EFFICACY OF EGb 761® 120 mg bid VERSUS PLACEBO IN PATIENTS SUFFERING FROM FRIEDREICH ATAXIAA 3 months, phase II, randomised, double blind, placebo-controlled, parallel groups, clinical study. - EGB 761 in FRIEDREICH ATAXIA

Phase 1

• Conditions: Out patient suffering from friedreich ataxia aged from 12 to 20 years

• Registration Number: EUCTR2007-005371-34-FR
• Lead Sponsor: BEAUFOUR IPSEN PHARMA

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2007-12-20
• Study Completion: 2011-10-20
• Last Update Posted: 4 April 2022

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 22

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
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To demonstrate an improvement in skeletal muscle energetics and particularly in mitochondrial oxidative phosphorylation.;Secondary Objective: To demonstrate in patients treated with EGb 761®:<br>OTHER MUSCLE IMPROVEMENTS<br>?in post exercise skeletal muscle perfusion.<br>?in muscle reoxygenation rate post-exercise.<br>?in muscle trophicity.<br>?in force developed during the exercise bout (in joules).<br>?in muscle cross section<br>CLINICAL IMPROVEMENTS in:<br>?General clinical symptoms of Friedreich ataxia<br>?Muscle performance.<br>?Cognitive improvement<br>?Global improvement<br>TOLERANCE<br>?To verify the tolerance of EGb 761® in patients suffering from Friedreich ataxia.<br>;Primary end point(s): Creatine rephosphorylation rate (sec-1) post-exercise using P-31 NMR spectroscopy assessed at the baseline (W0) and Week 12.