Table 2.
Drugs in clinical trials of fragile X syndrome.
| Drugs | Action | Study designs | Publications |
|---|---|---|---|
| Fenobam | mGluR5 antagonist | Open label pilot study; single dose | Berry-Kravis et al. (2009) |
| AFQ056 | mGluR5 antagonist | Randomized, double-blind, two treatment, two-period crossover study | Jacquemont et al. (2011) |
| RO4917523 | mGluR5 antagonist | Randomized, double-blinded, placebo-controlled study | Discontinued due to negative results† |
| Ganaxolone | GABAA receptor agonist | Double-blind, controlled trial | www.clinicaltrials.gov |
| Acamprosate | GABA receptor agonist | Open-label, uncontrolled trial | Erickson et al. (2010, 2013) |
| Arbaclofen (STX209) | GABAB receptor agonist | Randomized, double-blind, placebo-controlled study | Berry-Kravis et al. (2012) |
| Oxytocin | OXTR agonist | Double-blind, placebo-controlled study | Hall et al. (2012) |
| CX516 | AMPAR positive modulator | Randomized, double-blind, placebo-controlled study | Berry-Kravis et al. (2006) |
| Memantine | NMDAR antagonist | Open-label study | Erickson et al. (2009) |
| Lovastatin | ERK inhibitor | Open-label study | Çaku et al. (2014) |
| Lithium | GSK3 inhibitor | Open-label study | Berry-Kravis et al. (2008) |
| Minocycline | MMP9 inhibitor | Open-label add-on pilot trial | Paribello et al. (2010) |
| Randomized, double-blind, placebo-controlled and crossover trial | Leigh et al. (2013) |