Active and Anticipated 2019 Rett Syndrome Drug Trials

1.  STARS-Sarizotan (Newron Pharmaceuticals, active/not recruiting)

Focused on improving Rett-related breathing episodes


2.  Low Dose Ketamine (RSRT/Vanderbilt, active/recruiting)

Focused on relief of Rett-related breathing anomalies and other symptoms


3.  Triheptanoin UX007 (RSRT/Ultragenyx/Center for Rare Neurological Diseases, active/recruiting)

12-person trial focused on chemical reactions that produce mitochondrial energy


4.  Anavex 2-73 ( Life Sciences, active/recruiting)

Focused on improvement of neuronal function


5.  Safety of Cannabidiol Solution in Rett Syndrome ( Research Ltd., active/recruiting)

Focused on evaluating the safety and efficacy of GWP42003-P in reducing symptom severity in Rett Syndrome females, aged 2 to 18 years


6.  Trofinetide Phase 3 (, anticipated)

Focused on improving neuronal signal transmission pathways from/to the brain


7.  AVXS-201 Gene Therapy Phase 1 (RSRT/Novartis-AveXis, anticipated)

Cure-focused MECP2 gene therapy

GP2C is the most inspiring organization I've ever encountered! Not only does it provide funding but it provides support and LOVE!

— Rachel Dalton