E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
active severe endocrine orbitopathy |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10066617 |
E.1.2 | Term | Graves' ophthalmopathy worsened |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10014702 |
E.1.2 | Term | Endocrine exophthalmos |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10015684 |
E.1.2 | Term | Exophthalmos endocrine |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10043787 |
E.1.2 | Term | Thyrotoxic exophthalmos |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10057889 |
E.1.2 | Term | Graves' ophthalmopathy |
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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 |
Aim of the project is to find laboratory, clinical and ultrasonographic markers usable for the prediction of the response on intravenous GC administration in severe endocrine orbitopathy and to elucidate the differences in the treatment effect among individual patients. |
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E.2.2 | Secondary objectives of the trial |
To improve the efficiency of the treatment and diagnostics in patients with severe endocrine orbitopathy |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Pacients with active and moderate to severe or sight threatening endocrine orbitopathy (EO), age more than 18 years, laboratory positivity of autoantibodies against TSH receptor, patological monitored parameters for the ultrasonographical examination of the orbit. Activity is maesured on the classical features on inflamantion: clinical activity score (CAS). CAS more tahn 4 indicates active EO. Moderate to severe EO: Patients usualy have any one or more of the following: lid retraciton more than 2 mm, moderate or severe soft tissue involvement, exophthalmos more than 3 mm above normal for race and gender, inconstant, or constant diplopia. Sight threatening EO: Patients with dysthyroid optic neuropathy and/or corneal breakdown. Monitored parameters for the ultrasonographic examination of the orbit: thickness of the medial, lateral and inferior direct eye muscle and thickness of the upper muscle group - superior direct with eyelid levator - lateral and inferior, according to an arbitrary scale established by us the muscle echogenicity - I to IV, where I is a normal orbital muscle, i.e. deeply hypoechogeneic, IV is of the echogenicity as high that it is barely undistinguishable from surrounding connective tissue. Measurement of the flow rates especially in the arteria centralis retinae, artheries cilliares breves and in artheria ophthalmica.
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E.4 | Principal exclusion criteria |
hypersensitivity, TBC (patients with active disease and latent disease, including positive tuberculin test, which need careful monitoring for possibility of reactivation of disease and requirements of chemoprophylaxis), systemic mycosis, active peptic ulcer, fresh intestinal anastomoses, decompensated diabetes mellitus, severe heart insuficiency, severe uncorrected hypertension, psychosis (acute or in medical history), severe impairment of renal function, active hepatitis, gravidity, lactation, inability to sign the informed consent.
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E.5 End points |
E.5.1 | Primary end point(s) |
The characteristics of particular clinical, laboratory and ultrasonographic markers for prediction of response to iv glucocorticoid administration |
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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 | No |
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) | 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 | No |
E.8.1.1 | Randomised | Information not present in EudraCT |
E.8.1.2 | Open | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
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 | 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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undesirable effects of the pharmaceutical according to SPC of severe nature - significant changes of the subject health state, not in connection with the tested pharmaceutical - gravidity, lactation - subject non-compliance - subject request - in case of preterm cancellation of the study, collection of blood and clinical examination will be performed the same way as in the case of the last visit
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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 | 5 |
E.8.9.1 | In the Member State concerned months | 4 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |