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Efficacy of EGb761 in Patients Suffering From Friedreich Ataxia

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Ipsen
ClinicalTrials.gov Identifier:
NCT00824512
First received: January 15, 2009
Last updated: November 29, 2011
Last verified: November 2011
History of Changes
  Purpose

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


Condition Intervention Phase
Friedreich Ataxia
 Drug: EGb 761 120 mg
Drug: Placebo
 Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Supportive Care
Official Title: A Phase II, Randomised, Double Blind Study Assessing the Efficacy of EGb761 120mg Bid Versus Placebo in Patients Suffering From Friedreich Ataxia

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Ipsen:

Primary Outcome Measures:
  • Creatine rephosphorylation rate (sec-1) post-exercise using P-31 NMR spectroscopy, calculated with correction according to muscular pH. [ Time Frame: Assessed at the baseline (W0) and W12. ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Post-exercise skeletal muscle perfusion (ml/min/100 g of tissue) [ Time Frame: Assessed at the baseline (W0) and W12 ] [ Designated as safety issue: No ]
  • Peak post exercise perfusion (ml/mn/100 g of tissue) [ Time Frame: Assessed at the baseline (W0) and W12 ] [ Designated as safety issue: No ]
  • Adverse events [ Time Frame: Assessed at the baseline (W0) and W12 ] [ Designated as safety issue: Yes ]

Enrollment: 22
Study Start Date: June 2008
Study Completion Date: October 2011
Primary Completion Date: October 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: EGb 761® 120 mg Drug: EGb 761 120 mg
EGb 761® 120 mg bid, orally for 12 to 14 weeks
Placebo Comparator: Placebo Drug: Placebo
Placebo 1 tablet BID, orally for 12 to 14 weeks

  Eligibility

Ages Eligible for Study:   12 Years to 22 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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 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 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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00824512

Locations
France
Hospital Necker Enfants Malades
Paris, France, 75015
Sponsors and Collaborators
Ipsen
Investigators
Study Director: Cécile Merdrignac, MD Ipsen
  More Information

No publications provided

Responsible Party: Ipsen
ClinicalTrials.gov Identifier: NCT00824512     History of Changes
Other Study ID Numbers: 2-39-00240-133, 2007-005371-34
Study First Received: January 15, 2009
Last Updated: November 29, 2011
Health Authority: France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)

Additional relevant MeSH terms:
Ataxia
Friedreich Ataxia
Stress, Psychological
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Spinocerebellar Degenerations
Cerebellar Diseases
 Brain Diseases
Central Nervous System Diseases
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases
Behavioral Symptoms

ClinicalTrials.gov processed this record on May 16, 2013