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

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

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.

Inclusion Criteria (summary):

• Ability and willingness to provide informed assent or written consent or consent from their legal representative.

• Fluency in the language of the study staff

• Age 5 to 30 years at study entry

• A documented history of TSC

• Refractory seizure history

• Currently receiving one or more anti-epileptic drugs (AEDs)

• Stable medications or interventions for epilepsy

• Willingness to complete Patient Reported Outcome assessments

• For female patients of childbearing potential:

  1. Willingness to undergo serum or urinary pregnancy testing at screening and during the trial period.
  2. Willingness to use contraception.

Exclusion Criteria (summary):

• Neurologic disease other than TSC
• Recent anoxic episode
• Patient weight below 15kg
• Clinically significant unstable medical condition(s)
• Pregnancy or lactation