A Study of Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia (FRDA) Patients

NCT00905268 | Completed Phase 3 Results DMC

• 1 other identifier: SNT-III-001
• Study Type: interventional
• Target: 232
• Locations: 6 countries
• Sites: 13

Brief Summary

The purpose of this trial is to study the efficacy, safety and tolerability of idebenone in 12 months of treatment in children and adults with Friedreich's Ataxia. This is a randomised placebo-controlled double-blind trial conducted in Europe. Efficacy outcomes include measures of neurological impairment and function, and measures of the heart.

Conditions

Friedreich's Ataxia

Keywords

FRDA; idebenone; FRDA disease

Outcome Measures

Primary Outcomes (1)

• Absolute Change in International Cooperative Ataxia Rating Scale (ICARS) Scores From Baseline Assessment to Week 52

  The International Cooperative Ataxia Rating Scale (ICARS) is a commonly used evaluation and is composed of four clinical sub-scores involving the following: posture and gait, limb coordination, speech and oculomotor function.The ICARS score is the total sum of the sub scores and ranges from 0 to 100, with 100 indicative of the most severely affected outcome.

  Baseline and week 52

Secondary Outcomes (5)

• Absolute Change in Friedreich's Ataxia Rating Scale (FARS) Scores From Baseline Assessment to Week 52

  Baseline and week 52

• Proportion of Patients Improving (Responding) on ICARS by a Clinically Relevant Margin

  week 52

• Proportion of Patients Improving on Left Ventricular Peak Systolic Strain Rate or Showing a Reduction in Left Ventricular Mass Index (LVMI) With no Worsening in Strain Rate

  1 year

• Change in Peak Systolic Strain Rate From Baseline to Week 52

  1 year

• Change in Peak Workload From Baseline to Week 52

  1 year

Study Arms (4)

Group A: Idebenone

EXPERIMENTAL

Patients under/equal 45 kg: idebenone 180 mg/day Patients over 45 kg: idebenone 360 mg/day

Drug: idebenone

Group B: Idebenone

EXPERIMENTAL

Patients under/equal 45 kg: idebenone 450 mg/day Patients over 45 kg: idebenone 900 mg/day

Drug: idebenone

C: Idebenone

EXPERIMENTAL

Patients under/equal 45 kg: idebenone 1350 mg/day Patients over 45 kg: idebenone 2250 mg/day

Drug: idebenone

D: Placebo

PLACEBO COMPARATOR

placebo

Drug: Placebo

Interventions

idebenone DRUG

12 months of 1 of 3 treatments arms of oral idebenone or placebo.Treatment taken 3 times daily with meals.

C: Idebenone; Group A: Idebenone; Group B: Idebenone

Placebo DRUG

12 months of 1 of 3 treatments arms of oral idebenone or placebo.Treatment taken 3 times daily with meals.

D: Placebo

Eligibility Criteria

• Age: 8 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Documented diagnosis of FRDA with confirmed FRDA mutations
• Patients 8 years of age or older at baseline
• Patients with body weight ≥ 25kg
• Patients who in the opinion of the investigator are able to comply with the requirements of the study, including swallowing the medication
• Negative urine pregnancy test at screening and at baseline (women of childbearing potential)

You may not qualify if:

• Treatment with idebenone or Coenzyme Q10 within the past 1 month
• Pregnancy and/or breast-feeding
• Clinically significant abnormalities of clinical haematology or biochemistry including, but not limited to, elevations greater than 1.5 times the upper limit of normal of SGOT, SGPT, or creatinine
• Past or present history of abuse of drugs or alcohol

Sponsors & Collaborators

• Santhera Pharmaceuticals: lead

Study Sites (13)

Universitätsklinik Innsbruck

Innsbruck, 6020, Austria

Location

Hôpital Erasme - Université Libre de Bruxelles

Brussels, 1070, Belgium

Location

Hôpital de la Salpêtrière - INSERM U679, Neurologie et Thérapeutique expérimentale

Paris, 75651, France

Location

HELIOS Klinikum BerlinBuch

Berlin, 13125, Germany

Location

Neurologische Universitätsklinik und Poliklinik- Universitätsklinikum Bonn

Bonn, 53105, Germany

Location

Klinik II, Neuropädiatrie u.Muskelerkrankungen- Universitätsklinik Freiburg

Freiburg im Breisgau, 79106, Germany

Location

Zentrum für Neurologische Medizin

Göttingen, 37073, Germany

Location

UKE Hamburg Neuropädiatrie-Zentum für Frauen, Kinder und Jugendmedizin

Hamburg, 20246, Germany

Location

Neurologische Klinik- klinikum Grosshadern

München, 81377, Germany

Location

Neurologische Universitätsklinik und Poliklinik

Tübingen, 72076, Germany

Location

University Medical Center Groningen

Groningen, 9700 RB, Netherlands

Location

National Hospital for Neurology & Neurosurgery

London, WC1N 3BG, United Kingdom

Location

University of Newcastle upon Tyne -Mitochondrial Research Group

Newcastle, NE2 4HH, United Kingdom

Location

MeSH Terms

Conditions

Friedreich Ataxia

Interventions

idebenone

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: Prof. Nicholas William Wood
• Organization: The National Hospital, University College London

n.wood@ucl.ac.uk

020 7837 3611

Study Officials

• Nick Wood, Professor

  Dept of Molecular Neuroscience, Institute of Neurology. The National Hospital, University college London.

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 19, 2009
• First Posted: May 20, 2009
• Study Start: April 1, 2006
• Primary Completion: January 1, 2010
• Study Completion: January 1, 2010
• Last Updated: June 27, 2016
• Results First Posted: June 27, 2016

Record last verified: 2016-05

Locations

DE (7); AU (1); FR (1); NL (1); GB (2); BE (1)