Pediatric Phase 2

From RettSyndrome.org

http://www.rettsyndrome.org/blog/15...c-Phase-2-Clinical-Trial-Study-of-Trofinetide


More details are being released from Neuren Pharmaceuticals in regards to the new pediatric Phase 2 clinical trial with trofinetide. We are now learning how the clinical trial will be conducted. Read our latest blog post to learn more about the upcoming Phase 2 trial.

New Details of the Pediatric Phase 2 Clinical Trial Study of Trofinetide

December 8, 2015
Neuren Pharmaceuticals is preparing a new Phase 2 clinical trial with trofinetide in a pediatric population with Rett syndrome. This will be their second study in Rett syndrome and supplements the information from the first trial conducted in adolescent and adult women with Rett syndrome. The age range for the new trial will be from 5 to 15 years old.

This new Phase 2 trial will confirm the tolerability and safety of trofinetide in children and will be informative with respect to the planning of a single Phase 3 trial (a pivotal trial) that will include children, adolescents and adults. The new Phase 2 trial will examine higher dose levels than the first trial, the results of which showed trends of increasing clinical benefit with increasing dose levels.

The pediatric trial is a dose-ranging study looking at three fixed-doses of trofinetide (50, 100 and 200 mg/kg) administered orally twice daily. The study is aiming to enroll approximately 48 individuals, all of whom will receive trofinetide. The primary goal of the study will be to assess the tolerability and safety of the study medication in this younger population.  Additionally, Neuren will assess clinical benefit by determining change from baseline on a number of outcome measures including the clinician-completed Motor Behavior Assessment (MBA), the Clinical Global Impression Scale of Improvement and the Caregiver Top 3 Concerns assessment.  Each of these measures exhibited clinical benefit in the first study.  Several of the outcome measures in the study were developed collaboratively with Drs. Alan Percy, Daniel Glaze and Jeffrey Neul who are experienced PIs from the first Phase 2 trial.

The sites that will conduct the study in the United States, including sites participating in the Rett Natural History Study, are currently being selected and will be announced in due course.  The new study is planned to start in Q1 2016.

As Neuren is working through the study start-up details, we encourage families who are considering this Clinical Trial to once again read "A Participant's Guide to Drug Research in Rett Syndrome". We look forward to sharing more community announcements as they become known.


Comments:

Great news. We're beginning to see a pretty little light.