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

This study has been completed.

Sponsor:

Information provided by (Responsible Party):

Santhera Pharmaceuticals

ClinicalTrials.gov Identifier:

NCT00905268

First received: May 19, 2009

Last updated: May 19, 2016

Last verified: May 2016

History of Changes

Results First Received: October 20, 2015

Study Type:	Interventional
Study Design:	Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Double Blind (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
Condition:	Friedreich's Ataxia
Interventions:	Drug: idebenone Drug: Placebo

Participant Flow

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Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups

	Description
Group A: Idebenone	Patients under/equal 45 kg: idebenone 180 mg/day Patients over 45 kg: idebenone 360 mg/day idebenone: 12 months of 1 of 3 treatments arms of oral idebenone or placebo.Treatment taken 3 times daily with meals.
Group B: Idebenone	Patients under/equal 45 kg: idebenone 450 mg/day Patients over 45 kg: idebenone 900 mg/day idebenone: 12 months of 1 of 3 treatments arms of oral idebenone or placebo.Treatment taken 3 times daily with meals.
C: Idebenone	Patients under/equal 45 kg: idebenone 1350 mg/day Patients over 45 kg: idebenone 2250 mg/day idebenone: 12 months of 1 of 3 treatments arms of oral idebenone or placebo.Treatment taken 3 times daily with meals.
D: Placebo	placebo Placebo: 12 months of 1 of 3 treatments arms of oral idebenone or placebo.Treatment taken 3 times daily with meals.

Participant Flow: Overall Study

	Group A: Idebenone	Group B: Idebenone	C: Idebenone	D: Placebo
STARTED	57	57	59	59
COMPLETED	53	56	59	57
NOT COMPLETED	4	1	0	2

Baseline Characteristics

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Outcome Measures

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1. Primary:

Absolute Change in International Cooperative Ataxia Rating Scale (ICARS) Scores From Baseline Assessment to Week 52 [ Time Frame: Baseline and week 52 ]

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2. Secondary:

Absolute Change in Friedreich's Ataxia Rating Scale (FARS) Scores From Baseline Assessment to Week 52 [ Time Frame: Baseline and week 52 ]

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3. Secondary:

Proportion of Patients Improving (Responding) on ICARS by a Clinically Relevant Margin [ Time Frame: week 52 ]

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4. Secondary:

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 [ Time Frame: 1 year ]

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5. Secondary:

Change in Peak Systolic Strain Rate From Baseline to Week 52 [ Time Frame: 1 year ]

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6. Secondary:

Change in Peak Workload From Baseline to Week 52 [ Time Frame: 1 year ]

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Serious Adverse Events

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Other Adverse Events

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Limitations and Caveats

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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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More Information

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Certain Agreements:

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Results Point of Contact:

Name/Title: Prof. Nicholas William Wood
Organization: The National Hospital, University College London
phone: 020 7837 3611
e-mail: n.wood@ucl.ac.uk

Responsible Party:	Santhera Pharmaceuticals
ClinicalTrials.gov Identifier:	NCT00905268 History of Changes
Other Study ID Numbers:	SNT-III-001
Study First Received:	May 19, 2009
Results First Received:	October 20, 2015
Last Updated:	May 19, 2016

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