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Completed

NCT Number: NCT00824512

Efficacy of EGb761 in Patients Suffering From Friedreich Ataxia

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

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Key information

Conditions

Friedreich Ataxia Brain Diseases Central Nervous System Diseases Cerebellar Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Metabolic Diseases Mitochondrial Diseases Nervous System Diseases Neurodegenerative Diseases Nutritional and Metabolic Diseases Spinal Cord Diseases Spinocerebellar Degenerations

Age range

12 year–22 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Hospital Necker Enfants Malades

Paris, 75015, France

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

EGb 761 120 mg

Drug

EGb 761® 120 mg bid, orally for 12 to 14 weeks

Placebo

Drug

Placebo 1 tablet BID, orally for 12 to 14 weeks

Primary outcomes

  1. Creatine Rephosphorylation Rate Post Exercise

    Time frame: Baseline (Week 0) to Week 12

    Creatine Rephosphorylation Rate post exercise measured using Phosphorus 31 Nuclear Magnetic Resonance (P-31 NMR)spectroscopy and calculated with correction according to muscular pH.

Secondary outcomes

  1. Peak Post Exercise Perfusion

    Time frame: Baseline (Week 0) to Week 12

    Peak post exercise perfusion (mL/mn/100 g of tissue) was assessed using Arterial spin labelling combined with Nuclear Magnetic Resonance imaging.

  2. Time to Peak Perfusion

    Time frame: Baseline (Week 0) to Week 12

  3. Perfusion-time Integral During the First 9 Minutes Post Exercise.

    Time frame: Baseline (Week 0) to Week 12

    The integral of 'peak perfusion' over a period of 9 minutes post exercise.

  4. Muscle Reoxygenation Rate Post Exercise.

    Time frame: Baseline (Week 0) to Week 12

    Muscle reoxygenation rate post exercise was assessed using Myoglobin Hydrogen-1 Nuclear Magnetic Resonance spectroscopy.

  5. Muscle Trophicity: Maximum Cross Section of Muscle

    Time frame: Baseline (Week 0) to Week 12

    Muscle trophicity measured using Phosphorus 31 Nuclear Magnetic Resonance (P-31 NMR)spectroscopy and calculated based on maximum cross section of muscle (cm^2)

  6. Developed Force During the Exercise Bout

    Time frame: Baseline (Week 0) to Week 12

    Developed force during the exercise bout measured using Phosphorus 31 Nuclear Magnetic Resonance (P-31 NMR)spectroscopy

  7. Normalised Work Developed During the Exercise

    Time frame: Baseline (Week 0) to Week 12

    Normalised work developed during the exercise was derived as Work developed during the exercise/([60 X Maximum cross section of muscle]-1100).

    Normalised work measured using Phosphorus 31 Nuclear Magnetic Resonance (P-31 NMR)spectroscopy.

  8. Metabolism Efficacy Index

    Time frame: Baseline (Week 0) to Week 12

    The metabolism efficacy index was derived as Normalised work x creatine phosphorylation rate (sec-1). [Normalised work was derived as Work developed during the exercise/(60 X Maximum cross section of muscle-1100)]. Greater values of Metabolism Efficacy index indicate improvement in skeletal muscle energetics while lower values indicate the reverse. Negative values obtained using the formula indicated severe levels of muscle weakness.

  9. International Cooperative Ataxia Rating Scale [ICARS] (Total Score)

    Time frame: Baseline (Week 0) to Week 12

    The ICARS was used to measure the general clinical symptoms of Friedreich ataxia using four subscales (i.e. Posture and gait disturbances, Kinetic functions, Speech disorders, & Oculomotor disorders). Scores for each subscale quantify the extent of ataxia in each clinically important area and subscale scores are also summed to give a total score ranging from 0 to 100, with 100 indicative of the most severely affected outcome.

