Summary

Eligibility
for people ages 5-30 (full criteria)
Location
at Los Angeles, California and other locations
Dates
study started
completion around
Principal Investigator
by Rajsekar Rajaraman

Description

Summary

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

Official Title

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

Details

The study drug (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.

Keywords

Tuberous Sclerosis Complex, Basimglurant, TSC, Seizures, Tuberous Sclerosis, Sclerosis

Eligibility

You can join if…

Open to people ages 5-30

(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.

You CAN'T join if...

(summary):

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

Locations

  • David Geffen School of Medicine at UCLA (Site #: 101) accepting new patients
    Los Angeles California 90095 United States
  • Multicare Health System (Site #: 109) withdrawn
    Tacoma Washington 98405-4048 United States
  • Texas Scottish Rite Hospital For Children (Site #: 113) accepting new patients
    Dallas Texas 75219-3924 United States

Lead Scientist at UCLA

  • Rajsekar Rajaraman
    HS Assistant Clinical Professor, Pediatrics, Medicine. Authored (or co-authored) 20 research publications

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Noema Pharma AG
Links
Sign up for this study
ID
NCT05059327
Phase
Phase 2 research study
Study Type
Interventional
Participants
Expecting 54 study participants
Last Updated