Trial · NCT03214588
Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich Ataxia
NCT03214588, Phase 2, Completed, Neurocrine Biosciences; results first posted 2019-12-19
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich Ataxia. Trials, record NCT03214588. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03214588
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich Ataxia
- NCT number
- NCT03214588
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 67 participants, actual
- Lead sponsor
- Neurocrine Biosciences
- Conditions
- Friedreich Ataxia
- Interventions
- TAK-831, TAK-831 Placebo
- First posted
- 2017-07-11
- Last updated
- 2021-06-14
- Results first posted
- 2019-12-19
Source ClinicalTrials.gov
Registered outcomes20
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in the Inverse Time to Complete the 9-Hole Peg Test (9-HPT-1) | Baseline and Week 12 | The 9-HPT-1 is a measure of timed upper extremity (arm and hand) function and manual dexterity. 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. Each participant performs this task twice with each hand separately. Results on both tests are then averaged for an overall task completion time and the inverse transform is performed. A positive change from Baseline indicates improvement. Change from Baseline in 9-HPT-1 was analyzed using mixed model for repeated measures (MMRM) analysis of covariance (ANCOVA) with Baseline 9-HPT-1 as a covariate; pooled site, visit, treatment, and ambulation status (randomization factor) as fixed factors; and treatment-by-visit and Baseline 9-HPT-1-by-visit interactions. |
| Secondary | Change From Baseline in the Activities of Daily Living (ADL) Component Score of the Friedreich Ataxia Rating Scale (FARS) | Baseline and Weeks 2, 7 and 12 | The ADL component of the FARS includes 9 subscales: speech, swallowing, cutting food and handling utensils, dressing, personal hygiene, falling, walking, quality of sitting position, and bladder function. Each of these subscales is rated on a 5-point scale where 0=normal to 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. Change from Baseline in FARS ADL upper limb function items were analyzed using MMRM ANCOVA with Baseline FARS ADL as a covariate; pooled site, visit, treatment, and ambulation status (randomization factor) as fixed factors; and treatment-by-visit and Baseline FARS ADL-by-visit interactions. |
| Secondary | Change From Baseline in the Inverse Time to Complete the 9-HPT-1 | Baseline and Weeks 2 and 7 | The 9-HPT-1 is a measure of timed upper extremity (arm and hand) function and manual dexterity. 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. Each participant performs this task twice with each hand separately. Results on both tests are then averaged for an overall task completion time, and the inverse transform is performed. A positive change from Baseline indicates improvement. Change from Baseline in 9-HPT-1 was analyzed using MMRM ANCOVA with Baseline 9-HPT-1 as a covariate; pooled site, visit, treatment, and ambulation status (randomization factor) as fixed factors; and treatment-by-visit and Baseline 9-HPT-1-by-visit interactions. |
| Secondary | Change From Baseline in the ADL Component Individual Item Scores | Baseline and Weeks 2, 7 and 12 | The ADL component of the FARS includes 9 subscales: speech, swallowing, cutting food and handling utensils, dressing, personal hygiene, falling, walking, quality of sitting position, and bladder function. Each of these subscales is rated on a 5-point scale where 0=normal to 4=severe disability/inability to carry out activity independently. A negative change from Baseline indicates improvement. Statistical analyses were available for the following subscales: cutting food-handling utensils, dressing and personal hygiene. |
