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

August 21, 2017 updated by: Santhera Pharmaceuticals

A Phase III Open-Label, Single-Group, Extension Study to Obtain Long-Term Safety and Tolerability Data of Idebenone in the Treatment of Friedreich's Ataxia Patients.

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

200

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Austria
      • Innsbruck, Austria
        • Universitätsklinik Innsbruck
  • Belgium
      • Bruxelles, Belgium, 1070
        • Hôpital Erasme - Univeristé Libre de Bruxelles
  • France
      • Paris, France, 75651
        • Hôpital de la Salpétrière - INSERM U679, Neurologie et Thérapeutique expérimentale
  • Germany
      • Berlin, Germany, 13125
        • Helios Klinikum Berlin
      • Bonn, Germany, 53105
        • Neurologische Universitätsklinik und Ploklinik - Universitätsklimikum Bonn
      • Freiburg, Germany, 79106
        • Klinik II, Neuropädiatrie und Muskelerkrankungen - Universitätsklinik Freiburg
      • Göttingen, Germany, 37073
        • Zentrum für Neurologische Medizin
      • Hamburg, Germany, 20246
        • UKE Hamburg Neuropädiatrie - Zentrum für Frauen, Kinder und Jugendmedizin
      • München, Germany, 81377
        • Neurologische Klinik - Klinikum Grosshadern
      • Tübingen, Germany, 72076
        • Neurologische Universitätsklinik und Poliklinik
  • Netherlands
      • Groningen, Netherlands, 9700 RB
        • University Medical Center Groningen

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

9 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Idebenone
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
Idebenone 1350 mg/d, patients < or equal 45 kg Idebenone 2250 mg/d, patients > 45 kg

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Measures of Safety and Tolerability: Nature and Frequency of Adverse Events (AEs)
Time Frame: overall study, up to 24 months
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)
Time Frame: Baseline, Month 12 and month 24
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 Outcome Measures

Outcome Measure
Measure Description
Time Frame
Measures of Safety and Tolerability: Physical Examinations and Vital Signs
Time Frame: Month 1, 3, 6, 12, 18 and 24
Assessment of the head, eyes, ears, nose, throat, heart, chest, lungs, abdomen, extremities, peripheral pulses, skin and any other physical conditions of note.
Month 1, 3, 6, 12, 18 and 24
Measures of Safety and Tolerability: Electrocardiograms (ECGs)
Time Frame: Month 1, 3, 6, 12, 18 and 24
12-lead ECG recordings were performed at every visit. Each ECG was measured using 3 complexes: PR interval in lead II or V2, QRS and QT intervals and heart rate in lead II, corrected QT intervals QTcB and QTcF.
Month 1, 3, 6, 12, 18 and 24
Measures of Safety and Tolerability: Haematological and Biochemical Laboratory Parameters
Time Frame: Month 1, 3, 6, 12, 18 and 24
Safety haematological analysis were done at every visit. Analyses included red blood cell count, haemoglobin, haematocrit, red cell indices, white blood cell count including differential, platelet count Safety biochemistry were done at every visit. Analyses included sodium, potassium, chloride, bicarbonate, urea, creatinine, calcium, inorganic phosphate, glucose, total bilirubin, total protein, albumin, aspartate amiotransferase (AST), alanine aminotransferase (ALT), alkaline phosphotase, Gamma GT, creatine kinase (CK)^, cholesterol, triglycerides, uric acid.
Month 1, 3, 6, 12, 18 and 24

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Santhera Pharmaceuticals

Investigators

  • Principal Investigator: Nick Wood, Professor, Dept of Molecular Neuroscience, Institute of Neurology. The National Hospital, University College London

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

June 1, 2007

Primary Completion (Actual)

June 1, 2012

Study Completion (Actual)

June 1, 2012

Study Registration Dates

First Submitted

October 13, 2009

First Submitted That Met QC Criteria

October 13, 2009

First Posted (Estimate)

October 14, 2009

Study Record Updates

Last Update Posted (Actual)

March 5, 2018

Last Update Submitted That Met QC Criteria

August 21, 2017

Last Verified

August 1, 2017

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SNT-III-001-E

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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