Trial · NCT00537680
Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia
NCT00537680, Phase 3, Completed, Santhera Pharmaceuticals; results first posted 2011-12-29
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia. Trials, record NCT00537680. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00537680
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia
- NCT number
- NCT00537680
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 70 participants, actual
- Lead sponsor
- Santhera Pharmaceuticals
- Conditions
- Friedreich's Ataxia
- Interventions
- Idebenone, Idebenone, Placebo
- First posted
- 2007-10-01
- Last updated
- 2023-07-11
- Results first posted
- 2011-12-29
Source ClinicalTrials.gov
Registered outcomes12
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 24 | baseline and 6 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. Minimum score: 0, maximum score 100, higher score indicates greater impairment Calculation details: ICARS score at week 24 minus ICARS score baseline |
| Secondary | Change in Friedreich's Ataxia Rating Scale (FARS) Scores From Baseline to Week 24 | baseline and 6 months | To compare the efficacy of 6 months' treatment with 2 different doses of idebenone with that of placebo on neurological function as assessed by the Friedreich's Ataxia Rating Scale (FARS) 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 exam scores can be added to make a total score ranging from 0 to 159. A higher score indicates a greater level of disability. Calculation details FARS score at week 24 minus FARS score baseline |
| Secondary | Activities of Daily Living (ADL) of Friedreich's Ataxia Rating Scale (FARS), Change in ADL (Total Score ) From Baseline to Week 24 | baseline and 6 months | To compare the efficacy of 6 months' treatment with 2 different doses of idebenone with that of placebo on Activities of Daily Living (ADL) assessed by the ADL scale of the Friedreich's Ataxia Rating Scale (FARS) score 0 to 36, higher score indicates greater impairment |
| Secondary | Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 12 | baseline and 12 weeks | To compare the efficacy of 3 months' treatment with 2 different doses of idebenone with that of placebo on neurological impairment as assessed by the International Cooperative Ataxia Rating Scale (ICARS) 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. Minimum score: 0, maximum score 100, higher score indicates greater impairment Calculation details: ICARS score at week 12 minus ICARS score baseline |
| Secondary | Absolute Change From Baseline to Week 24 in the Relative Wall Thickness (RWT) of the Left Ventricle | baseline and 6 months | To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography |
| Secondary | Percent Change From Baseline to Week 24 in the Relative Wall Thickness (RWT) of the Left Ventricle | baseline and 6 months | To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography |
| Secondary | Absolute Change From Baseline to Week 24 in Left Ventricular Mass Index (LVMI) | baseline and 6 months | To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography |
| Secondary | Percent Change From Baseline to Week 24 in Left Ventricular Mass Index | baseline and 6 months | To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography |
| Secondary | Absolute Change From Baseline to Week 24 in the Thickness of the Posterior Wall (PW) of the Left Ventricle | baseline and 6 months | To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography |
| Secondary | Percent Change From Baseline to Week 24 in the Thickness of the Posterior Wall (PW) of the Left Ventricle | baseline and 6 months | To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 registered outcomesOutcomes12
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 24 | baseline and 6 months | ICARS points | MEAN | |
| SECONDARY | Change in Friedreich's Ataxia Rating Scale (FARS) Scores From Baseline to Week 24 | baseline and 6 months | FARS score | MEAN | |
| SECONDARY | Activities of Daily Living (ADL) of Friedreich's Ataxia Rating Scale (FARS), Change in ADL (Total Score ) From Baseline to Week 24 | baseline and 6 months | ADL points | MEAN | |
| SECONDARY | Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 12 | baseline and 12 weeks | ICARS points | MEAN | |
| SECONDARY | Absolute Change From Baseline to Week 24 in the Relative Wall Thickness (RWT) of the Left Ventricle | baseline and 6 months | cm | MEAN | |
| SECONDARY | Percent Change From Baseline to Week 24 in the Relative Wall Thickness (RWT) of the Left Ventricle | baseline and 6 months | % change | MEAN | |