| Secondary | Change From Baseline in the Modified Friedreich Ataxia Rating Scale Neurological Examination (mFARS-neuro) Total Score | Baseline and Weeks 2, 7 and 12 | The mFARS-neuro neurological examination is a clinician-rated measure based on neural substrates affected in Friedreich ataxia including: bulbar on a scale of 0-11, upper limb coordination on a scale of 0-36, lower limb coordination on a scale of 0-16, and upright stability/gait functions on a scale of 0-36 for a total possible score of 0 to 99 with higher scores representing greater disability. A negative change from Baseline indicates improvement. Change from Baseline in mFARS-neuro was analyzed using MMRM ANCOVA with Baseline mFARS-neuro as a covariate; pooled site, visit, treatment, and ambulation status (randomization factor) as fixed factors; and treatment-by-visit and Baseline mFARS-neuro-by-visit interactions. |
| Secondary | Change From Baseline in the mFARS-neuro Subscales Scores | Baseline and Weeks 2, 7, and 12 | The mFARS-neuro neurological examination is a clinician-rated measure based on neural substrates affected in Friedreich ataxia including: bulbar on a scale of 0-11, upper limb coordination on a scale of 0-36, lower limb coordination on a scale of 0-16, and upright stability/gait functions on a scale of 0-36, with the higher scores representing greater disability. A negative change from Baseline indicates improvement. Change from Baseline in mFARS-neuro was analyzed using MMRM ANCOVA with Baseline mFARS-neuro as a covariate; pooled site, visit, treatment, and ambulation status (randomization factor) as fixed factors; and treatment-by-visit and Baseline mFARS-neuro-by visit interactions. |
| Secondary | Change From Baseline in the mFARS-neuro Individual Item Scores | Baseline and Weeks 2, 7, and 12 | mFARS-neuro examination is a clinician-rated measure based on neural substrates affected in Friedreich ataxia individual items:Cough,Speech,Right(R)Finger to Finger Test,Left(L)Finger to Finger Test,R-Nose to Finger Test,L-Nose to Finger Test,R-Dysmetria Test,L-Dysmetria Test,Rapid Alternating Movement(RAM)of R-Hands,RAM of L-Hands,R-Finger Taps(FT),L-FT,R-Heel Along Shin Slide,L-Heel Along Shin Slide,R-Heel Along Shin Tap,L-Heel Along Shin Tap,Siting Posture,Stance Feet Apart(SFA)-3 Trial Average(TTA),SFA(Eyes Closed)-TTA,Stance Feet Together(SFT)-TTA,SFT(Eyes Closed)-TTA,Tandem Stance-TTA,Stance on Dominant Foot-TTA,Tandem Walk and Gait.Items were scored on scale of 0 to 2,3,4 or 5,with higher scores indicating greater disability.Negative change from Baseline(BL)indicates improvement.Change from BL in mFARS-neuro was analyzed using MMRM ANCOVA with BL as covariate;pooled site,visit,treatment,ambulation status as fixed factors;treatment-by-visit,BL mFARS-neuro-by visit interactions. |
| Secondary | Change From Baseline in the Timed 25-Foot Walk (T25FW) | Baseline and Weeks 2, 7 and 12 | The participant was instructed to walk 25 feet as quickly as possible, but safely. The time was calculated from the initiation of the instruction to start and ends when the participant has reached the 25-foot mark. The task was immediately administered again by having the participant walk back the same distance. The two trials were averaged. A negative change from Baseline indicates improvement. Change from Baseline in T25FW was analyzed using MMRM ANCOVA with Baseline T25FW as a covariate; pooled site, visit, treatment, and ambulation status (randomization factor) as fixed factors; and treatment-by-visit and Baseline T25FW-by-visit interactions. |
| Secondary | Change From Baseline in the 9-HPT and T25FW Composite Score | Baseline and Weeks 2, 7, and 12 | 9-HPT and T25FW were evaluated together as a performance-based composite measure. The inverse transform of each score was computed. The inverse scores from each test were tabulated and converted to test-specific Z scores by subtracting the cohort mean from the raw score, and then dividing by the cohort standard deviation (SD) to create a Z score for the test. The composite Z scores were created by subtracting Z-score for T25FW from the Z-score for 9-HPT-1. A larger Z-score represents a better outcome. A positive change from Baseline indicates improvement. Change from Baseline in composite score was analyzed using MMRM ANCOVA with Baseline composite score as a covariate; pooled site, visit, treatment, and ambulation status (randomization factor) as fixed factors; and treatment-by-visit and Baseline composite score-by-visit interactions. |
