Phase 2
N=22
Efficacy of EGb761 in Patients Suffering From Friedreich Ataxia
Friedreich Ataxia
Bottom Line
View on ClinicalTrials.gov: NCT00824512 ↗Enrolled (actual)
22
Serious AEs
9.1%
Results posted
May 2013
Primary outcome: Primary: Creatine Rephosphorylation Rate Post Exercise — 0.024; 0.029; 0.022; 0.029 pH per second — p=0.9133
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- EGb 761 120 mg (Drug); Placebo (Drug)
- Age
- Pediatric, Adult · 12+ yrs
- Sex
- All
- Sponsor
- Ipsen
- Primary completion
- Oct 2011
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Creatine Rephosphorylation Rate Post Exercise |
0.024; 0.029; 0.022; 0.029; 0.001; 0.000 | 0.9133 |
| SECONDARY Peak Post Exercise Perfusion |
54.30; 51.80; 58.80; 46.60; 3.60; -1.55 | — |
| SECONDARY Time to Peak Perfusion |
38.30; 58.55; 39.80; 76.55; 8.50; 14.25 | — |
| SECONDARY Perfusion-time Integral During the First 9 Minutes Post Exercise. |
166.90; 157.20; 191.60; 185.70; -5.20; 0.05 | — |
| SECONDARY Muscle Reoxygenation Rate Post Exercise. |
0.0870; 0.0540; 0.0580; 0.0620; -0.0350; -0.0035 | — |
| SECONDARY Muscle Trophicity: Maximum Cross Section of Muscle |
28.10; 28.95; 26.8; 29.85; 0.10; 0.55 | — |
| SECONDARY Developed Force During the Exercise Bout |
305.30; 358.10; 277.50; 354.35; -32.40; 19.20 | — |
| SECONDARY Normalised Work Developed During the Exercise |
12.07; 12.00; 9.23; 11.53; -1.03; 0.47 | — |
| SECONDARY Metabolism Efficacy Index |
0.0180; 0.0150; 0.0130; 0.0145; 0.0010; -0.0010 | — |
| SECONDARY International Cooperative Ataxia Rating Scale [ICARS] (Total Score) |
35; 26.5; 33.0; 29.0; 0.0; 0.5 | — |
| SECONDARY ICARS (Posture and Gait Disturbance Score) |
19.0; 12.5; 18.0; 12.0; 1.0; 2.8 | — |
| SECONDARY ICARS (Kinetic Function Score) |
15.0; 11.5; 13.0; 13.0; 0.0; 0.5 | — |
| SECONDARY ICARS (Speech Disorders Score) |
2.0; 0.5; 1.0; 1.0; 0.0; 0.0 | — |
| SECONDARY ICARS (Oculomotor Disorders Score) |
1.0; 1.5; 2.0; 2.0; 0.0; 0.0 | — |
| SECONDARY Timed 25-foot Walk Test |
8.50; 6.75; 9.00; 6.75; 0.50; 0.00 | — |
| SECONDARY Nine Hole Peg Test (Dominant Hand) |
38.50; 43.50; 42.00; 40.75; 1.50; -1.50 | — |
| SECONDARY Nine Hole Peg Test (Nondominant Hand) |
47.50; 48.50; 53.00; 46.50; 0.50; 1.75 | — |
| SECONDARY Choice Reaction Time Test- Reaction Time |
513.5; 536.0; 491.0; 531.0; 8.5; 9.0 | — |
| SECONDARY Choice Reaction Time Test- Movement Time |
561.5; 531.0; 555.0; 496.5; 4.5; -31.0 | — |
| SECONDARY Visual Assessment Scale (VAS) of Global Impression - Patient |
60.0; 68.5; 67.0; 63.0; -2.0; -2.0 | — |
| SECONDARY Visual Assessment Scale (VAS) of Global Impression - Parents |
64.0; 62.0; 64.0; 57.0; -7.0; -10.0 | — |
| SECONDARY Visual Assessment Scale (VAS) of Global Impression - Investigator |
78.0; 76.0; 80.0; 74.0; -2.0; -1.0 | — |
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
Data sourced from ClinicalTrials.gov (NCT00824512). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.