthropathies, connective tissue diseases, drug-induced myopathies, active and untreated thyroid disorders, Parkinson disorder or fibromyalgia
·Previous hypersensitivity for prednisone, RTX or murine peptides
·Contra-indications to RTX such as active current infection, including hepatitis B or tuberculosis infection, state of severe immunodeficiency, severe heart failure (NYHA-class IV)
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E.5 End points |
E.5.1 | Primary end point(s) |
The proportion of PMR patients in GC-free remission. |
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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) |
-GC cumulative dose
-The change of ESR and CRP, PMR-AS, inner core domain set as proposed by the OMERACT, SF-36, EQ5D-5L, HAQ-DI from baseline to 20 weeks;
-Assessing biomarkers such as B-cells, BAFF, IL-6, T-cells and anti-ferritin antibodies and RTX antibodies;
-The frequency and types of GC-related adverse events during the study measured using the GTI;
-The frequency and types of GC- and RTX-related adverse events during the study.
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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 | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
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) | 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
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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 | 2 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |