Friedreich AtaxiaPhase 2NCT00824512
Efficacy of EGb761 in Patients Suffering From Friedreich Ataxia
A Phase 2 study testing EGb 761 120 mg in people with Friedreich Ataxia, run by Ipsen at 1 site in France.
Trial 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 Read more about this study
- Who is it for?
- Ages 12 to 22 years
- Conditions the study is for
- Friedreich Ataxia
- What is being tested?
- EGb 761 120 mg
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 22 participants
- Where
- 1 siteFrance
- Study dates
- Jun 2008to Oct 2011
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 22 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official 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.
The purpose of this protocol is to determine the efficacy of EGb 761 120 mg bid versus placebo in patients suffering from Friedreich Ataxia
- Conditions
- Friedreich Ataxia
- Being tested
- EGb 761 120 mg, Placebo
- Medical subject headings
- Friedreich Ataxia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EGb 761 120 mgDrug
EGb 761® 120 mg bid, orally for 12 to 14 weeks
PlaceboDrug
Placebo 1 tablet BID, orally for 12 to 14 weeks
| Study group | Role | What they receive |
|---|---|---|
| EGb 761® 120 mg | Experimental | Not described Receives: Drug: EGb 761 120 mg |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline (Week 0) to Week 12
Secondary outcomes (21)
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.
Measured: Baseline (Week 0) to Week 12
Time to Peak Perfusion
Measured: 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.
Measured: 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.
Measured: 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)
Measured: 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
Measured: Baseline (Week 0) to Week 12
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.
Measured: 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.
Measured: 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.
Measured: 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).
Measured: 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).
Measured: 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).
Measured: 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).
Measured: Baseline (Week 0) to Week 12
Timed 25-foot Walk Test
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Baseline (Week 0) to Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Jan 2009 (estimated)
- Study startJun 2008
- Primary completionOct 2011
- Study completionOct 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Friedreich AtaxiaStudies of the same condition, open and under way.
- More trials run by IpsenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 22 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ipsen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ipsen Medical Director, Study director, Ipsen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00824512
- Sponsor's protocol ID
- 2-39-00240-133
- Other IDs
- 2007-005371-34
- Status last verified
- Mar 2020
- First submitted
- 15 Jan 2009
- Last updated
- 7 Apr 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Apr 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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.
- ClinicalTrials.gov ID
- NCT00824512
- Acronym / sponsor ID
- 2-39-00240-133
- Conditions
- Friedreich Ataxia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 22 participants (actual)
- Lead sponsor
- IpsenIndustry (pharma, biotech, devices)
- Study start
- Jun 2008
- Primary completion
- Oct 2011 (actual)
- Study completion
- Oct 2011 (actual)
- First posted
- 16 Jan 2009 (estimated)
- Results
- Posted 21 May 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| EGb 761® 120 mg | Experimental | Drug: EGb 761 120 mg |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 12 to 22 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (7 listed)
- 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),
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EGb 761 120 mgEGb 761® 120 mg bid, orally for 12 to 14 weeks | Drug | EGb 761® 120 mg |
| PlaceboPlacebo 1 tablet BID, orally for 12 to 14 weeks | Drug | Placebo |
Primary outcome
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.
21 secondary outcomes
Peak Post Exercise Perfusion
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.
Time frame: Baseline (Week 0) to Week 12
Muscle Reoxygenation Rate Post Exercise.
Time frame: Baseline (Week 0) to Week 12
Muscle Trophicity: Maximum Cross Section of Muscle
Time frame: Baseline (Week 0) to Week 12
Developed Force During the Exercise Bout
Time frame: Baseline (Week 0) to Week 12
Normalised Work Developed During the Exercise
Time frame: Baseline (Week 0) to Week 12
Metabolism Efficacy Index
Time frame: Baseline (Week 0) to Week 12
International Cooperative Ataxia Rating Scale [ICARS] (Total Score)
Time frame: Baseline (Week 0) to Week 12
ICARS (Posture and Gait Disturbance Score)
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
ICARS (Oculomotor Disorders Score)
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)
Time frame: Baseline (Week 0) to Week 12
Nine Hole Peg Test (Nondominant Hand)
Time frame: Baseline (Week 0) to Week 12
Choice Reaction Time Test- Reaction Time
Time frame: Baseline (Week 0) to Week 12
Choice Reaction Time Test- Movement Time
Time frame: Baseline (Week 0) to Week 12
Visual Assessment Scale (VAS) of Global Impression - Patient
Time frame: Baseline (Week 0) to Week 12
Visual Assessment Scale (VAS) of Global Impression - Parents
Time frame: Baseline (Week 0) to Week 12
Visual Assessment Scale (VAS) of Global Impression - Investigator
Time frame: Baseline (Week 0) to Week 12
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Apr 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ipsen Medical Director study director
Ipsen
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing EGb 761 120 mg in people with Friedreich Ataxia, run by Ipsen at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 7 Apr 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 21 May 2013). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Creatine Rephosphorylation Rate Post Exercise (time frame: Baseline (Week 0) to Week 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Apr 2020.