E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Turner Syndrome is a common chromosomal abnormalities, characterized by one X-chromosome or a partial deletion in all or some cell-lines. The syndrome is known for features as short stature and absent or insufficient puberty with subsequent infertility, but it may also imply symptoms from other organs f.ex. heart. Recent year’s research has documented a significantly raised morbidity as well as mortality among girls and adult women with Turner Syndrome. |
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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 |
-examine if a larger dosage of oestrogen than the one used to day will secure the development of a normal size uterus and thereby reduce the risk of complications in case of a future pregnancy and increase the bone mineralization with stronger bones as the result. |
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E.2.2 | Secondary objectives of the trial |
- evaluate if aortic dilatation, a potential forerunner of later aortic dissection, is present in girls and young women with Turner Syndrome. - examine if oestrogen treatment will influence the emotional well-being and self-esteem in a positive manner in girls and young women with Turner Syndrome.
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E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
Criteria of inclusion to the sectional study (part A): - age between 10 to 21 years - diagnosed and verified (caryotype) Turner Syndrome
Criteria’s of inclusion to the longitudinal study (part B): - age between 15 to 21 years - diagnosed and verified (caryotype) Turner Syndrome
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E.4 | Principal exclusion criteria |
Criteria of exclusion to the sectional study (part A): - contradictions to the MR-scan - other severe or chronic sickness with a known or assumed impact on the parameters in the study (f.ex. epilepsy) evaluated by doctor Line Cleemann
Criteria’s of exclusion to the longitudinal study (part B): - contradictions to the MR-scan - contradictions to the trial medication: a) known, earlier or clinical suspicion of breast cancer b) known or clinical suspicion of oestrogen dependant tumors (ex. endometrial cancer) c) undiagnosed bleeding from the vagina d) untreated endometrial hyperplasia e) actual or earlier venous tromboembolisme (deep venous thrombosis, thrombosis of the lungs) f) actual or earlier arterial tromboembolic disease g) acute or earlier liver disease, where the liver parameters have not yet normalized h) known hypersensitivity to the additives in the trial medication i) porphyry - conditions, other severe or chronic sickness with known or assumed impact on the parameters in the study or implying an unacceptable risk of relapse or worsening in receiving the trial medication (ex. Leiomyom or endometriosis a family history of or physical risk of tromboembolisme, risks of oestrogen dependant tumors, liver diseases as adenomas, diabetes, gall-stone-disease, migraine or severe headache, SLE, epilepsy, asthma, cardinal or renal dysfunction, familiar hyper triglycerides, earlier endometriosis hyperplasia) evaluated by doctor Line Cleemann - necessary intake of medications which will affect the effects of the trial medication (ex. certain anti-epileptic medicine, antibiotics, medicine containing pericum or imipramin) and where participation in the study will cause a too big risk for the patient and/or affect the test results, evaluated by doctor Line Cleemann
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E.5 End points |
E.5.1 | Primary end point(s) |
The size of the uterus evaluated by MR-scan The bones evaluated by DEXA-scan
Secondary: - The diameter of the aortic root evaluated by MR-scan - Development of biochemical markers - Body composition evaluated by DEXA-scan - Emotional well-being and self-esteem evaluated by questionnaires designed for the patients and their parents - The size of the uterus evaluated by ultrasound
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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 | Yes |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | No |
E.6.5 | Efficacy | No |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
E.6.10 | Pharmacogenetic | Information not present in EudraCT |
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 | 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) | Yes |
E.8.2.2 | Placebo | Yes |
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 | Information not present in EudraCT |
E.8.7 | Trial has a data monitoring committee | Information not present in EudraCT |
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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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 | 5 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | |