Trial

From Rettsyndrome.org Facebook site

https://www.rettsyndrome.org/blog/16-14th-rett-syndrome-research-symposium-outcomes

14th Rett Syndrome Research Symposium Outcomes
July 18, 2016
Rettsyndrome.org is pleased to announce we undoubtedly had another successful research symposium to encourage communication and collaboration within the Rett researcher community.  Together, our session chairs put together a program that brought together clinical researchers, academic basic discovery and translational research faculty, pharma and biotech scientists and leaders, students and postdoc trainees, and other medical professionals.  Over 125 attended the research symposium that included 35 individual platform talks and 43 poster presentations.  We could not have asked for more.  The feeling of unity and strength was present during the meeting, and we believe all left with the feeling of hope for the future.  Hope for the treatments and therapies on the horizon for all those touched by Rett syndrome.  Thank you to our session chairs, speakers, attendees, partnerships, board of directors, and most importantly to our families, sponsors and donors.

Advances in Clinical Research & Trials Speaker Jeffrey Neul, MD, PhD speaking at Wednesday’s opening session.“Past, Present and Future: A program to develop and establish Trofinetide as a safe and effective treatment for Rett syndrome”

Dr. Jeffrey Neul, session co-chair of Wednesday’s session, stepped in to speak on behalf of Dr. Daniel Glaze who could not attend the symposium due to the unfortunate storms that delayed flights around Chicago.  Dr. Neul covered the past and current trial of trofinetide (glycyl-L-2-methylprolyl-L-glutamic acid, developed by Neuren Pharmaceuticals LTD), which is an analog of IGF1 [1-3] (glypromate) that shares similar biological properties with this agent. Trofinetide can be given as an oral preparation with improved systemic availability and increased plasma half-life.

Currently Neuren is conducting a clinical trial to examine safety and tolerability (primary outcome measure) in children with Rett syndrome during a multi-center, phase 2 double blinded, randomized placebo controlled clinical trial comparing 56 days of treatment with placebo or one of 3 doses (50, 100, 200 mg/kg twice a day) of Trofinetide.  Anticipated enrollment is 76 female subjects with Rett syndrome and a MECP2 mutation, and between the ages of 5 and 15 years.  Nine (9) of the planned eleven (11) sites are actively enrolling, see rettstudy.com for more details. Upon successful completion of the current study, they anticipate initiation of a phase 3, pivotal clinical study of trofinetide in Rett syndrome.   Neuren is working closely with the FDA to develop primary and secondary efficacy measures.  The results of the first 2 trials will provide informative data regarding these measures, as well as, pharmacokinetics and dosing of trofinetide.

Rettsyndrome.org has supported the first Phase 2 trial in adults with Rett syndrome through an ANGEL grant, and will fund $1M to the current and largest ever clinical trial for Rett syndrome.