  10. ICARS (Posture and Gait Disturbance Score)

    Time frame: Baseline (Week 0) to Week 12

    The ICARS was used to measure the general clinical symptoms of Friedreich ataxia using four subscales including Posture and gait disturbances. Posture and gait disturbances score range from 0 to 34 (Higher scores indicate higher levels of impairment).

  11. ICARS (Kinetic Function Score)

    Time frame: Baseline (Week 0) to Week 12

    The ICARS was used to measure the general clinical symptoms of Friedreich ataxia using four subscales including Kinetic Function. Kinetic Function score range from 0 to 52 (Higher scores indicate higher levels of impairment).

  12. ICARS (Speech Disorders Score)

    Time frame: Baseline (Week 0) to Week 12

    The ICARS was used to measure the general clinical symptoms of Friedreich ataxia using four subscales including Speech Disorders. Speech Disorders Score range from 0 to 8 (Higher scores indicate higher levels of impairment).

  13. ICARS (Oculomotor Disorders Score)

    Time frame: Baseline (Week 0) to Week 12

    The ICARS was used to measure the general clinical symptoms of Friedreich ataxia using four subscales including Oculomotor Disorders. Oculomotor Disorders score range from 0 to 6 (Higher scores indicate higher levels of impairment).

  14. Timed 25-foot Walk Test

    Time frame: Baseline (Week 0) to Week 12

  15. Nine Hole Peg Test (Dominant Hand)

    Time frame: Baseline (Week 0) to Week 12

    The nine hole peg test was used to assess cognitive function and in particular, fine motor coordination. The patient was asked to place nine pegs in nine holes and was scored on the amount of time it took to place and remove all nine pegs.

  16. Nine Hole Peg Test (Nondominant Hand)

    Time frame: Baseline (Week 0) to Week 12

    The nine hole peg test was used to assess cognitive function and in particular, fine motor coordination. The patient was asked to place nine pegs in nine holes and was scored on the amount of time it took to place and remove all nine pegs.

  17. Choice Reaction Time Test- Reaction Time

    Time frame: Baseline (Week 0) to Week 12

    The choice reaction time test was used to assess cognitive functioning. On random presentation of one of six signal lights, the patient was asked to respond as quickly and accurately as possible by removing their index finger of the dominant hand from the bottom key and pressing whichever of the top six keys was indicated by the signal. Reaction time was the time elapsed between the presentation of the stimulus and the release of the finger and movement time was defined as the time elapsed between release of the finger and pressure of the second key.

  18. Choice Reaction Time Test- Movement Time

    Time frame: Baseline (Week 0) to Week 12

    The choice reaction time test was used to assess cognitive functioning. On random presentation of one of six signal lights, the patient was asked to respond as quickly and accurately as possible by removing their index finger of the dominant hand from the bottom key and pressing whichever of the top six keys was indicated by the signal. Reaction time was the time elapsed between the presentation of the stimulus and the release of the finger and movement time was defined as the time elapsed between release of the finger and pressure of the second key.

  19. Visual Assessment Scale (VAS) of Global Impression - Patient

    Time frame: Baseline (Week 0) to Week 12

    The VAS used a 10-cm scoring scale in which values were reported in mm such that 0=bad and 100=good. Total score range on VAS is from 0 to 100.

  20. Visual Assessment Scale (VAS) of Global Impression - Parents

    Time frame: Baseline (Week 0) to Week 12

    The VAS used a 10-cm scoring scale in which values were reported in mm such that 0=bad and 100=good. Total score range on VAS is from 0 to 100.

  21. Visual Assessment Scale (VAS) of Global Impression - Investigator

    Time frame: Baseline (Week 0) to Week 12

    The VAS used a 10-cm scoring scale in which values were reported in mm such that 0=bad and 100=good. Total score range on VAS is from 0 to 100.

Sponsors and collaborators

Lead sponsor

Ipsen

Industry

Registry information

Official study 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.

Important dates

Study start
2008
Primary completion
2011
Study completion
2011
First posted
Jan 16, 2009
Registry last updated
Apr 7, 2020

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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