Efficacy of EGb761 in Patients Suffering From Friedreich Ataxia

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00824512
• Date of registration: 15/01/2009
• Prospective Registration: No
• Primary sponsor: Ipsen
• Public title: Efficacy of EGb761 in Patients Suffering From Friedreich Ataxia
• Scientific 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.
• Date of first enrolment: June 2008
• Target sample size: 22
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00824512
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Supportive Care. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

France

Contacts

• Name: Ipsen Medical Director
• Affiliation: Ipsen

Key inclusion & exclusion 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



Age minimum: 12 Years
Age maximum: 22 Years
Gender: All

Health Condition(s) or Problem(s) studied

Friedreich Ataxia

Intervention(s)

Drug: EGb 761 120 mg

Drug: Placebo

Primary Outcome(s)

Creatine Rephosphorylation Rate Post Exercise [Time Frame: Baseline (Week 0) to Week 12]

Secondary Outcome(s)

ICARS (Posture and Gait Disturbance Score) [Time Frame: Baseline (Week 0) to Week 12]

Choice Reaction Time Test- Reaction Time [Time Frame: Baseline (Week 0) to Week 12]

International Cooperative Ataxia Rating Scale [ICARS] (Total Score) [Time Frame: Baseline (Week 0) to Week 12]

Metabolism Efficacy Index [Time Frame: Baseline (Week 0) to Week 12]

Normalised Work Developed During the Exercise [Time Frame: Baseline (Week 0) to Week 12]

Muscle Trophicity: Maximum Cross Section of Muscle [Time Frame: Baseline (Week 0) to Week 12]

Nine Hole Peg Test (Dominant Hand) [Time Frame: Baseline (Week 0) to Week 12]

Peak Post Exercise Perfusion [Time Frame: Baseline (Week 0) to Week 12]

Visual Assessment Scale (VAS) of Global Impression - Parents [Time Frame: Baseline (Week 0) to Week 12]

ICARS (Kinetic Function Score) [Time Frame: Baseline (Week 0) to Week 12]

ICARS (Speech Disorders Score) [Time Frame: Baseline (Week 0) to Week 12]

Perfusion-time Integral During the First 9 Minutes Post Exercise. [Time Frame: Baseline (Week 0) to Week 12]

Time to Peak Perfusion [Time Frame: Baseline (Week 0) to Week 12]

Timed 25-foot Walk Test [Time Frame: Baseline (Week 0) to Week 12]

Visual Assessment Scale (VAS) of Global Impression - Patient [Time Frame: Baseline (Week 0) to Week 12]

Choice Reaction Time Test- Movement Time [Time Frame: Baseline (Week 0) to Week 12]

ICARS (Oculomotor Disorders Score) [Time Frame: Baseline (Week 0) to Week 12]

Developed Force During the Exercise Bout [Time Frame: Baseline (Week 0) to Week 12]

Muscle Reoxygenation Rate Post Exercise. [Time Frame: Baseline (Week 0) to Week 12]

Nine Hole Peg Test (Nondominant Hand) [Time Frame: Baseline (Week 0) to Week 12]

Visual Assessment Scale (VAS) of Global Impression - Investigator [Time Frame: Baseline (Week 0) to Week 12]

Secondary ID(s)

2007-005371-34

2-39-00240-133

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 21/05/2013
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00824512