| Secondary | Change From Baseline in Low-Contrast Letter Acuity (LCLA) Test Score | Baseline and Weeks 2, 7, and, 12 | The LCLA test assessed visual function in both eyes using the Low-Contrast Sloan Letter Charts at different contrast levels. The score ranged from 0 to 70, where 0=worst visual functioning and 70=best visual functioning. A positive change from Baseline indicates improvement. The change from Baseline in LCLA was analyzed using MMRM ANCOVA with Baseline LCLA as a covariate; pooled site, visit, treatment, and ambulation status (randomization factor) as fixed factors; and treatment-by-visit and Baseline LCLA-by-visit interactions. |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 registered outcomesOutcomes20
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 the Inverse Time to Complete the 9-Hole Peg Test (9-HPT-1) | Baseline and Week 12 | Full analysis set (FAS) included all randomized participant who received at least 1 dose of the study drug for the treatment period. Number analyzed is the number of participants with data available for analysis at the given time-point. | 1/seconds | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in the Activities of Daily Living (ADL) Component Score of the Friedreich Ataxia Rating Scale (FARS) | Baseline and Weeks 2, 7 and 12 | FAS included all randomized participant who received at least 1 dose of the study drug for the treatment period. Number analyzed is the number of participants with data available for analysis at the given time-point. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in the Inverse Time to Complete the 9-HPT-1 | Baseline and Weeks 2 and 7 | FAS included all randomized participant who received at least 1 dose of the study drug for the treatment period. Number analyzed is the number of participants with data available for analysis at the given time-point. | 1/seconds | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in the ADL Component Individual Item Scores | Baseline and Weeks 2, 7 and 12 | FAS included all randomized participant who received at least 1 dose of the study drug for the treatment period. Number analyzed is the number of participants with data available for analysis at the given time-point. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in the Modified Friedreich Ataxia Rating Scale Neurological Examination (mFARS-neuro) Total Score | Baseline and Weeks 2, 7 and 12 | FAS included all randomized participant who received at least 1 dose of the study drug for the treatment period. Number analyzed is the number of participants with data available for analysis at the given time-point. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in the mFARS-neuro Subscales Scores | Baseline and Weeks 2, 7, and 12 | FAS included all randomized participant who received at least 1 dose of the study drug for the treatment period. Number analyzed is the number of participants with data available for analysis at the given time-point. | unit on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in the mFARS-neuro Individual Item Scores | Baseline and Weeks 2, 7, and 12 | FAS included all randomized participant who received at least 1 dose of the study drug for the treatment period. Number analyzed is the number of participants with data available for analysis at the given time-point. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in the Timed 25-Foot Walk (T25FW) | Baseline and Weeks 2, 7 and 12 | FAS included all randomized participant who received at least 1 dose of the study drug for the treatment period. Number analyzed is the number of participants with data available for analysis at the given time-point. | seconds | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in the 9-HPT and T25FW Composite Score | Baseline and Weeks 2, 7, and 12 | FAS included all randomized participant who received at least 1 dose of the study drug for the treatment period. Number analyzed is the number of participants with data available for analysis at the given time-point. | z-score | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in Low-Contrast Letter Acuity (LCLA) Test Score | Baseline and Weeks 2, 7, and, 12 | FAS included all randomized participant who received at least 1 dose of the study drug for the treatment period. Number analyzed is the number of participants with data available for analysis at the given time-point. | score on a scale | LEAST_SQUARES_MEAN |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 outcomesResults by group1,122
