Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
TAK-831 · 5 trials · 3 indications
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.
Bioavailability is defined as the proportion of a drug which enters the circulation when introduced into the body and so is able to have an active effect. Percent absolute bioavailability, calculated for plasma TAK-831 as \[Actual Dose (IV) x AUCinf (oral)\] / \[Actual Dose (oral) x AUCinf (IV)\] x 100.
DAO occupancy is calculated as percent difference between baseline and postdose \[18F\]PGM299 BPND for each participant.
| 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. |
| TAK-831 500 mg + [14C]TAK-831 50 μg + [14C]TAK-831 500 mg | EXPERIMENTAL | TAK-831 500 mg, tablet, orally, once on Day 1, followed by \[14C\]TAK-831 50 micrograms (μg) \[approximately 1 microcurie (μCi)\], infusion, intravenously (IV), once on Day 1 of Treatment Period 1, followed by a washout period of 8 days, further followed by \[14C\]TAK-831 500 mg (approximately 100 μCi), suspension, orally, once under fasted state on Day 1 of Treatment Period 2. |
| Japanese Cohort 1-A; TAK-831 100 mg + TAK-831 300 mg | EXPERIMENTAL | TAK-831 100 milligrams (mg), tablets, orally, once daily on Day 1, followed by TAK-831 300 mg, tablets, orally, once daily on Day 9 in healthy Japanese participants. |
| Japanese Cohort 1-B; TAK-831 100 mg + Placebo | EXPERIMENTAL | TAK-831 100 mg, tablets, orally, once daily on Day 1 followed by TAK-831 matching placebo, tablets, orally, once daily on Day 9 in healthy Japanese participants. |
| Japanese Cohort 1-C; Placebo + TAK-831 300 mg | EXPERIMENTAL | TAK-831 matching placebo, tablets, orally, once daily on Day 1 followed by TAK-831 300 mg, tablets, orally, once daily on Day 9 in healthy Japanese participants. |
| Japanese Cohort 2; TAK-831 300 mg | EXPERIMENTAL | TAK-831 300 mg or TAK-831 matching placebo, tablets, orally, once daily on Day 1 followed by TAK-831 300 mg or TAK-831 matching placebo, tablets, orally, once daily from Day 4 to Day 17 in healthy Japanese participants. |
| Chinese Cohort 3; TAK-831 600 mg | EXPERIMENTAL | TAK-831 600 mg or TAK-831 matching placebo, tablets, orally, once daily on Day 1 followed by TAK-831 600 mg or TAK-831 matching placebo, tablets, orally, once daily from Day 4 to Day 17 in healthy Chinese participants. This cohort is optional and will be decided to run based on the data of Cohorts 1 and 2. The dose will be defined based on the result of Cohort 1 or Cohort 2. |
| Japanese Cohort 4; TAK-831 600 mg | EXPERIMENTAL | TAK-831 600 mg or TAK-831 matching placebo, tablets, orally, once daily on Day 1 followed by TAK-831 600 mg or TAK-831 matching placebo, tablets, orally, once daily from Day 4 to Day 17 in healthy Japanese participants. This cohort is optional and will be decided to run based on the data of Cohorts 1 and 2. The dose will be defined based on the result of Cohort 1 or Cohort 2. |
| Japanese Cohort 5; TAK-831 | EXPERIMENTAL | TAK-831 50 mg or TAK-831 matching placebo, tablets, orally, once daily on Day 1 followed by TAK-831 50 mg or TAK-831 matching placebo, tablets, orally, once daily from Day 4 to Day 17 in healthy Japanese participants. |
| TAK-831 400 mg Fasted + TAK-831 400 mg Fed | EXPERIMENTAL | TAK-831 400 mg, film-coated tablets, orally under fasted state, once on Day 1 of Intervention Period 1, followed by 7 days washout period, further followed by TAK-831 400 mg, film-coated tablets, orally under fed state, once on Day 1 of Intervention Period 2. |
