Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03214588 | Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich Ataxia | PHASE2 | COMPLETED | 67 | — | — | Nov 8, 2017 | Dec 27, 2018 | Jun 14, 2021 | 6 | United States |
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.
| Arm | Type | Description |
|---|---|---|
| Placebo | PLACEBO_COMPARATOR | TAK-831 placebo-matching tablets, orally, twice daily for up to 12 weeks. |
| TAK-831 75 mg | EXPERIMENTAL | TAK-831 75 mg, tablets, orally, twice daily for up to 12 weeks. |
| TAK-831 300 mg | EXPERIMENTAL | TAK-831 300 mg, tablets, orally, twice daily for up to 12 weeks. |
| Name | Type | Description |
|---|---|---|
| TAK-831 | DRUG | TAK-831 tablets |
| TAK-831 Placebo | DRUG | TAK-831 placebo matching tablets |
Key Inclusion Criteria: 1\. Has a genetically-confirmed diagnosis (homozygous for guanine-adenine-adenine \[GAA\] repeat expansions in the frataxin gene \[FXN\] in the affected range of Friedreich ataxia \[FRDA\] or a compound heterozygous expansion with a point mutation or deletion), with an estab...