Trial · NCT00905268
A Study of Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia (FRDA) Patients
NCT00905268, Phase 3, Completed, Santhera Pharmaceuticals; results first posted 2016-06-27
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study of Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia (FRDA) Patients. Trials, record NCT00905268. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00905268
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia (FRDA) Patients
- NCT number
- NCT00905268
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 232 participants, actual
- Lead sponsor
- Santhera Pharmaceuticals
- Conditions
- Friedreich's Ataxia
- Interventions
- idebenone, Placebo
- First posted
- 2009-05-20
- Last updated
- 2016-06-27
- Results first posted
- 2016-06-27
Source ClinicalTrials.gov
Registered outcomes6
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Absolute Change in International Cooperative Ataxia Rating Scale (ICARS) Scores From Baseline Assessment to Week 52 | Baseline and week 52 | 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. |
| Secondary | Absolute Change in Friedreich's Ataxia Rating Scale (FARS) Scores From Baseline Assessment to Week 52 | Baseline and week 52 | The Friedreich Ataxia Rating Scale (FARS) is made up of a measure of ataxia, and activities of daily living subscale and a neurological subscale. The scores from the three subscales are added to generate a total score ranging from 0 to 159, with a higher score indicating a greater level of disability. |
| Secondary | Proportion of Patients Improving (Responding) on ICARS by a Clinically Relevant Margin | week 52 | 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. ICARS Responder Analysis at Week 52: Percentage of subjects Improving by 2.5 Points or More. |
| 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 | 1 year | (In the statistical analysis sub-population presenting with cardiac involvement as defined by the FRDA cardiomyopathy criteria) |
| Secondary | Change in Peak Systolic Strain Rate From Baseline to Week 52 | 1 year | Mean Change of Peak systolic longitudinal strain rate (PSLSR) from Baseline to Week 52 in subjects with cardiac involvement (FRDA-CM criteria), where positive value in PSLSR is a deterioration and negative value an improvement. |
| Secondary | Change in Peak Workload From Baseline to Week 52 | 1 year | Assessed by a modified exercise test, in a subset of patients able to undertake this. Wpeak has been calculated from the following formula: Workload last fully completed stage + (seconds completed in last stage / 60 \* (4 \[if arm ergonometry\] or 10 \[if leg ergonometry\])). |
Source ClinicalTrials.gov
Outcomes6
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Absolute Change in International Cooperative Ataxia Rating Scale (ICARS) Scores From Baseline Assessment to Week 52 | Baseline and week 52 | The Intent-To-Treat (ITT) population included all randomized subjects who received at least one dose of the study medication and had a confirmed diagnosis of FRDA, did not take idebenone within one month pre-Screening or between Screening and Visit 1, and were not under idebenone treatment at Baseline according to available PK results at Visit 1. | units on a scale | MEAN |
| SECONDARY | Absolute Change in Friedreich's Ataxia Rating Scale (FARS) Scores From Baseline Assessment to Week 52 | Baseline and week 52 | The comparison was carried out in the ITT population, on data imputed using the last observation carried forward (LOCF) method. | units on a scale | MEAN |
| SECONDARY | Proportion of Patients Improving (Responding) on ICARS by a Clinically Relevant Margin | week 52 | The Intent-To-Treat (ITT) population included all randomized subjects who received at least one dose of the study medication and had a confirmed diagnosis of FRDA, did not take idebenone within one month pre-Screening or between Screening and Visit 1, and were not under idebenone treatment at Baseline according to available PK results at Visit 1. | percentage of patients | NUMBER |
| 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 | 1 year | Subgroup of subjects with cardiac involvement as defined by Friedreich's ataxia cardiomyopathy (FRDA-CM) | percentage of patients | NUMBER |
| SECONDARY | Change in Peak Systolic Strain Rate From Baseline to Week 52 | 1 year | Subgroup of subjects with cardiac involvement as defined by Friedreich's ataxia cardiomyopathy (FRDA-CM) | 1/s | MEAN |
