Friedreich Ataxia
Conditions
Keywords
RTA 408, RTA 408 Capsules, Oxidative Stress, Mitochondrial dysfunction, omaveloxolone
Brief summary
In this study, researchers are learning more about RTA 408, also known as omaveloxolone, BIIB141, or SKYCLARYS®. The main goal of this study is to learn more about the safety of RTA 408 and how it affects physical effort, movement, coordination, and how participants feel in daily life. The main questions researchers want to answer in this study are: * How much physical effort can a participant produce during a cycling test after 12 weeks of treatment? * How do scores on the modified Friedreich's Ataxia Rating Scale (mFARS) change after 48 weeks? Researchers will use the modified Friedreich's Ataxia Rating Scale (mFARS) to measure how FA affects the nervous system. The mFARS looks at movement ability, balance, coordination, speech, and how well the arms and legs work. They will also use a cycling test to measure physical effort, along with questionnaires to learn how participants feel and function in daily life. Safety will also be tested using physical exams, vital sign checks, echocardiograms (ECHO), electrocardiograms (ECG), and blood and urine tests. The study will be done in 2 main parts, followed by an optional Extension period: * In Part 1, participants will be randomly assigned to take different doses of RTA 408 or a placebo by mouth once a day for 12 weeks. A placebo looks like the study drug but contains no real medicine. * Researchers will compare these doses to decide which one to use in Part 2. * In Part 2, a different group of participants will take either the chosen dose of RTA 408 (150 mg) or placebo once a day for 48 weeks. * Participants who complete Part 1 or Part 2 may be able to join an Extension period, where everyone receives RTA 408. * In the Extension period, participants will continue to receive RTA 408 until the drug becomes commercially available or until they leave the study * Participants in Part 1 will have up to 9 study visits and 2 phone calls. If they do not move onto the Extension period, they will stay in the study for up to 20 weeks. * Participants in Part 2 will have up to 10 study visits and 3 phone calls. If they do not move onto the Extension period, they will stay in the study for up to 61 weeks. * Participants in the Extension period will have 2 visits in the first month, followed by visits every 6 months.
Detailed description
Friedreich's ataxia is an autosomal recessive cerebellar ataxia caused by triplet-repeat expansions. The causative mutation is a trinucleotide (GAA) repeat expansion in the first intron of the frataxin gene, leading to impaired transcription of frataxin. The pathological consequences of frataxin deficiency include a severe disruption of iron-sulfur cluster biosynthesis, mitochondrial iron overload coupled to cellular iron dysregulation, and an increased sensitivity to oxidative stress. A hallmark of Friedreich's ataxia is impairment of antioxidative defense mechanisms, which play a major role in disease progression. Studies have demonstrated that nuclear factor erythroid-derived 2-related factor 2 (Nrf2) signaling is grossly impaired in participants with Friedreich's ataxia. Therefore, the ability of omaveloxolone (RTA 408) to activate Nrf2 and induce antioxidant target genes is hypothesized to be therapeutic in participants with Friedreich's ataxia. This 2-part study will evaluate the efficacy, safety, and pharmacodynamics of omaveloxolone (RTA 408) in the treatment of participants with Friedreich's ataxia. Part 1: The first part of this study will be a randomized, placebo-controlled, double-blind, dose-escalation study to evaluate the safety of omaveloxolone (RTA 408) at various doses in participants with Friedreich's ataxia. Part 2: The second part of this study is a randomized, placebo-controlled, double-blind, parallel-group study to evaluate the safety and efficacy of omaveloxolone (RTA 408) 150 mg in participants with Friedreich's ataxia. Participants enrolled in Part 2 will be randomized 1:1 to receive omaveloxolone (RTA 408) 150 mg or placebo. Extension: The extension will assess long-term safety and tolerability of omaveloxolone (RTA 408) in qualified participants with Friedreich's ataxia following completion of Part 1 or Part 2. Participants will not be unblinded to study treatment in Part 1 or Part 2 upon entering the extension study. Participants will receive open-label omaveloxolone (RTA 408) at 150 mg once daily.
Interventions
DRUGPlacebo
DRUGOmaveloxolone Capsules, 300 mg
DRUGOmaveloxolone Capsules, 150 mg
Sponsors
Biogen
AbbVie
Friedreich's Ataxia Research Alliance
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
16 Years to 40 Years
Healthy volunteers
No
Inclusion criteria
1. Have genetically confirmed Friedreich's ataxia 2. Have a modified FARS score ≥20 and ≤80 3. Be male or female and ≥16 years of age and ≤40 years of age 4. Have no changes to exercise regimen within 30 days prior to Study Day 1 and be willing to remain on the same exercise regimen during the 16-week study period 5. Have the ability to complete maximal exercise testing 6. Be able to swallow capsules
Exclusion criteria
1. Have uncontrolled diabetes (HbA1c \>11.0%) 2. Have B-type natriuretic peptide value \>200 pg/mL 3. Have a history of clinically significant left-sided heart disease and/or clinically significant cardiac disease 4. Have known active fungal, bacterial, and/or viral infection, including human immunodeficiency virus or hepatitis virus (B or C) 5. Have known or suspected active drug or alcohol abuse 6. Have clinically significant abnormalities of clinical hematology or biochemistry, including but not limited to elevations greater than 1.5 times the upper limit of normal of aspartate aminotransferase, or alanine aminotransferase 7. Have any abnormal laboratory test value or serious pre-existing medical condition that, in the opinion of the investigator, would put the patient at risk by study enrollment 8. Have taken any of the following drugs within 7 days prior to Study Day 1 or plan to take any of these drugs during the time of study participation: 1. Sensitive substrates for cytochrome P450 2C8 or 3A4 (e.g., repaglinide, midazolam, sildenafil) 2. Moderate or strong inhibitors or inducers of cytochrome P450 3A4 (e.g., carbamazepine, phenytoin, ciprofloxacin, grapefruit juice) 3. Substrates for p-glycoprotein transporter (e.g., ambrisentan, digoxin) 9. Have participated in any other interventional clinical study within 30 days prior to Study Day 1 10. Have a cognitive impairment that may preclude ability to comply with study procedures 11. Prior participation in a trial with omaveloxolone (RTA 408)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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. |
| 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. |
| 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. |
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
| 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. |
| 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. |
| 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. |
| 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. |
| 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. |
| Part 2: Change From Baseline in Peak Work During Maximal Exercise Testing at Week 48 | Baseline, Week 48 | 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. |
| Part 2: Change From Baseline in the Activities of Daily Living (ADL) Score at Week 48 | Baseline, Week 48 | The ADL scale examines the ability of participants to complete everyday tasks (e.g., holding a fork, dressing). The ADL is a 9-question assessment, with the total score being the sum of 9 questions. The ADL survey assesses 9 concepts: (1) speech; (2) swallowing; (3) cutting food and handling utensils; (4) dressing; (5) personal hygiene; (6) falling; (7) walking; (8) quality of sitting position; and (9) bladder function. Each of these is rated on a 5-point scale where 0= normal and 4= severe disability/inability to carry out activity independently, for a total possible score of 0 to 36, with higher scores representing greater disability/dependency. A negative change from baseline indicates improvement. |
Countries
Australia, Austria, Italy, United Kingdom, United States
Participant flow
Recruitment details
Participants took part in the study at multiple investigative sites from 31 January 2015 to 19 December 2025.
Pre-assignment details
A total of 172 participants diagnosed with Friedreich's Ataxia (FA) were enrolled in this study. The study consists of 3 parts: Part 1, Part 2, and the Open-Label Extension (OLE) period. Out of the 68 participants who completed Part 1 and 96 participants who completed Part 2, 57 participants from Part 1 and 92 participants from Part 2 entered the OLE period, and 105 participants completed the OLE period.
Participants by arm
| Arm | Count |
|---|---|
| Part 1 Omaveloxolone Capsules 2.5 and 5 mg Omaveloxolone (RTA 408) Capsules, 2.5 mg administered orally one daily for 2 weeks, then 5 mg administered orally once daily for 10 weeks
Omaveloxolone Capsules, 2.5 mg
Omaveloxolone Capsules, 5 mg | 6 |
| Part 1 Omaveloxolone Capsules 10 mg Omaveloxolone (RTA 408) Capsules, 10 mg administered orally once daily for 12 weeks
Omaveloxolone Capsules, 10 mg | 6 |
| Part 1 Omaveloxolone Capsules 20 mg Omaveloxolone (RTA 408) Capsules, 20 mg administered orally once daily for 12 weeks
Omaveloxolone Capsules, 20 mg | 6 |
| Part 1 Omaveloxolone Capsules 40 mg Omaveloxolone (RTA 408) Capsules, 40 mg administered orally once daily for 12 weeks
Omaveloxolone Capsules, 40 mg | 6 |
| Part 1 Omaveloxolone Capsules 80 mg Omaveloxolone (RTA 408) Capsules, 80 mg administered orally once daily for 12 weeks
Omaveloxolone Capsules, 80 mg | 6 |
| Part 1 Omaveloxolone Capsules 160 mg Omaveloxolone (RTA 408) Capsules, 160 mg administered orally once daily for 12 weeks
Omaveloxolone Capsules, 160 mg | 12 |
| Part 1 Omaveloxolone Capsules 300 mg Omaveloxolone (RTA 408) Capsules, 300 mg administered orally once daily for 12 weeks
Omaveloxolone Capsules, 300 mg | 10 |
| Part 1 Placebo Capsules Placebo capsules administered orally once daily for 12 weeks
Placebo | 17 |
| Part 2 Placebo Capsules Placebo capsules administered orally once daily for 48 weeks
Placebo | 52 |
| Part 2 Omaveloxolone Capsules 150 mg Omaveloxolone (RTA 408) Capsules, 150 mg administered orally once daily for 48 weeks
Omaveloxolone Capsules, 150 mg | 51 |
| Total | 172 |
Baseline characteristics
| Characteristic | Part 1 Omaveloxolone Capsules 2.5 and 5 mg | Part 1 Omaveloxolone Capsules 10 mg | Part 1 Omaveloxolone Capsules 20 mg | Part 1 Omaveloxolone Capsules 40 mg | Part 1 Omaveloxolone Capsules 80 mg | Part 1 Omaveloxolone Capsules 160 mg | Part 1 Omaveloxolone Capsules 300 mg | Part 1 Placebo Capsules | Part 2 Placebo Capsules | Part 2 Omaveloxolone Capsules 150 mg | Total |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | 25.8 years STANDARD_DEVIATION 5.98 | 25.5 years STANDARD_DEVIATION 7.4 | 28.3 years STANDARD_DEVIATION 6.8 | 27.7 years STANDARD_DEVIATION 7.53 | 24.3 years STANDARD_DEVIATION 4.8 | 25.3 years STANDARD_DEVIATION 6.51 | 25.6 years STANDARD_DEVIATION 7.24 | 24.4 years STANDARD_DEVIATION 6.74 | 23.4 years STANDARD_DEVIATION 6.08 | 24.1 years STANDARD_DEVIATION 7.85 | 24.5 years STANDARD_DEVIATION 6.84 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants | 1 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 1 Participants | 2 Participants | 3 Participants | 7 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 6 Participants | 5 Participants | 6 Participants | 6 Participants | 6 Participants | 12 Participants | 10 Participants | 16 Participants | 49 Participants | 49 Participants | 165 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 1 Participants | 0 Participants | 1 Participants | 1 Participants | 2 Participants | 5 Participants |
| Race (NIH/OMB) White | 6 Participants | 6 Participants | 6 Participants | 6 Participants | 6 Participants | 11 Participants | 10 Participants | 16 Participants | 50 Participants | 50 Participants | 167 Participants |
| Sex: Female, Male Female | 4 Participants | 4 Participants | 5 Participants | 1 Participants | 3 Participants | 5 Participants | 5 Participants | 10 Participants | 31 Participants | 17 Participants | 85 Participants |
