Trial · NCT00993967

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

NCT00993967, Phase 3, Completed, Santhera Pharmaceuticals; results first posted 2018-03-05

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Long-Term Safety and Tolerability of Idebenone in Friedreich's Ataxia Patients (MICONOS Extension). Trials, record NCT00993967. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00993967

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Long-Term Safety and Tolerability of Idebenone in Friedreich's Ataxia Patients (MICONOS Extension)
NCT number: NCT00993967
Status: Completed
Phases: Phase 3
Enrollment: 200 participants, actual
Lead sponsor: Santhera Pharmaceuticals
Conditions: Freidreich's Ataxia
Interventions: idebenone
First posted: 2009-10-14
Last updated: 2018-03-05
Results first posted: 2018-03-05

Source ClinicalTrials.gov

Registered outcomes5

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Measures of Safety and Tolerability: Nature and Frequency of Adverse Events (AEs)	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.
Primary	Absolute Change in The International Cooperative Ataxia Rating Scale (ICARS)	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.
Other	Measures of Safety and Tolerability: Physical Examinations and Vital Signs	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.
Other	Measures of Safety and Tolerability: Electrocardiograms (ECGs)	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.
Other	Measures of Safety and Tolerability: Haematological and Biochemical Laboratory Parameters	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.

Source ClinicalTrials.gov

Outcomes5

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Measures of Safety and Tolerability: Nature and Frequency of Adverse Events (AEs)	overall study, up to 24 months		participants	NUMBER
PRIMARY	Absolute Change in The International Cooperative Ataxia Rating Scale (ICARS)	Baseline, Month 12 and month 24	Changes in Total ICARS Score for all patients completing the study (CC Population)	units on a scale	MEAN
OTHER_PRE_SPECIFIED	Measures of Safety and Tolerability: Physical Examinations and Vital Signs	Month 1, 3, 6, 12, 18 and 24
OTHER_PRE_SPECIFIED	Measures of Safety and Tolerability: Electrocardiograms (ECGs)	Month 1, 3, 6, 12, 18 and 24
OTHER_PRE_SPECIFIED	Measures of Safety and Tolerability: Haematological and Biochemical Laboratory Parameters	Month 1, 3, 6, 12, 18 and 24

Source ClinicalTrials.gov

Results by group9

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Measures of Safety and Tolerability: Nature and Frequency of Adverse Events (AEs)	Idebenone	With at least one AE	188		participants
Measures of Safety and Tolerability: Nature and Frequency of Adverse Events (AEs)	Idebenone	With at least one drug-related AE	109		participants
Measures of Safety and Tolerability: Nature and Frequency of Adverse Events (AEs)	Idebenone	With at least one severe AE	8		participants
Measures of Safety and Tolerability: Nature and Frequency of Adverse Events (AEs)	Idebenone	With any significant AE	54		participants
Measures of Safety and Tolerability: Nature and Frequency of Adverse Events (AEs)	Idebenone	Death	1		participants
Measures of Safety and Tolerability: Nature and Frequency of Adverse Events (AEs)	Idebenone	Other serious adverse events than death	48		participants
Measures of Safety and Tolerability: Nature and Frequency of Adverse Events (AEs)	Idebenone	AEs leading to discontinuation	26		participants
Absolute Change in The International Cooperative Ataxia Rating Scale (ICARS)	Idebenone	From Baseline to Month 12	1.41	Standard Deviation 7.14	units on a scale
Absolute Change in The International Cooperative Ataxia Rating Scale (ICARS)	Idebenone	From Baseline to Month 24	2.88	Standard Deviation 7.57	units on a scale

Source ClinicalTrials.gov

Participant flow3

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Idebenone	200
Overall Study	COMPLETED	Idebenone	139
Overall Study	NOT COMPLETED	Idebenone	61

Source ClinicalTrials.gov

Adverse events75

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	abdominal pain upper	Gastrointestinal disorders	Idebenone	16	200	16
other	diarrhoea	Gastrointestinal disorders	Idebenone	37	200	37
other	nausea	Gastrointestinal disorders	Idebenone	20	200	20
other	vomiting	Gastrointestinal disorders	Idebenone	11	200	11
other	pyrexia	General disorders	Idebenone	10	200	10
other	bronchitis	Infections and infestations	Idebenone	12	200	12
other	cystitis	Infections and infestations	Idebenone	12	200	12
other	influenza	Infections and infestations	Idebenone	19	200	19
other	nasopharyngitis	Infections and infestations	Idebenone	67	200	67
other	fall	Injury, poisoning and procedural complications	Idebenone	13	200	13

1 to 10 of 75Source ClinicalTrials.gov

See all 75 adverse events

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Idebenone
Total, other adverse events	Idebenone	188	200
Total, serious adverse events	Idebenone	49	200

Source ClinicalTrials.gov

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

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GET https://conductscience.org/api/trials/v1/NCT00993967/events?page=2

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