Clinical trial NCT00824512: Efficacy of EGb761 in Patients Suffering From Friedreich Ataxia
Official title: Efficacy of EGb761 120mg Bid Versus Placebo in Patients Suffering From Friedreich Ataxia. A 3 Month, Phase II, Randomised, Double Blind, Placebo Controlled, Parallel Group Clinical Study.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Friedreich Ataxia
Lead sponsor: Ipsen
Sponsor class: INDUSTRY
First posted: 2009-01-16
Enrollment: 22
Sex: ALL
Minimum age (years): 12
Maximum age (years): 22
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: SUPPORTIVE_CARE

Brief summary: The purpose of this protocol is to determine the efficacy of EGb 761 120 mg bid versus placebo in patients suffering from Friedreich Ataxia


Eligibility criteria: Inclusion Criteria:

* Friedreich ataxia diagnosis confirmed by evidenced mutation expansion of Frataxin gene
* Ambulatory patient, with depressed tendon reflexes and pyramidal syndrome associated or not to a loss of position or vibration senses or dysarthria
* Patient able to perform the tests of the study

Exclusion Criteria:

* Severe cardiac disease as assessed by echocardiography performed at least within 6 months before screening or during the wash out period (4 weeks)
* Absolute contra-indication to Nuclear Magnetic Resonance spectroscopy(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
* Patient who did not deplete at baseline phosphocreatine (PCr) pool by more than 30 % during the exercise bout
* 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
* tranquilizer such as benzodiazepine, meprobamate or buspirone, and/or antidepressant (only one), at non stable dose

Primary outcomes:
measure: Creatine Rephosphorylation Rate Post Exercise
timeFrame: Baseline (Week 0) to Week 12
Secondary outcomes:
measure: Peak Post Exercise Perfusion
timeFrame: Baseline (Week 0) to Week 12
measure: Time to Peak Perfusion
timeFrame: Baseline (Week 0) to Week 12
measure: Perfusion-time Integral During the First 9 Minutes Post Exercise.
timeFrame: Baseline (Week 0) to Week 12
measure: Muscle Reoxygenation Rate Post Exercise.
timeFrame: Baseline (Week 0) to Week 12
measure: Muscle Trophicity: Maximum Cross Section of Muscle
timeFrame: Baseline (Week 0) to Week 12
measure: Developed Force During the Exercise Bout
timeFrame: Baseline (Week 0) to Week 12
measure: Normalised Work Developed During the Exercise
timeFrame: Baseline (Week 0) to Week 12
measure: Metabolism Efficacy Index
timeFrame: Baseline (Week 0) to Week 12
measure: International Cooperative Ataxia Rating Scale [ICARS] (Total Score)
timeFrame: Baseline (Week 0) to Week 12
measure: ICARS (Posture and Gait Disturbance Score)
timeFrame: Baseline (Week 0) to Week 12
measure: ICARS (Kinetic Function Score)
timeFrame: Baseline (Week 0) to Week 12
measure: ICARS (Speech Disorders Score)
timeFrame: Baseline (Week 0) to Week 12
measure: ICARS (Oculomotor Disorders Score)
timeFrame: Baseline (Week 0) to Week 12
measure: Timed 25-foot Walk Test
timeFrame: Baseline (Week 0) to Week 12
measure: Nine Hole Peg Test (Dominant Hand)
timeFrame: Baseline (Week 0) to Week 12
measure: Nine Hole Peg Test (Nondominant Hand)
timeFrame: Baseline (Week 0) to Week 12
measure: Choice Reaction Time Test- Reaction Time
timeFrame: Baseline (Week 0) to Week 12
measure: Choice Reaction Time Test- Movement Time
timeFrame: Baseline (Week 0) to Week 12
measure: Visual Assessment Scale (VAS) of Global Impression - Patient
timeFrame: Baseline (Week 0) to Week 12
measure: Visual Assessment Scale (VAS) of Global Impression - Parents
timeFrame: Baseline (Week 0) to Week 12
measure: Visual Assessment Scale (VAS) of Global Impression - Investigator
timeFrame: Baseline (Week 0) to Week 12
Other outcomes:
Locations:
city: Paris
country: France
facility: Hospital Necker Enfants Malades
Study officials:
affiliation: Ipsen
name: Ipsen Medical Director
role: STUDY_DIRECTOR
References: