E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Out patient suffering from friedreich ataxia aged from 12 to 20 years |
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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 |
To demonstrate an improvement in skeletal muscle energetics and particularly in mitochondrial oxidative phosphorylation. |
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E.2.2 | Secondary objectives of the trial |
To demonstrate in patients treated with EGb 761®: OTHER MUSCLE IMPROVEMENTS in post exercise skeletal muscle perfusion. in muscle reoxygenation rate post-exercise. in muscle trophicity. in force developed during the exercise bout (in joules). in muscle cross section CLINICAL IMPROVEMENTS in: General clinical symptoms of Friedreich ataxia Muscle performance. Cognitive improvement Global improvement TOLERANCE To verify the tolerance of EGb 761® in patients suffering from Friedreich ataxia.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1) Friedreich ataxia diagnosis confirmed by evidenced mutation expansion of frataxin gene, 2) Age from 12 to 20 years, 3) Ambulatory patient, with depressed tendon reflexes and pyramidal syndrome associated or not to a loss of position or vibration senses or dysarthria, 4) Patient able to perform the tests of the study, 5) Patient affiliated to a social security scheme, 6) Patient signed consent form or both parents’ signatures for children < 18 years |
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E.4 | Principal exclusion criteria |
Non inclusion Criteria Related to cardiac status: 1) Contra-indication to idebenone interruption as assessed by cardiac examination and echocardiography within 6 months or during the wash out period (4 weeks), 2) Severe cardiac disease as assessed by echocardiography performed at least within 6 months before screening or during the wash out period (4 weeks), Related to NMR examination: 3) Absolute contra-indication to NMR examination: iron and any magnetic objects implanted in the whole body, e.g. some neurostimulators, cardiac pace-makers, vascular clips and other implanted orthopaedic prosthesis, 4) Relative contraindications to MNR examination: amagnetic object implanted in the legs (which could induce artefacts), claustrophobia, pregnancy, 5) Patient who did not deplete at baseline PCr pool by more than 40 % during the exercise bout. Related to medication use: 6) Any continuous use of the following forbidden medications: - other antioxidant such as idebenone, coenzyme Q, vitamin E/C, taken for less than 4 weeks prior study treatment start (ie for antioxidant drugs a mandatory wash-out period of 4 weeks prior study drug start has to be observed), - any other vasodilators. 7) Tranquilizer such as benzodiazepine, meprobamate or buspirone, and/or antidepressant (only one), at non stable dose (These concomitant drug could be accepted if taken at stable dose ie the dose is the same since at least 8 weeks, without any changes planned during the study), 8) Any disease with potential impairment of drug absorption, 9) known hypersensitivity to any of the tested drug or related compounds, 10) Patient treated with any unlicensed drug within the previous 30 days, 11) Patients suffering from congenital galactosemia, glucose or galactose malabsorption syndrome, lactase deficiency (as EGB761® contains lactose). Other non inclusion criteria 12) Childbearing potential, young women without effective contraception, 13) Life expectancy < 1 year (whatever the cause), 14) Patient previously included in this study, 15) Patient unable or unwilling to comply fully with the protocol.
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E.5 End points |
E.5.1 | Primary end point(s) |
Creatine rephosphorylation rate (sec-1) post-exercise using P-31 NMR spectroscopy assessed at the baseline (W0) and Week 12. |
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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 | 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 | 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) | No |
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 | 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 | 0 |
E.8.9.1 | In the Member State concerned days | 0 |