| Sex: Female, Male Male | 2 Participants | 2 Participants | 1 Participants | 5 Participants | 3 Participants | 7 Participants | 5 Participants | 7 Participants | 20 Participants | 35 Participants | 87 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk | EG004 affected / at risk | EG005 affected / at risk | EG006 affected / at risk | EG007 affected / at risk | EG008 affected / at risk | EG009 affected / at risk | EG010 affected / at risk | EG011 affected / at risk |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 6 | 0 / 6 | 0 / 6 | 0 / 6 | 0 / 6 | 0 / 12 | 0 / 10 | 0 / 17 | 0 / 51 | 0 / 52 | 0 / 106 | 0 / 43 |
| other Total, other adverse events | 6 / 6 | 5 / 6 | 5 / 6 | 6 / 6 | 6 / 6 | 11 / 12 | 10 / 10 | 16 / 17 | 51 / 51 | 50 / 52 | 103 / 106 | 42 / 43 |
| serious Total, serious adverse events | 0 / 6 | 0 / 6 | 0 / 6 | 0 / 6 | 0 / 6 | 0 / 12 | 0 / 10 | 2 / 17 | 5 / 51 | 3 / 52 | 12 / 106 | 13 / 43 |
Outcome results
Primary
Change From Baseline in Peak Work (in Watts/kg) During Exercise Testing at Week 12 in Part 1
Peak work attained during maximal exercise testing. Cycle ergometry using a recumbent stationary bicycle was used, and workload was increased incrementally. Peak work is defined as the workload at which patients reach maximal volition (defined as an inability to continue to exercise due to exhaustion).
Time frame: Baseline through 12 weeks after participant receives the first dose in Part 1.
Population: All randomized patients in Part 1, whether or not they received study drug
| Arm | Measure | Value (LEAST_SQUARES_MEAN) |
|---|---|---|
| Part 1 Omaveloxolone Capsules 2.5 and 5 mg | Change From Baseline in Peak Work (in Watts/kg) During Exercise Testing at Week 12 in Part 1 | 0.14 W/kg |
| Part 1 Omaveloxolone Capsules 10 mg | Change From Baseline in Peak Work (in Watts/kg) During Exercise Testing at Week 12 in Part 1 | 0.07 W/kg |
| Part 1 Omaveloxolone Capsules 20 mg | Change From Baseline in Peak Work (in Watts/kg) During Exercise Testing at Week 12 in Part 1 | -0.09 W/kg |
| Part 1 Omaveloxolone Capsules 40 mg | Change From Baseline in Peak Work (in Watts/kg) During Exercise Testing at Week 12 in Part 1 | 0.06 W/kg |
| Part 1 Omaveloxolone Capsules 80 mg | Change From Baseline in Peak Work (in Watts/kg) During Exercise Testing at Week 12 in Part 1 | 0 W/kg |
| Part 1 Omaveloxolone Capsules 160 mg | Change From Baseline in Peak Work (in Watts/kg) During Exercise Testing at Week 12 in Part 1 | 0.02 W/kg |
| Part 1 Omaveloxolone Capsules 300 mg | Change From Baseline in Peak Work (in Watts/kg) During Exercise Testing at Week 12 in Part 1 | 0.07 W/kg |
| Part 1 Placebo Capsules | Change From Baseline in Peak Work (in Watts/kg) During Exercise Testing at Week 12 in Part 1 | 0.04 W/kg |
p-value: 0.152495% CI: [-0.04, 0.23]Mixed Models Analysis
p-value: 0.708695% CI: [-0.11, 0.16]Mixed Models Analysis
p-value: 0.065195% CI: [-0.26, 0.01]Mixed Models Analysis
p-value: 0.793795% CI: [-0.12, 0.15]Mixed Models Analysis
p-value: 0.558795% CI: [-0.18, 0.1]Mixed Models Analysis
p-value: 0.728595% CI: [-0.13, 0.09]Mixed Models Analysis
p-value: 0.580295% CI: [-0.09, 0.15]Mixed Models Analysis
Comparison: Comparison of Omaveloxolone capsules pooled with Placebo capsulesp-value: 0.969895% CI: [-0.08, 0.08]Mixed Models Analysis
Primary
Change in the Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 48 in Part 2
The mFARS includes 4 of the 5 sections of the Friedreich's Ataxia Rating Scale (FARS): bulbar (score 0 to 11), upper limb coordination (score 0 to 36), lower limb coordination (score 0 to 16), and upright stability (score 0 to 36). The minimum score is 0 and the maximum score is 99. A lower score indicates better neurological function.
