Skip to main content
OpenTrials
Completed

NCT Number: NCT05059327

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

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

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Tuberous Sclerosis Complex Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hamartoma Heredodegenerative Disorders, Nervous System Malformations of Cortical Development Malformations of Cortical Development, Group I Neoplasms Neoplasms, Multiple Primary Neoplastic Syndromes, Hereditary Nervous System Diseases Nervous System Malformations Neurocutaneous Syndromes Neurodegenerative Diseases Neurologic Manifestations Pathological Conditions, Signs and Symptoms Seizures Signs and Symptoms Tuberous Sclerosis

Age range

5 year–30 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Citi Neuro Centre (Site # 806), Hyderabad, Andhra Pradesh, India

Loading trial locations.

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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:
  • Willingness to undergo serum or urinary pregnancy testing at screening and during the trial period.
  • 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

Treatment and study plan

Basimglurant with crossover to Placebo

Drug

Basimglurant with crossover to Placebo

Placebo with crossover to Basimglurant

Drug

Placebo with crossover to Basimglurant

Primary outcomes

  1. Mean percentage in monthly seizure frequency during the maintenance dosing in Period 2 (Weeks 13 to 16) and Period 4 (Weeks 27-30).

    Time frame: 30 weeks

Secondary outcomes

  1. Number of patients considered treatment responders.

    Time frame: 30 weeks

  2. Longest seizure free interval (i.e., seizure free days).

    Time frame: 30 weeks

  3. Change in the severity of symptoms of TSC as measured by Caregiver Global Impression of Change (CGIC) score during maintenance dosing in Period 2 (Weeks 13 to 16) and Period 4 (Weeks 27-30) compared to Baseline.

    Time frame: 30 weeks

  4. Change in the Sheehan Disability Scale during maintenance dosing in Period 2 (Weeks 13 to 16) and Period 4 (Weeks 27-30) compared to baseline.

    Time frame: 30 weeks

  5. Safety of the study drug in children, adolescents and young adults with seizures associated with TSC.

    Time frame: 82 weeks

    Measured in terms of incidence, nature, and severity of adverse events, vital signs, physical examination, clinical chemistry, hematology, electrocardiograms, and urinalysis, as well as treatment delays, dose reductions, and dose discontinuations. In addition, suicidal ideation will be assessed using S-STS.

Other outcomes

  1. Change in seriousness of disease as assessed by Most Impactful Symptoms Scale in Periods 2 (weeks 13 to 16) and Period 4 (weeks 27 to 30) compared to baseline.

    Time frame: 30 weeks

  2. Frequency of seizures detected by the wearable device evaluated as the change from Baseline compared to study treatment in Period 1 (weeks 13 to 16) and Period 4 (weeks 27 to 30).

    Time frame: 30 weeks

  3. Intensity of seizures detected by the wearable device evaluated as the change from Baseline compared to study treatment Periods 1 and 4.

    Time frame: 30 weeks

Sponsors and collaborators

Lead sponsor

Noema Pharma AG

Industry

Registry information

Official study 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

Important dates

Study start
2022
Primary completion
2025
Study completion
2025
First posted
Sep 28, 2021
Registry last updated
Jun 4, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.