| SECONDARY | Change in Peak Workload From Baseline to Week 52 | 1 year | Watts | MEAN |
Source ClinicalTrials.gov
Results by group24
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Absolute Change in International Cooperative Ataxia Rating Scale (ICARS) Scores From Baseline Assessment to Week 52 | Group A: Idebenone | 1.6 | Standard Deviation 5.85 | units on a scale | |
| Absolute Change in International Cooperative Ataxia Rating Scale (ICARS) Scores From Baseline Assessment to Week 52 | Group B: Idebenone | 1.7 | Standard Deviation 6.64 | units on a scale | |
| Absolute Change in International Cooperative Ataxia Rating Scale (ICARS) Scores From Baseline Assessment to Week 52 | C: Idebenone | 1.2 | Standard Deviation 5.22 | units on a scale | |
| Absolute Change in International Cooperative Ataxia Rating Scale (ICARS) Scores From Baseline Assessment to Week 52 | D: Placebo | 1.1 | Standard Deviation 6.76 | units on a scale | |
| Absolute Change in Friedreich's Ataxia Rating Scale (FARS) Scores From Baseline Assessment to Week 52 | Group A: Idebenone | 0.9 | Standard Deviation 7.19 | units on a scale | |
| Absolute Change in Friedreich's Ataxia Rating Scale (FARS) Scores From Baseline Assessment to Week 52 | Group B: Idebenone | 1.2 | Standard Deviation 5.24 | units on a scale | |
| Absolute Change in Friedreich's Ataxia Rating Scale (FARS) Scores From Baseline Assessment to Week 52 | C: Idebenone | 1.4 | Standard Deviation 5.60 | units on a scale | |
| Absolute Change in Friedreich's Ataxia Rating Scale (FARS) Scores From Baseline Assessment to Week 52 | D: Placebo | 0.9 | Standard Deviation 6.77 | units on a scale | |
| Proportion of Patients Improving (Responding) on ICARS by a Clinically Relevant Margin | Group A: Idebenone | 18.2 | percentage of patients | ||
| Proportion of Patients Improving (Responding) on ICARS by a Clinically Relevant Margin | Group B: Idebenone | 23.6 | percentage of patients |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Group A: Idebenone | 57 | |
| Overall Study | STARTED | Group B: Idebenone | 57 | |
| Overall Study | STARTED | C: Idebenone | 59 | |
| Overall Study | STARTED | D: Placebo | 59 | |
| Overall Study | COMPLETED | Group A: Idebenone | 53 | |
| Overall Study | COMPLETED | Group B: Idebenone | 56 | |
| Overall Study | COMPLETED | C: Idebenone | 59 | |
| Overall Study | COMPLETED | D: Placebo | 57 | |
| Overall Study | NOT COMPLETED | Group A: Idebenone | 4 | |
| Overall Study | NOT COMPLETED | Group B: Idebenone | 1 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events236
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 | palpitations | Cardiac disorders | Group A: Idebenone | 1 | 57 | 1 |
| other | palpitations | Cardiac disorders | Group B: Idebenone | 1 | 57 | 1 |
| other | palpitations | Cardiac disorders | C: Idebenone | 3 | 59 | 3 |
| other | palpitations | Cardiac disorders | D: Placebo | 2 | 59 | 2 |
| other | tachycardia | Cardiac disorders | Group A: Idebenone | 3 | 57 | 3 |
| other | tachycardia | Cardiac disorders | Group B: Idebenone | 0 | 57 | 0 |
| other | tachycardia | Cardiac disorders | C: Idebenone | 1 | 59 | 1 |
| other | tachycardia | Cardiac disorders | D: Placebo | 2 | 59 | 2 |
| other | vertigo | Ear and labyrinth disorders | Group A: Idebenone | 1 | 57 | 1 |
| other | vertigo | Ear and labyrinth disorders | Group B: Idebenone | 2 | 57 | 2 |
1 to 10 of 236Source ClinicalTrials.gov
See all 236 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Group A: Idebenone | ||
| Total, all-cause mortality | Group B: Idebenone | ||
| Total, all-cause mortality | C: Idebenone | ||
| Total, all-cause mortality | D: Placebo | ||
| Total, other adverse events | Group A: Idebenone | 55 | 57 |
| Total, other adverse events | Group B: Idebenone | 52 | 57 |
| Total, other adverse events | C: Idebenone | 52 | 59 |
| Total, other adverse events | D: Placebo | 55 | 59 |
| Total, serious adverse events | Group A: Idebenone | 7 | 57 |
| Total, serious adverse events | Group B: Idebenone | 4 | 57 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers0
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.
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/NCT00905268
GET https://conductscience.org/api/trials/v1/NCT00905268/results?page=2