The purpose of this protocol is to determine the efficacy of EGb 761 120 mg bid versus placebo in patients suffering from Friedreich Ataxia
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
SUPPORTIVE_CARE
Masking
QUADRUPLE
Enrollment
22
EGb 761® 120 mg bid, orally for 12 to 14 weeks
Placebo 1 tablet BID, orally for 12 to 14 weeks
Hospital Necker Enfants Malades
Paris, France
Creatine Rephosphorylation Rate Post Exercise
Creatine Rephosphorylation Rate post exercise measured using Phosphorus 31 Nuclear Magnetic Resonance (P-31 NMR)spectroscopy and calculated with correction according to muscular pH.
Time frame: Baseline (Week 0) to Week 12
Peak Post Exercise Perfusion
Peak post exercise perfusion (mL/mn/100 g of tissue) was assessed using Arterial spin labelling combined with Nuclear Magnetic Resonance imaging.
Time frame: Baseline (Week 0) to Week 12
Time to Peak Perfusion
Time frame: Baseline (Week 0) to Week 12
Perfusion-time Integral During the First 9 Minutes Post Exercise.
The integral of 'peak perfusion' over a period of 9 minutes post exercise.
Time frame: Baseline (Week 0) to Week 12
Muscle Reoxygenation Rate Post Exercise.
Muscle reoxygenation rate post exercise was assessed using Myoglobin Hydrogen-1 Nuclear Magnetic Resonance spectroscopy.
Time frame: Baseline (Week 0) to Week 12
Muscle Trophicity: Maximum Cross Section of Muscle
Muscle trophicity measured using Phosphorus 31 Nuclear Magnetic Resonance (P-31 NMR)spectroscopy and calculated based on maximum cross section of muscle (cm\^2)
Time frame: Baseline (Week 0) to Week 12
Developed Force During the Exercise Bout
Developed force during the exercise bout measured using Phosphorus 31 Nuclear Magnetic Resonance (P-31 NMR)spectroscopy
Time frame: Baseline (Week 0) to Week 12
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Normalised Work Developed During the Exercise
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.
Time frame: Baseline (Week 0) to Week 12
Metabolism Efficacy Index
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.
Time frame: Baseline (Week 0) to Week 12
International Cooperative Ataxia Rating Scale [ICARS] (Total Score)
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.
Time frame: Baseline (Week 0) to Week 12
ICARS (Posture and Gait Disturbance Score)
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).
Time frame: Baseline (Week 0) to Week 12
ICARS (Kinetic Function Score)
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).
Time frame: Baseline (Week 0) to Week 12
ICARS (Speech Disorders Score)
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).
Time frame: Baseline (Week 0) to Week 12
ICARS (Oculomotor Disorders Score)
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).
Time frame: Baseline (Week 0) to Week 12
Timed 25-foot Walk Test
Time frame: Baseline (Week 0) to Week 12
Nine Hole Peg Test (Dominant Hand)
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.
Time frame: Baseline (Week 0) to Week 12
Nine Hole Peg Test (Nondominant Hand)
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.
Time frame: Baseline (Week 0) to Week 12
Choice Reaction Time Test- Reaction Time
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.
Time frame: Baseline (Week 0) to Week 12
Choice Reaction Time Test- Movement Time
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.
Time frame: Baseline (Week 0) to Week 12
Visual Assessment Scale (VAS) of Global Impression - Patient
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.
Time frame: Baseline (Week 0) to Week 12
Visual Assessment Scale (VAS) of Global Impression - Parents
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.
Time frame: Baseline (Week 0) to Week 12
Visual Assessment Scale (VAS) of Global Impression - Investigator
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.
Time frame: Baseline (Week 0) to Week 12