Time frame: 48 weeks after participant receives the first dose in Part 2
Population: Full analysis set (all patients in Part 2 randomized without pes cavus who have at least one post-baseline measurement)
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Part 1 Omaveloxolone Capsules 2.5 and 5 mg | Change in the Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 48 in Part 2 | 0.85 score on a scale | Standard Error 0.64 |
| Part 1 Omaveloxolone Capsules 10 mg | Change in the Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 48 in Part 2 | -1.55 score on a scale | Standard Error 0.689 |
p-value: 0.014195% CI: [-4.31, -0.5]Mixed Models Analysis
Secondary
Change in the Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 12 in Part 1
The mFARS includes 4 of the 5 sections of the Friedreich's Ataxia Rating Scale (FARS): bulbar (score 0 to 11), upper limb coordination (score 0 to 36), lower limb coordination (score 0 to 16), and upright stability (score 0 to 36). The minimum score is 0 and the maximum score is 99. A lower score indicates better neurological function.
Time frame: 12 weeks after participant receives the first dose in Part 1
Population: All randomized patients in Part 1, whether or not they received study drug
| Arm | Measure | Value (LEAST_SQUARES_MEAN) |
|---|---|---|
| Part 1 Omaveloxolone Capsules 2.5 and 5 mg | Change in the Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 12 in Part 1 | -3.26 score on a scale |
| Part 1 Omaveloxolone Capsules 10 mg | Change in the Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 12 in Part 1 | -1.97 score on a scale |
| Part 1 Omaveloxolone Capsules 20 mg | Change in the Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 12 in Part 1 | -2.44 score on a scale |
| Part 1 Omaveloxolone Capsules 40 mg | Change in the Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 12 in Part 1 | -2.4 score on a scale |
| Part 1 Omaveloxolone Capsules 80 mg | Change in the Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 12 in Part 1 | -2.88 score on a scale |
| Part 1 Omaveloxolone Capsules 160 mg | Change in the Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 12 in Part 1 | -3.75 score on a scale |
| Part 1 Omaveloxolone Capsules 300 mg | Change in the Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 12 in Part 1 | -0.88 score on a scale |
| Part 1 Placebo Capsules | Change in the Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 12 in Part 1 | -1.43 score on a scale |
p-value: 0.23195% CI: [-4.8, 1.18]Mixed Models Analysis
p-value: 0.729895% CI: [-3.51, 2.47]Mixed Models Analysis
p-value: 0.510295% CI: [-3.99, 2]Mixed Models Analysis
p-value: 0.528195% CI: [-3.94, 2.04]Mixed Models Analysis
p-value: 0.345895% CI: [-4.42, 1.57]Mixed Models Analysis
p-value: 0.058795% CI: [-4.68, 0.09]Mixed Models Analysis
p-value: 0.64895% CI: [-1.94, 3.1]Mixed Models Analysis
Comparison: Comparison of Omaveloxolone capsules pooled with Placebo capsulesp-value: 0.217495% CI: [-2.87, 0.66]Mixed Models Analysis