Long-Term Safety and Tolerability of Idebenone in Friedreich's Ataxia Patients (MICONOS Extension)

NCT00993967 | Completed Phase 3 Results DMC

• 1 other identifier: SNT-III-001-E
• Study Type: interventional
• Target: 200
• Locations: 5 countries
• Sites: 11

Brief Summary

This is an Extension study of the MICONOS main randomised placebo-controlled trial (NCT00905268), and open to those patients completing the main study. The scientific aim of this extension study is to monitor safety and tolerability of idebenone over two years in patients with Friedreich's Ataxia.

Conditions

Freidreich's Ataxia

Keywords

FRDA; idebenone; FRDA disease

Outcome Measures

Primary Outcomes (2)

• Measures of Safety and Tolerability: Nature and Frequency of Adverse Events (AEs)

  Global Overvbiew of accurance of AEs-Safety population. The Safety population included all subjects who received at least one dose of the study medication.

  overall study, up to 24 months

• Absolute Change in The International Cooperative Ataxia Rating Scale (ICARS)

  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, Month 12 and month 24

Other Outcomes (3)

• Measures of Safety and Tolerability: Physical Examinations and Vital Signs

  Month 1, 3, 6, 12, 18 and 24

• Measures of Safety and Tolerability: Electrocardiograms (ECGs)

  Month 1, 3, 6, 12, 18 and 24

• Measures of Safety and Tolerability: Haematological and Biochemical Laboratory Parameters

  Month 1, 3, 6, 12, 18 and 24

Study Arms (1)

Idebenone

EXPERIMENTAL

1350 mg/day or 2250 mg/day for patients weighing ≤45 kg or \>45 kg, respectively.In case of poor tolerability, dose reduction to 450 mg/day or 900 mg/day, respectively, were allowed.

Drug: idebenone

Interventions

idebenone DRUG

Idebenone 1350 mg/d, patients \< or equal 45 kg Idebenone 2250 mg/d, patients \> 45 kg

Idebenone

Eligibility Criteria

• Age: 9 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Completion of 52 weeks in study SNT-III-001
• Body weight ≥ 25 kg
• Negative urine pregnancy test
• Eligibility to participate in the present extension study as confirmed by investigator

You may not qualify if:

• Safety or tolerability issues arising during the course of SNT-III-001 which in the opinion of the investigator preclude further treatment with idebenone
• Clinically significant abnormalities of haematology or biochemistry including, but not limited to, elevations greater than 1.5 times the upper limit of SGOT, SGPT or creatinine
• Parallel participation in another clinical drug trial
• Pregnancy or breast-feeding
• Abuse of drugs or alcohol

Sponsors & Collaborators

• Santhera Pharmaceuticals: lead

Study Sites (11)

Universitätsklinik Innsbruck

Innsbruck, Austria

Location

Hôpital Erasme - Univeristé Libre de Bruxelles

Brussels, 1070, Belgium

Location

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

Paris, 75651, France

Location

HELIOS Klinikum Berlin

Berlin, 13125, Germany

Location

Neurologische Universitätsklinik und Ploklinik - Universitätsklimikum Bonn

Bonn, 53105, Germany

Location

Klinik II, Neuropädiatrie und Muskelerkrankungen - Universitätsklinik Freiburg

Freiburg im Breisgau, 79106, Germany

Location

Zentrum für Neurologische Medizin

Göttingen, 37073, Germany

Location

UKE Hamburg Neuropädiatrie - Zentrum 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

MeSH Terms

Interventions

idebenone

Results Point of Contact

• Title: Thomas Meier, PhD
• Organization: Santhera Pharmaceuticals

thomas.meier@santhera.com <Thomas.Meier@santhera.com>

+41 61 906 89 50

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: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 13, 2009
• First Posted: October 14, 2009
• Study Start: June 1, 2007
• Primary Completion: June 1, 2012
• Study Completion: June 1, 2012
• Last Updated: March 5, 2018
• Results First Posted: March 5, 2018

Record last verified: 2017-08

Locations

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