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Efficacy of EGb761 in Patients Suffering From Friedreich Ataxia

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ClinicalTrials.gov Identifier: NCT00824512
Recruitment Status : Completed
First Posted : January 16, 2009
Results First Posted : May 21, 2013
Last Update Posted : May 21, 2013
Sponsor:
Information provided by (Responsible Party):

January 15, 2009
January 16, 2009
October 30, 2012
May 21, 2013
May 21, 2013
June 2008
October 2011   (Final data collection date for primary outcome measure)
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.
Creatine rephosphorylation rate (sec-1) post-exercise using P-31 NMR spectroscopy [ Time Frame: Assessed at the baseline (W0) and W12. ]
Complete list of historical versions of study NCT00824512 on ClinicalTrials.gov Archive Site
  • 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.
  • Time to Peak Perfusion [ 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 ]
    The integral of 'peak perfusion' over a period of 9 minutes post exercise.
  • 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.
  • 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)
  • 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
  • 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.

  • 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.
  • 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.
  • 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).
  • 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).
  • 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).
  • 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).
  • Timed 25-foot Walk Test [ Time Frame: Baseline (Week 0) to Week 12 ]
  • 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.
  • 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.
  • 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.
  • 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.
  • 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.
  • 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.
  • 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.
  • Post-exercise skeletal muscle perfusion (ml/min/100 g of tissue) [ Time Frame: Assessed at the baseline (W0) and W12 ]
  • Peak post exercise perfusion (ml/mn/100 g of tissue) [ Time Frame: Assessed at the baseline (W0) and W12 ]
  • Adverse events [ Time Frame: Assessed at the baseline (W0) and W12 ]
Not Provided
Not Provided
 
Efficacy of EGb761 in Patients Suffering From Friedreich Ataxia
A Phase II, Randomised, Double Blind Study Assessing the Efficacy of EGb761 120mg Bid Versus Placebo 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
Not Provided
Interventional
Phase 2
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Supportive Care
Friedreich Ataxia
  • Drug: EGb 761 120 mg
    EGb 761® 120 mg bid, orally for 12 to 14 weeks
  • Drug: Placebo
    Placebo 1 tablet BID, orally for 12 to 14 weeks
  • Experimental: EGb 761® 120 mg
    Intervention: Drug: EGb 761 120 mg
  • Placebo Comparator: Placebo
    Intervention: Drug: Placebo
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
22
October 2011
October 2011   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
12 Years to 22 Years   (Child, Adult)
No
Contact information is only displayed when the study is recruiting subjects
France
 
 
NCT00824512
2-39-00240-133
2007-005371-34 ( EudraCT Number )
No
Not Provided
Not Provided
Ipsen
Ipsen
Not Provided
Study Director: Cécile Merdrignac, MD Ipsen
Ipsen
April 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP