Active and Anticipated 2019 Rett Syndrome Drug Trials


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

Focused on improving Rett-related breathing episodes

https://www.rettstars.com/

 

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

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

https://clinicaltrials.gov/ct2/show/NCT03633058

 

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

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

https://reverserett.org/research/project/triheptanoin/

 

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

Focused on improvement of neuronal function

http://www.rettsyndrometrial.com/

 

5.  Safety of Cannabidiol Solution in Rett Syndrome (RettSyndrome.org/GW 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

https://clinicaltrials.gov/ct2/show/NCT03848832

https://www.rettsyndrome.org/file/19---downloadable-docs/GWND18064-External-Communication_Final-28Feb19.pdf

 

6.  Trofinetide Phase 3 (RettSyndrome.org/Acadia-Neuren, anticipated)

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

https://www.rettsyndrome.org/file/18-research-files/Trofinetide-Phase-3-Clinical-Study-Update_12.20.18.pdf

http://www.acadia-pharm.com/pipeline/rett-syndrome/

 

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

Cure-focused MECP2 gene therapy

https://reverserett.org/avexis-reports-rett-gene-therapy-program-avxs-201/

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— Rachel Dalton