We are very excited! Spring is in the air and temperatures are rising. We hope this letter finds you and your families doing well after a long cold winter. There are exciting developments across the universe of Rett Syndrome, and 2019 is shaping up to be the “Year of the Trial” for Rett Syndrome symptom and curative treatments. I want to share just a sample of the activities we are monitoring, and I invite you to read more about them at the links below.
These trials are so important to our loved ones. Full participation is critical to advancing treatments through FDA approval phases. While important, we understand that decisions to participate in trials are very personal family/physician matters, but please know that we are here to help should you have any questions… And stay tuned – we are anticipating a highly important announcement in mid-April that I’m sure will inspire our community to action. Watch your inbox!
Active and anticipated 2019 Rett Syndrome drug trials
STARS-Sarizotan (Newron Pharmaceuticals, active/not recruiting)
- Focused on improving Rett-related breathing episodes
- https://www.rettstars.com/
Low Dose Ketamine (RSRT/Vanderbilt, active/recruiting)
- Focused on relief of Rett-related breathing anomalies and other symptoms
- https://clinicaltrials.gov/ct2/show/NCT03633058
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/
Anavex 2-73 (RettSyndrome.org/Anavex Life Sciences, active/recruiting)
- Focused on improvement of neuronal function
- http://www.rettsyndrometrial.com/
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
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/
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/
Other Events:
Coming very soon! Carolyn Fowler (with husband Stephen) will be hosting An Evening of Hope for Rett Syndrome in honor of their daughter Evelyn in the Jacksonville, Florida area on April 12th. Please join us! And also, don’t forget to check-in with the ladies from Team Sparkle as they are preparing for Ragnar for Rett on April 12th and 13th!
Finally, our redesigned GP2C and RettGirl.org websites are under development, with a goal to deliver dynamic and relevant content to Rett families world-wide. Please reach out to our Family Advocacy and Support team, Jenny Tesler and Carolyn Fowler, with any suggestions you might have! You can reach them at support@gp2c.org, or drop them a note on Facebook or Instagram!
Have a great April!
Kevin, Development Director – Girl Power 2 Cure
