Table 2.
Active clinical trials for oral medications for treatment of Tourette syndrome.
| Agent | Mechanism | Trial ID | Phase | Target enrollment | Ages (years) | Treatment period (weeks) | Groups | Primary outcome |
|---|---|---|---|---|---|---|---|---|
| Ecopipam | D1 antagonist | D1AMOND (NCT04007991) | 2b | 150 | 6–17 | 12 | Treatment vs placebo (1:1) | YGTSS |
| Deutetrabenazine | VMAT2 inhibitor | ARTISTS1 (NCT03452943) | 2/3 | 116 | 6–16 | 12 | Treatment vs placebo (1:1) | YGTSS |
| ARTISTS2 (NCT03571256) | 3 | 150 | 6–16 | 8 | Low dose and high dose vs placebo (1:1:1) | YGTSS | ||
| ARTISTS (NCT03567291) | 3 | 227 | 6–17 | 55 | OL with 2 week randomized withdrawal | YGTSS | ||
| Valbenazine | VMAT2 inhibitor | NCT03444038 | 2 | 85 | 6–18 | 24 | OL | YGTSS |
| NCT03530293 | 2 | 81 | 6–17 | 36 | OL (12 weeks) then treatment vs placebo (24 weeks) | YGTSS | ||
| NCT03325010 | 2 | 127 | 6–17 | 12 | Treatment vs placebo | YGTSS | ||
| NCT02679079 | 2 | 98 | 6–17 | 6 | Dose 1 vs Dose 2 vs placebo | YGTSS | ||
| NCT02581865 | 2 | 124 | 18–64 | 8 | Dose 1 vs Dose 2 vs placebo | YGTSS | ||
| NCT02879578 | 2 | 155 | 6–64 | 155 | OL | YGTSS | ||
| Aripiprazole (oral solution) | D2 antagonist | NCT03487783 | 3 | 120 | 6–17 | 8 | Treatment vs placebo (1:1) | YGTSS |
| ABX-1431 | MGLL inhibitor | NCT03625453 | 2 | 48 | 18–64 | 8 (+4 OL) | Treatment vs placebo (1:1) 4 week OL extension | YGTSS |
| Ondansetron | 5-HT3 antagonist | NCT03239210 | 4 | 60 (OCD and tics) | 18–60 | 4 | Treatment vs placebo (1:1) | Sensory phenomena scale |
| Pimavanserin | 5-HT2A inverse agonist | Pilot | 20 | >18 | 8 | OL | YGTSS | |
| THC/CBD | THC/CBD | NCT03247244 | 2 | 12 | 18–65 | Crossover (3 agents with different THC/CBD ratios and placebo) | mRVRS | |
| Nabiximols | THC/CBD | CANNA-TICS (NCT03087201) | 3 | 96 | >18 | 13 | Treatment vs placebo (1:1) | YGTSS |
| Yi-Gan San | NCT03564132 | 2 | 154 | 6–17 | 4 | Treatment vs placebo (1:1) | YGTSS |
Legend: YGTSS = Yale Global Tic Severity Score, VMAT2 = vesicular monoamine transporter 2, MGLL = monacylgylcerol lipase, THC = tetrahydrocannabinol, CBD = cannabidiol, mRVRS = modified Rush Video-based Tic Rating Scale, OL = open-label.