Trial · NCT00697073

Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients

NCT00697073, Phase 3, Completed, Santhera Pharmaceuticals; results first posted 2011-08-04

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients. Trials, record NCT00697073. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00697073

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Trial

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

Title: Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients
NCT number: NCT00697073
Status: Completed
Phases: Phase 3
Enrollment: 68 participants, actual
Lead sponsor: Santhera Pharmaceuticals
Conditions: Friedreich's Ataxia
Interventions: Idebenone
First posted: 2008-06-13
Last updated: 2023-07-11
Results first posted: 2011-08-04

Source ClinicalTrials.gov

Registered outcomes4

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

List	Outcome	Time frame	Description
Primary	Change in ICARS	baseline and 12 months	International Cooperative Ataxia Rating Scale (ICARS): ICARS consists of a one-hundred-point semi-quantitative scale based upon 19 simple testing manoeuvres compartmentalized into postural and stance disorders, limb ataxia, dysarthria and oculomotor disorders and has been previously used in this patient population with good inter-rater reliability. A total score of 0 points represents the best possible score, and a value of 100 points is the worst possible score. Therefore, a negative change in ICARS score represents an improvement. A higher score also indicates more disability.
Secondary	FARS (Friedreich's Ataxia Rating Scale)	baseline and 12 Months	FARS consists of a 25 manoeuvre exam along with 4 quantitative performance measures. The neurological exam covers bulbar function, upper limb coordination, lower limb coordination, peripheral nervous system function, deep tendon reflexes, stability and gait. The use of FARS has been recently validated as a sensitive scale for this population. The FARS consists of three subscales, comprising a general score for ataxia, a score for activities of daily living (ADL) and a neurological examination. The scores can be added to make a total score ranging from 0 to 159. A higher score indicates a greater level of disability.
Secondary	Nature of Adverse Events	12 Months
Secondary	Frequency/Number of Mild, Moderate, and Severe Adverse Events	12 months

Source ClinicalTrials.gov

Outcomes4

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

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Change in ICARS	baseline and 12 months	A total of 59 subjects completed the study. Nine subjects did not complete the study. However, neurological efficacy data (ICARS, FARS) are available for Week 52 from 61 patients.	units on a scale	MEAN
SECONDARY	FARS (Friedreich's Ataxia Rating Scale)	baseline and 12 Months	A total of 59 subjects completed the study. Nine subjects did not complete the study. However, neurological efficacy data (ICARS, FARS) are available for Week 52 from 61 patients.	units on a scale	MEAN
SECONDARY	Nature of Adverse Events	12 Months	All 68 subjects enrolled in this study were included in the Safety population.	events	NUMBER
SECONDARY	Frequency/Number of Mild, Moderate, and Severe Adverse Events	12 months	All 68 subjects enrolled in this study were included in the Safety population.	events	NUMBER

Source ClinicalTrials.gov

Results by group8

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

Outcome	Group	Category	Value	Spread	Units
Change in ICARS	High Dose Idebenone		1.1	Standard Deviation 5.64	units on a scale
FARS (Friedreich's Ataxia Rating Scale)	High Dose Idebenone		2.3	Standard Deviation 5.59	units on a scale
Nature of Adverse Events	High Dose Idebenone	Infections and Infestations	117		events
Nature of Adverse Events	High Dose Idebenone	Gastrointestinal Disorders	97		events
Nature of Adverse Events	High Dose Idebenone	Musculoskeletal and Connective Tissue Disorders	75		events
Frequency/Number of Mild, Moderate, and Severe Adverse Events	High Dose Idebenone	mild	30		events
Frequency/Number of Mild, Moderate, and Severe Adverse Events	High Dose Idebenone	moderate	32		events
Frequency/Number of Mild, Moderate, and Severe Adverse Events	High Dose Idebenone	severe	4		events

Source ClinicalTrials.gov

Participant flow3

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

Period	Milestone	Group	Participants
Overall Study	STARTED	High Dose Idebenone	68
Overall Study	COMPLETED	High Dose Idebenone	59
Overall Study	NOT COMPLETED	High Dose Idebenone	9

Source ClinicalTrials.gov

Adverse events15

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	headache	General disorders	High Dose Idebenone	21	68
other	upper respiratory tract infection	Infections and infestations	High Dose Idebenone	18	68
other	nausea	Gastrointestinal disorders	High Dose Idebenone	15	68
other	pyrexia	General disorders	High Dose Idebenone	14	68
serious	cardiac failure	Cardiac disorders	High Dose Idebenone	1	68	1
serious	tachycardia	Cardiac disorders	High Dose Idebenone	1	68	1
serious	Hyperthyroidism	Endocrine disorders	High Dose Idebenone	1	68	1
serious	Hypotension	General disorders	High Dose Idebenone	1	68	1
serious	Deep vein thrombosis	General disorders	High Dose Idebenone	1	68	1
serious	Dehydratation	General disorders	High Dose Idebenone	1	68	1

1 to 10 of 15Source ClinicalTrials.gov

See all 15 adverse events

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	High Dose Idebenone
Total, other adverse events	High Dose Idebenone	66	68
Total, serious adverse events	High Dose Idebenone	4	68

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
21779958	RESULT

Source PubMed

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.

GET https://conductscience.org/api/trials/v1/NCT00697073

GET https://conductscience.org/api/trials/v1/NCT00697073/registered

API reference For AI assistants