Trial · NCT02255435
A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia
NCT02255435, Phase 2, Completed, Biogen; results first posted 2022-11-29
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia. Trials, record NCT02255435. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02255435
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia
- NCT number
- NCT02255435
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 172 participants, actual
- Lead sponsor
- Biogen
- Conditions
- Friedreich Ataxia
- Interventions
Omaveloxolone Capsules, 2.5 mg, Omaveloxolone Capsules, 5 mg, Omaveloxolone Capsules, 10 mg, Placebo, Omaveloxolone Capsules, 20 mg, Omaveloxolone Capsules, 40 mg, Omaveloxolone Capsules, 80 mg, Omaveloxolone Capsules, 160 mg, Omaveloxolone Capsules, 300 mg, Omaveloxolone Capsules, 150 mg
- First posted
- 2014-10-02
- Last updated
- 2026-09-03
- Results first posted
- 2022-11-29
Source ClinicalTrials.gov
Registered outcomes12
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Part 1: Change From Baseline in Peak Work During Maximal Exercise Testing at Week 12 | Baseline, Week 12 | Cycle ergometry using a recumbent stationary bicycle was used to conduct maximal exercise testing and workload was increased incrementally. Peak work is defined as the workload at which participants reach maximal volition (defined as an inability to continue to exercise due to exhaustion). A positive change from baseline suggests an improvement. |
| Primary | Part 1: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs) | From first dose of study drug up to end of Part 1 of the study (up to Week 16) | An adverse event (AE) was any unfavorable and unintended sign (including any clinically significant abnormal laboratory test result), symptom, or disease temporally associated with the use of the study drug whether or not it is considered to be study drug related. An SAE was any untoward medical occurrence that at any dose resulted in death, in the view of the Investigator, placed the participant at immediate risk of death, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, resulted in a birth defect. TEAEs were defined as any AEs, regardless of relationship to study drug, that had an onset or worsened in severity on or after the first dose of study drug. |
| Primary | Part 2: Change From Baseline in the Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 48 | Baseline, Week 48 | The Friedreich Ataxia Rating Scale (FARS) is a neurological-exam-based rating scale with five sections: Bulbar (section A), Upper Limb Coordination (section B), Lower Limb Coordination (section C), Peripheral Nervous System (section D), and Upright Stability (section E). mFARS is the sum of sections A (score 0 to 11), B (score 0 to 36), C (score 0 to 16), and E (score 0 to 36). The minimum score is 0 and the maximum score is 99. A lower score indicates better neurological function. |
| Primary | Part 2: Number of Participants With TEAEs and TESAEs | From first dose of study drug up to end of Part 2 of the study (up to Week 52) | An AE was any unfavorable and unintended sign (including any clinically significant abnormal laboratory test result), symptom, or disease temporally associated with the use of the study drug whether or not it is considered to be study drug related. An SAE was any untoward medical occurrence that at any dose resulted in death, in the view of the Investigator, placed the participant at immediate risk of death, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, resulted in a birth defect. TEAEs were defined as any AEs, regardless of relationship to study drug, that had an onset or worsened in severity on or after the first dose of study drug. |
| Secondary | Part 1: Change From Baseline in the mFARS at Week 12 | Baseline, Week 12 | The FARS is a neurological-exam-based rating scale with five sections: Bulbar (section A), Upper Limb Coordination (section B), Lower Limb Coordination (section C), Peripheral Nervous System (section D), and Upright Stability (section E). mFARS is the sum of sections A (score 0 to 11), B (score 0 to 36), C (score 0 to 16), and E (score 0 to 36). The minimum score is 0 and the maximum score is 99. A lower score indicates better neurological function. |
