E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Active and moderate to severe Graves’ Orbitopathy |
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MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | Information not present in EudraCT |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The primary objective of the study is to investigate whether add-on treatment of ATG-Fresenius S to standard steroid therapy has a beneficial effect on response to therapy defined as Clinical Activity Score (CAS) and Clinical Severity Score (CSS). |
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E.2.2 | Secondary objectives of the trial |
The secondary objectives of the study are the assessment of the safety and further efficacy parameters in terms of T-cell subpopulations, cytokines, specific and unspecific autoantibodies, thyroid-related hormones, thyroid sonography, glycosaminoglycans, quality of life and requirement of additional treatment for GO. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Patients will be enrolled in this study only if they meet all of the following criteria: • Signed and dated informed consent form. • Active and moderate to severe GO. • Age: 18 – 70 years. • Patients able to comply with all study related requirements. • Patients able to receive oral medication. • Women of childbearing age with a safe contraceptive method throughout the study.
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E.4 | Principal exclusion criteria |
Patients will not be enrolled in this study if they meet any of the following criteria: • Women who are pregnant or breast feeding, or with positive urine pregnancy test • Known Human Immunodeficiency Virus (HIV) infection. • Unable or unwilling to comply fully with the protocol. • Confirmed optic neuropathy with major deterioration of visual acuity. • Patients with anamnestically known hypersensitivity to rabbit immunoglobulin antibodies. • Patients with major organ dysfunctions (> threefold of normal values). • Serious psychiatric or psychological disorders. • Severe actual viral, bacterial or fungal infection not adequately controlled. • Thrombocytopenia: < 80,000 thrombocytes/µl. • Leukopenia: < 4,000 leukocytes/µl. • Concurrent or previous malignancy. • Participation in another study of an investigational medicinal product concurrently or within the last 30 days.
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary endpoint is the response to therapy at Week 12 vs. Week 0 after commencement of therapy. Response to treatment is defined as clinically relevant improvement of major objective ophthalmic evaluation parameters in at least one eye, which are: • Decrease in Clinical Activity Score (CAS) by >= 2 points at Week 12 compared to Week 0. • Decrease in Clinical Severity Score (CSS) by >= 2 points at Week 12 compared to Week 0. |
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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 | Yes |
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 | Information not present in EudraCT |
E.7.1.2 | Bioequivalence study | Information not present in EudraCT |
E.7.1.3 | Other | Information not present in EudraCT |
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 | Yes |
E.8.1.1 | Randomised | Yes |
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 | Yes |
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 |
standard corticosteroid therapy |
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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 | Information not present in EudraCT |
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 | 1 |
E.8.9.1 | In the Member State concerned months | 9 |
E.8.9.1 | In the Member State concerned days | |