Table 4.
Current clinical trials in RTT individuals.
| Agent | Results | Trial outcome | Sponsor | Type |
|---|---|---|---|---|
| Growth Factors | ||||
| Full length IGF-1 | Phase 2 | Safety confirmed on-going; scheduled through Fall 2015 | Approved drug in children with growth failure | Double-blind; placebo controlled; injection |
| NNZ-2566 [1–3]IGF-1 analogue | Phase 2 | Safety confirmed short-term trial positive | Neuren pharmaceuticals | Double-blind; placebo controlled; oral |
| BDNF Effectors | ||||
| Copaxone | Phase 2 | Improved ambulation | Approved drug | Open-label; injection |
| Fingolimod | Phase 1 | On-going; scheduled through August 2017 | Novartis pharmaceuticals; Approved drug-adults | Open-label; oral |
| NMDA Antagonist | ||||
| Dextromethorphan | Phase 2 | On-going; scheduled through June 2015 | Approved drug in cough suppressant | Double-blind; placebo controlled |
| Norepinephrine Effector | ||||
| Desipramine | Phase 2 | On-going; scheduled through December 2014 | Approved drug in Europe | Double-blind; placebo controlled |
| Mitochondrial Effector | ||||
| EPI-743 | Phase 2 | Safety confirmed Improved head circumference growth | Edison pharmaceuticals | Double-blind; placebo controlled; oral |