| Secondary | Part 2: Number of Participants With Patient Global Impression of Change (PGI-C) | Week 48 | The PGI-C is a 7-point scale that requires participants to assess how much their illness has improved or worsened relative to their baseline state at the beginning of the trial. Participants self-rated their perceived change by completing the following statement: "Since I began trial treatment, my overall status is:" 1=Very much improved, 2=Much improved, 3=Minimally improved, 4=No change, 5=Minimally worse, 6=Much worse and 7=Very much worse. Lower score indicates better improvement. The categories with at least one participant having a PGI-C score are reported. |
| Secondary | Part 2: Number of Participants With Clinical Global Impression of Change (CGI-C) | Week 48 | The CGIC scale is a 7 point scale that requires the clinician to assess how much the participant's illness has improved or worsened relative to a baseline state at the beginning of an intervention. The CGIC was assessed by completing the following statement "Compared to the participant's condition at the start of the trial, this participant's overall status is", where 1=very much improved, 2=much improved, 3=minimally improved, 4=no change, 5=minimally worse, 6=much worse, 7=very much worse. Lower score indicates better improvement. The categories with at least one participant having a CGIC score are reported. |
| Secondary | Part 2: Change From Baseline in Performance on a 9-Hole Peg Test (9-HPT) at Week 48 | Baseline, Week 48 | The 9-HPT is a brief, standardized, quantitative test of upper extremity (arm and hand) function. Both the dominant and nondominant hands were tested twice (2 consecutive trials of the dominant hand, followed immediately by 2 consecutive trials of the nondominant hand). The participant picks up pegs 1 at a time (9 in total), using 1 hand only, and places them into holes on the board as quickly as possible, in any order until all holes are filled. Then, without pausing, the participant removes the pegs 1 at a time and returns them as quickly as possible. The times recorded for the two trials for each hand are averaged, and the reciprocal average value for each hand was used in analyses. Longer test times reflect more impairment on the participant's upper extremity function, thus a negative change from baseline suggests an improvement. |
| Secondary | Part 2: Change From Baseline in Performance on a 25-Foot Timed Walk Test (T25-FWT) at Week 48 | Baseline, Week 48 | The T25-FWT is a quantitative mobility and leg function performance test based on time (in seconds) to complete a 25-foot walk. Participants were instructed to attempt two T25-FWT trials at each visit. Both walk times were averaged, and the reciprocal average value was used in analyses. Longer test times reflect more impairment on the participant's ability to walk, thus a negative change from baseline suggests an improvement. |
| Secondary | Part 2: Total Number of Falls | Up to Week 48 | A fall was defined as "the participant unintentionally coming to rest on the ground or at a lower level." The total number of falls before and after study drug administration has been reported. |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 registered outcomesOutcomes3
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in Peak Work (in Watts/kg) During Exercise Testing at Week 12 in Part 1 | Baseline through 12 weeks after participant receives the first dose in Part 1. | All randomized patients in Part 1, whether or not they received study drug | W/kg | LEAST_SQUARES_MEAN |
| PRIMARY | Change in the Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 48 in Part 2 | 48 weeks after participant receives the first dose in Part 2 | Full analysis set (all patients in Part 2 randomized without pes cavus who have at least one post-baseline measurement) | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change in the Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 12 in Part 1 | 12 weeks after participant receives the first dose in Part 1 | All randomized patients in Part 1, whether or not they received study drug | score on a scale | LEAST_SQUARES_MEAN |
Source ClinicalTrials.gov
Results by group18
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in Peak Work (in Watts/kg) During Exercise Testing at Week 12 in Part 1 | Part 1 Omaveloxolone Capsules 2.5 and 5 mg | 0.14 | 95% Confidence Interval 0.02 to 0.26 | W/kg | |
| Change From Baseline in Peak Work (in Watts/kg) During Exercise Testing at Week 12 in Part 1 | Part 1 Omaveloxolone Capsules 10 mg | 0.07 | 95% Confidence Interval -0.05 to 0.18 | W/kg | |
