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

NCT ID: NCT05059327

Last Updated: 2025-06-04

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

61 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-03-03

Study Completion Date

2025-04-28

Brief Summary

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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

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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

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Tuberous Sclerosis Complex

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

This is a multi-center, randomized, double-blind, placebo-controlled, 30-week, cross-over (Part A) followed by a 52-week open-label extension (OLE) (Part B) study.
Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Arm A (Basimglurant/NOE-101 to Placebo)

Basimglurant to Placebo

Group Type EXPERIMENTAL

Basimglurant with crossover to Placebo

Intervention Type DRUG

Basimglurant with crossover to Placebo

Arm B (Placebo to Basimglurant/NOE-101)

Placebo to Basimglurant

Group Type PLACEBO_COMPARATOR

Placebo with crossover to Basimglurant

Intervention Type DRUG

Placebo with crossover to Basimglurant

Interventions

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Basimglurant with crossover to Placebo

Basimglurant with crossover to Placebo

Intervention Type DRUG

Placebo with crossover to Basimglurant

Placebo with crossover to Basimglurant

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* 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

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

5 Years

Maximum Eligible Age

30 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Noema Pharma AG

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Clinical Director, MD

Role: STUDY_DIRECTOR

Noema Pharma AG

Locations

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David Geffen School of Medicine at UCLA (Site #: 101)

Los Angeles, California, United States

Site Status

Kennedy Krieger Institute (Site #: 110)

Baltimore, Maryland, United States

Site Status

Boston Children's Hospital (Site #: 102)

Boston, Massachusetts, United States

Site Status

William Beaumont Hospital - Royal Oak (Site #: 104)

Royal Oak, Michigan, United States

Site Status

Minnesota Epilepsy Group PA (Site #: 105)

Roseville, Minnesota, United States

Site Status

Boston Children's Health Physicians (BCHP) (Site #: 111)

Hawthorne, New York, United States

Site Status

Duke Children's Hospital and Health Center (Site #: 106)

Durham, North Carolina, United States

Site Status

University Hospitals Cleveland Medical Center, Rainbow Babies and Childrens Hospital (Site #: 107)

Cleveland, Ohio, United States

Site Status

The University of Texas Medical School at Houston (Site #: 103)

Houston, Texas, United States

Site Status

Citi Neuro Centre (Site # 806)

Hyderabad, Andhra Pradesh, India

Site Status

Rainbow Childrens Hospital (Site # 803)

Hyderabad, Andhra Pradesh, India

Site Status

Jaslok Hospital and Research Centre (Site # 801)

Mumbai, Maharashtra, India

Site Status

Deenanath Mangeshkar Hospital and Research Centre (Site # 805)

Pune, Maharashtra, India

Site Status

All India Institute of Medical Sciences (Site # 804)

New Delhi, National Capital Territory of Delhi, India

Site Status

Christian Medical College (Site # 807)

Vellore, Tamil Nadu, India

Site Status

Hadassah Medical Center - PPDS (Site #: 503)

Jerusalem, , Israel

Site Status

ASST Grande Ospedale Metropolitano Niguarda - Presidio Ospedaliero Ospedale Niguarda (Site # 183)

Milan, Lombardy, Italy

Site Status

Instytut Pomnik Centrum Zdrowia Dziecka (Site # 262)

Warsaw, Masovian Voivodeship, Poland

Site Status

Hospital Universitario Germans Trias i Pujol (Site # 194)

Badalona, Barcelona, Spain

Site Status

Hospital Sant Joan de Deu - PIN (Site # 192)

Esplugues de Llobregat, Barcelona, Spain

Site Status

Hospital Regional Universitario de Malaga - Hospital General (Site # 193)

Málaga, , Spain

Site Status

Centro de Neurología Avanzada (Site # 191)

Seville, , Spain

Site Status

Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi (Site #: 905)

Fatih, Istanbul, Turkey (Türkiye)

Site Status

Yeditepe University Kosuyolu Hospital (Site #: 904)

Kadıköy, Istanbul, Turkey (Türkiye)

Site Status

Istanbul Egitim ve Arastirma Hastanesi (Site #: 901)

Sultangazi, Istanbul, Turkey (Türkiye)

Site Status

Salford Royal Hospital - PPDS (Site # 304)

Salford, Lancashire, United Kingdom

Site Status

Noahs Ark Children's Hospital (Site # 306)

Cardiff, South Glamorgan, United Kingdom

Site Status

Countries

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United States India Israel Italy Poland Spain Turkey (Türkiye) United Kingdom

Other Identifiers

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NOE-TSC-201

Identifier Type: -

Identifier Source: org_study_id

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