Efficacy of EGb761 in Patients Suffering From Friedreich Ataxia

NCT00824512 | Completed Phase 2 Results

• 2 other identifiers: 2-39-00240-1332007-005371-34
• Study Type: interventional
• Target: 22
• Locations: 1 country
• Sites: 1

Brief 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

Conditions

Friedreich Ataxia

Outcome Measures

Primary Outcomes (1)

• 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.

  Baseline (Week 0) to Week 12

Secondary Outcomes (21)

• Peak Post Exercise Perfusion

  Baseline (Week 0) to Week 12

• Time to Peak Perfusion

  Baseline (Week 0) to Week 12

• Perfusion-time Integral During the First 9 Minutes Post Exercise.

  Baseline (Week 0) to Week 12

• Muscle Reoxygenation Rate Post Exercise.

  Baseline (Week 0) to Week 12

• Muscle Trophicity: Maximum Cross Section of Muscle

  Baseline (Week 0) to Week 12

Study Arms (2)

EGb 761® 120 mg

EXPERIMENTAL

Drug: EGb 761 120 mg

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

EGb 761 120 mg DRUG

EGb 761® 120 mg bid, orally for 12 to 14 weeks

EGb 761® 120 mg

Placebo DRUG

Placebo 1 tablet BID, orally for 12 to 14 weeks

Placebo

Eligibility Criteria

• Age: 12 Years - 22 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Ipsen: lead

Study Sites (1)

Hospital Necker Enfants Malades

Paris, 75015, France

Location

MeSH Terms

Conditions

Friedreich Ataxia

Interventions

Ginkgo biloba extract

Condition Hierarchy (Ancestors)

Spinocerebellar Degenerations; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mitochondrial Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Medical Director, Primary Care
• Organization: Ipsen

clinical.trials@ipsen.com

clinical.trials@ipsen.com

Study Officials

• Ipsen Medical Director

  Ipsen

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: SUPPORTIVE CARE
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 15, 2009
• First Posted: January 16, 2009
• Study Start: June 1, 2008
• Primary Completion: October 1, 2011
• Study Completion: October 1, 2011
• Last Updated: April 7, 2020
• Results First Posted: May 21, 2013

Record last verified: 2020-03

Locations

FR (1)