| Change From Baseline in Peak Work (in Watts/kg) During Exercise Testing at Week 12 in Part 1 | Part 1 Omaveloxolone Capsules 20 mg | -0.09 | 95% Confidence Interval -0.20 to 0.03 | W/kg | |
| Change From Baseline in Peak Work (in Watts/kg) During Exercise Testing at Week 12 in Part 1 | Part 1 Omaveloxolone Capsules 40 mg | 0.06 | 95% Confidence Interval -0.06 to 0.18 | W/kg | |
| Change From Baseline in Peak Work (in Watts/kg) During Exercise Testing at Week 12 in Part 1 | Part 1 Omaveloxolone Capsules 80 mg | 0 | 95% Confidence Interval -0.11 to 0.12 | W/kg | |
| Change From Baseline in Peak Work (in Watts/kg) During Exercise Testing at Week 12 in Part 1 | Part 1 Omaveloxolone Capsules 160 mg | 0.02 | 95% Confidence Interval -0.06 to 0.10 | W/kg | |
| Change From Baseline in Peak Work (in Watts/kg) During Exercise Testing at Week 12 in Part 1 | Part 1 Omaveloxolone Capsules 300 mg | 0.07 | 95% Confidence Interval -0.02 to 0.17 | W/kg | |
| Change From Baseline in Peak Work (in Watts/kg) During Exercise Testing at Week 12 in Part 1 | Part 1 Placebo Capsules | 0.04 | 95% Confidence Interval -0.03 to 0.11 | W/kg | |
| Change in the Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 48 in Part 2 | Part 2 Placebo Capsules | 0.85 | Standard Error 0.640 | score on a scale | |
| Change in the Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 48 in Part 2 | Part 2 Omaveloxolone Capsules 150 mg | -1.55 | Standard Error 0.689 | score on a scale |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 results by groupParticipant flow40
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Part 1 Omaveloxolone Capsules 2.5 and 5 mg | 6 | |
| Overall Study | STARTED | Part 1 Omaveloxolone Capsules 10 mg | 6 | |
| Overall Study | STARTED | Part 1 Omaveloxolone Capsules 20 mg | 6 | |
| Overall Study | STARTED | Part 1 Omaveloxolone Capsules 40 mg | 6 | |
| Overall Study | STARTED | Part 1 Omaveloxolone Capsules 80 mg | 6 | |
| Overall Study | STARTED | Part 1 Omaveloxolone Capsules 160 mg | 12 | |
| Overall Study | STARTED | Part 1 Omaveloxolone Capsules 300 mg | 10 | |
| Overall Study | STARTED | Part 1 Placebo Capsules | 17 | |
| Overall Study | STARTED | Part 2 Placebo Capsules | 52 | |
| Overall Study | STARTED | Part 2 Omaveloxolone Capsules 150 mg | 51 |
1 to 10 of 40Source ClinicalTrials.gov
See all 40 participant flowAdverse events1,450
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 | Lymphadenopathy | Blood and lymphatic system disorders | Part 1 Omaveloxolone Capsules 2.5 and 5 mg | 0 | 6 | 0 |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Part 1 Omaveloxolone Capsules 10 mg | 0 | 6 | 0 |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Part 1 Omaveloxolone Capsules 20 mg | 0 | 6 | 0 |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Part 1 Omaveloxolone Capsules 40 mg | 0 | 6 | 0 |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Part 1 Omaveloxolone Capsules 80 mg | 0 | 6 | 0 |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Part 1 Omaveloxolone Capsules 160 mg | 0 | 12 | 0 |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Part 1 Omaveloxolone Capsules 300 mg | 0 | 10 | 0 |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Part 1 Placebo Capsules | 1 | 17 | 1 |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Part 2 Placebo Capsules | 0 | 52 | 0 |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Part 2 Omaveloxolone Capsules 150 mg | 1 | 51 | 1 |
1 to 10 of 1,450Source ClinicalTrials.gov
See all 1,450 adverse eventsAdverse event totals30
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Part 1 Omaveloxolone Capsules 2.5 and 5 mg | 0 | 6 |
| Total, all-cause mortality | Part 1 Omaveloxolone Capsules 10 mg | 0 | 6 |
| Total, all-cause mortality | Part 1 Omaveloxolone Capsules 20 mg | 0 | 6 |
| Total, all-cause mortality | Part 1 Omaveloxolone Capsules 40 mg | 0 | 6 |
| Total, all-cause mortality | Part 1 Omaveloxolone Capsules 80 mg | 0 | 6 |
| Total, all-cause mortality | Part 1 Omaveloxolone Capsules 160 mg | 0 | 12 |
| Total, all-cause mortality | Part 1 Omaveloxolone Capsules 300 mg | 0 | 10 |
| Total, all-cause mortality | Part 1 Placebo Capsules | 0 | 17 |
| Total, all-cause mortality | Part 2 Placebo Capsules | 0 | 52 |
| Total, all-cause mortality | Part 2 Omaveloxolone Capsules 150 mg | 0 | 51 |
1 to 10 of 30Source ClinicalTrials.gov
See all 30 adverse event totalsPapers3
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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