| TAK-831 400 mg Fed + TAK-831 400 mg Fasted | EXPERIMENTAL | TAK-831 400 mg, film-coated tablets, orally under fed state, once on Day 1 of Intervention Period 1, followed by 7 days washout period, further followed by TAK-831 400 mg, film-coated tablets, orally under fasted state, once on Day 1 of Intervention Period 2. |
| Set A: TAK-831 100 mg | EXPERIMENTAL | TAK-831 100 milligram (mg), suspension, orally, once on Day 1 and up to 100 megabecquerel (MBq) of Positron Emission Tomography (PET) ligand PGM028299 labeled with \[18F\] (\[18F\]PGM299) with a maximal mass up to 12.5 microgram (mcg), injection, intravenously, prior to PET imaging at Baseline, 2 hours and 26 hours post-TAK-831 dose. |
| Set A: TAK-831 200 mg | EXPERIMENTAL | TAK-831 200 mg, suspension, orally, once on Day 1 and up to 100 MBq of \[18F\]PGM299 with a maximal mass up to 12.5 mcg, injection, intravenously, prior to PET imaging at Baseline, 2 hours and 26 hours post-TAK-831 dose. |
| Set A: TAK-831 250 mg | EXPERIMENTAL | TAK-831 250 mg, suspension, orally, once on Day 1 and up to 100 MBq of \[18F\]PGM299 with a maximal mass up to 12.5 mcg, injection, intravenously, prior to PET imaging at Baseline, 2 hours and 26 hours post-TAK-831 dose. |
| Set A: TAK-831 500 mg | EXPERIMENTAL | TAK-831 500 mg, suspension, orally, once on Day 1 and up to 100 MBq of \[18F\]PGM299 with a maximal mass up to 12.5 mcg, injection, intravenously, prior to PET imaging at Baseline, 2 hours and 26 hours post-TAK-831 dose. |
| Set B: [18F]PGM299 | EXPERIMENTAL | \[18F\]PGM299 up to 100 MBq (with a maximal mass up to 12.5 mcg), injection, intravenously, prior to PET imaging on Days 1 and 10. |
| Name | Type | Description |
|---|---|---|
| TAK-831 | DRUG | TAK-831 tablets |
| TAK-831 Placebo | DRUG | TAK-831 placebo matching tablets |
| TAK-831 Oral Tablet | DRUG | TAK-831 tablet. |
| [14C]TAK-831 IV Infusion | DRUG | \[14C\]TAK-831 IV infusion. |
| [14C]TAK-831 Oral Suspension | DRUG | \[14C\]TAK-831 oral suspension. |
| Placebo | DRUG | TAK-831 Matching Placebo Tablets. |
| [18F]PGM299 | DRUG | \[18F\]PGM299 injection |
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...
TAK-831 is an investigational small molecule being studied for the treatment of Friedreich Ataxia, a rare inherited disease that affects the nervous system. It has also been evaluated in healthy volunteers in early-phase clinical trials to assess its safety, tolerability, and pharmacokinetics.
TAK-831 is being developed by Neurocrine Biosciences, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol NBIX. The company is conducting clinical trials of TAK-831 in the United States and Japan for Friedreich Ataxia and in healthy volunteer studies.
TAK-831 is in Phase 2 clinical development for Friedreich Ataxia. A Phase 2 trial evaluating its efficacy, tolerability, and pharmacokinetics in adults with Friedreich Ataxia has been completed. The drug is investigational and has not been approved by regulatory authorities.
TAK-831 has been studied in several completed clinical trials. NCT03214588 is a Phase 2 trial in adults with Friedreich Ataxia. NCT03101293 and NCT03687684 are Phase 1 trials in healthy volunteers in the United States and Japan, respectively. NCT04234672 is a Phase 1 study in healthy male participants.
TAK-831 is not FDA approved. It is an investigational drug that has completed Phase 1 and Phase 2 clinical trials, but it remains in clinical development and has not received marketing authorization from the U.S. Food and Drug Administration or any other regulatory agency.