| SECONDARY | Absolute Change From Baseline to Week 24 in Left Ventricular Mass Index (LVMI) | baseline and 6 months | g/m2 | MEAN | |
| SECONDARY | Percent Change From Baseline to Week 24 in Left Ventricular Mass Index | baseline and 6 months | % change | MEAN | |
| SECONDARY | Absolute Change From Baseline to Week 24 in the Thickness of the Posterior Wall (PW) of the Left Ventricle | baseline and 6 months | cm | MEAN | |
| SECONDARY | Percent Change From Baseline to Week 24 in the Thickness of the Posterior Wall (PW) of the Left Ventricle | baseline and 6 months | % change | MEAN |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 outcomesResults by group36
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 24 | Mid Dose Idebenone | -2.5 | Standard Deviation 6.3 | ICARS points | |
| Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 24 | High Dose Idebenone | -2.4 | Standard Deviation 4.8 | ICARS points | |
| Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 24 | Placebo | -1.3 | Standard Deviation 4.4 | ICARS points | |
| Change in Friedreich's Ataxia Rating Scale (FARS) Scores From Baseline to Week 24 | Mid Dose Idebenone | -1.6 | Standard Deviation 6.52 | FARS score | |
| Change in Friedreich's Ataxia Rating Scale (FARS) Scores From Baseline to Week 24 | High Dose Idebenone | -1.2 | Standard Deviation 4.53 | FARS score | |
| Change in Friedreich's Ataxia Rating Scale (FARS) Scores From Baseline to Week 24 | Placebo | 0.6 | Standard Deviation 5.91 | FARS score | |
| Activities of Daily Living (ADL) of Friedreich's Ataxia Rating Scale (FARS), Change in ADL (Total Score ) From Baseline to Week 24 | Mid Dose Idebenone | 0.2 | Standard Deviation 2.45 | ADL points | |
| Activities of Daily Living (ADL) of Friedreich's Ataxia Rating Scale (FARS), Change in ADL (Total Score ) From Baseline to Week 24 | High Dose Idebenone | 0.6 | Standard Deviation 3.13 | ADL points | |
| Activities of Daily Living (ADL) of Friedreich's Ataxia Rating Scale (FARS), Change in ADL (Total Score ) From Baseline to Week 24 | Placebo | 1.0 | Standard Deviation 2.97 | ADL points | |
| Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 12 | Mid Dose Idebenone | -1.5 | Standard Deviation 5.35 | ICARS points |
1 to 10 of 36Source ClinicalTrials.gov
See all 36 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Mid Dose Idebenone | 22 | |
| Overall Study | STARTED | High Dose Idebenone | 24 | |
| Overall Study | STARTED | Placebo | 24 | |
| Overall Study | COMPLETED | Mid Dose Idebenone | 22 | |
| Overall Study | COMPLETED | High Dose Idebenone | 22 | |
| Overall Study | COMPLETED | Placebo | 24 | |
| Overall Study | NOT COMPLETED | Mid Dose Idebenone | 0 | |
| Overall Study | NOT COMPLETED | High Dose Idebenone | 2 | |
| Overall Study | NOT COMPLETED | Placebo | 0 |
Source ClinicalTrials.gov
Adverse events21
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 | Infections | Infections and infestations | Mid Dose Idebenone | 11 | 22 | |
| other | Infections | Infections and infestations | High Dose Idebenone | 15 | 24 | |
| other | Infections | Infections and infestations | Placebo | 16 | 24 | |
| other | Nervous system disorders | Nervous system disorders | Mid Dose Idebenone | 10 | 22 | |
| other | Nervous system disorders | Nervous system disorders | High Dose Idebenone | 14 | 24 | |
| other | Nervous system disorders | Nervous system disorders | Placebo | 12 | 24 | |
| other | Gastrointestinal disorders | Gastrointestinal disorders | Mid Dose Idebenone | 8 | 22 | |
| other | Gastrointestinal disorders | Gastrointestinal disorders | High Dose Idebenone | 15 | 24 | |
| other | Gastrointestinal disorders | Gastrointestinal disorders | Placebo | 12 | 24 | |
| other | General disorders | General disorders | Mid Dose Idebenone | 10 | 22 |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Mid Dose Idebenone | ||
| Total, all-cause mortality | High Dose Idebenone | ||
| Total, all-cause mortality | Placebo | ||
| Total, other adverse events | Mid Dose Idebenone | 18 | 22 |
| Total, other adverse events | High Dose Idebenone | 23 | 24 |
| Total, other adverse events | Placebo | 21 | 24 |
| Total, serious adverse events | Mid Dose Idebenone | 0 | 22 |
| Total, serious adverse events | High Dose Idebenone | 2 | 24 |
| Total, serious adverse events | Placebo | 0 | 24 |
Source ClinicalTrials.gov
Papers3
The papers the trial lists on ClinicalTrials.gov.
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.
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.
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