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 the Inverse Time to Complete the 9-Hole Peg Test (9-HPT-1) | Placebo | 0.00038 | Standard Error 0.000448 | 1/seconds | |
| Change From Baseline in the Inverse Time to Complete the 9-Hole Peg Test (9-HPT-1) | TAK-831 75 mg | -0.00017 | Standard Error 0.000622 | 1/seconds | |
| Change From Baseline in the Inverse Time to Complete the 9-Hole Peg Test (9-HPT-1) | TAK-831 300 mg | -0.00032 | Standard Error 0.000445 | 1/seconds | |
| Change From Baseline in the Activities of Daily Living (ADL) Component Score of the Friedreich Ataxia Rating Scale (FARS) | Placebo | Change from Baseline at Week 2 | -0.25 | Standard Error 0.468 | score on a scale |
| Change From Baseline in the Activities of Daily Living (ADL) Component Score of the Friedreich Ataxia Rating Scale (FARS) | TAK-831 75 mg | Change from Baseline at Week 2 | -1.12 | Standard Error 0.665 | score on a scale |
| Change From Baseline in the Activities of Daily Living (ADL) Component Score of the Friedreich Ataxia Rating Scale (FARS) | TAK-831 300 mg | Change from Baseline at Week 2 | 0.34 | Standard Error 0.463 | score on a scale |
| Change From Baseline in the Activities of Daily Living (ADL) Component Score of the Friedreich Ataxia Rating Scale (FARS) | Placebo | Change from Baseline at Week 7 | -0.33 | Standard Error 0.465 | score on a scale |
| Change From Baseline in the Activities of Daily Living (ADL) Component Score of the Friedreich Ataxia Rating Scale (FARS) | TAK-831 75 mg | Change from Baseline at Week 7 | -0.15 | Standard Error 0.655 | score on a scale |
| Change From Baseline in the Activities of Daily Living (ADL) Component Score of the Friedreich Ataxia Rating Scale (FARS) | TAK-831 300 mg | Change from Baseline at Week 7 | 0.03 | Standard Error 0.467 | score on a scale |
| Change From Baseline in the Activities of Daily Living (ADL) Component Score of the Friedreich Ataxia Rating Scale (FARS) | Placebo | Change from Baseline at Week 12 | -0.61 | Standard Error 0.493 | score on a scale |
1 to 10 of 1,122Source ClinicalTrials.gov
See all 1,122 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Placebo | 27 | |
| Overall Study | STARTED | TAK-831 75 mg | 14 | |
| Overall Study | STARTED | TAK-831 300 mg | 26 | |
| Overall Study | COMPLETED | Placebo | 26 | |
| Overall Study | COMPLETED | TAK-831 75 mg | 13 | |
| Overall Study | COMPLETED | TAK-831 300 mg | 24 | |
| Overall Study | NOT COMPLETED | Placebo | 1 | |
| Overall Study | NOT COMPLETED | TAK-831 75 mg | 1 | |
| Overall Study | NOT COMPLETED | TAK-831 300 mg | 2 |
Source ClinicalTrials.gov
Adverse events144
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 | Angina pectoris | Cardiac disorders | Placebo | 0 | 27 | |
| other | Angina pectoris | Cardiac disorders | TAK-831 75 mg | 1 | 14 | |
| other | Angina pectoris | Cardiac disorders | TAK-831 300 mg | 0 | 26 | |
| other | Vertigo | Ear and labyrinth disorders | Placebo | 1 | 27 | |
| other | Vertigo | Ear and labyrinth disorders | TAK-831 75 mg | 1 | 14 | |
| other | Vertigo | Ear and labyrinth disorders | TAK-831 300 mg | 0 | 26 | |
| other | Altered visual depth perception | Eye disorders | Placebo | 0 | 27 | |
| other | Altered visual depth perception | Eye disorders | TAK-831 75 mg | 1 | 14 | |
| other | Altered visual depth perception | Eye disorders | TAK-831 300 mg | 0 | 26 | |
| other | Nausea | Gastrointestinal disorders | Placebo | 4 | 27 |
1 to 10 of 144Source ClinicalTrials.gov
See all 144 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Placebo | 0 | 27 |
| Total, all-cause mortality | TAK-831 75 mg | 0 | 14 |
| Total, all-cause mortality | TAK-831 300 mg | 0 | 26 |
| Total, other adverse events | Placebo | 21 | 27 |
| Total, other adverse events | TAK-831 75 mg | 10 | 14 |
| Total, other adverse events | TAK-831 300 mg | 24 | 26 |
| Total, serious adverse events | Placebo | 0 | 27 |
| Total, serious adverse events | TAK-831 75 mg | 0 | 14 |
| Total, serious adverse events | TAK-831 300 mg | 0 | 26 |
Source ClinicalTrials.gov
Papers0
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.
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