NCT05059327

Basimglurant (NOE-101) in Children, Adolescents, and Young Adults With TSC

A Phase 2B, Multicenter, 30-week, Prospective, Cross-over, Double-blind, Randomized, Placebo-controlled Study Followed by a 52-Week Open-label Extension Study to Evaluate the Efficacy and Safety of Basimglurant Adjunctive to Ongoing Anticonvulsive Therapy in Children, Adolescents, and Young Adults With Uncontrolled Seizures Associated With Tuberous Sclerosis Complex

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 61 (actual)
• Sponsor: Noema Pharma AG · Industry
• Sex: All
• Age: 5 Years – 30 Years
• Healthy volunteers: Not accepted

Summary

The study intends to show that basimglurant (NOE-101) provides effective seizure control in children, adolescents and young adults with Tuberous Sclerosis Complex (TSC).

Detailed description

The study drug (NOE-101, basimglurant) is a potent inhibitor of metabotropic glutamate receptor 5 (mGluR5) which controls a wide range of processes in the brain, spinal cord, retina, and peripheral nervous system. In animal studies, the inhibition of this receptor has shown therapeutic potential for the treatment of Tuberous Sclerosis Complex (TSC). This receptor's inhibition decreases the frequency of seizures. In previous clinical trials, the study drug has shown an advantageous safety profile in children and adolescents. The objective of this study is to find an optimal dose at which the study drug will lead to a decrease in the duration, frequency and intensity of seizures in children, adolescents and young adults with TSC, while being well tolerated. All patients who positively respond and tolerate the medicine will be offered the possibility to continue in an open label extension.

Conditions

• Tuberous Sclerosis Complex

Interventions

Type	Name	Description
DRUG	Basimglurant with crossover to Placebo	Basimglurant with crossover to Placebo
DRUG	Placebo with crossover to Basimglurant	Placebo with crossover to Basimglurant

Timeline

• Start date: 2022-03-03
• Primary completion: 2025-02-06
• Completion: 2025-04-28
• First posted: 2021-09-28
• Last updated: 2025-06-04

Locations

27 sites across 8 countries: United States, India, Israel, Italy, Poland, Spain, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study