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

Phase 2

Active, not recruiting

• Conditions: Tuberous Sclerosis Complex
• Interventions: Drug: Basimglurant with crossover to Placebo; Drug: Placebo with crossover to Basimglurant

• Registration Number: NCT05059327
• Lead Sponsor: Noema Pharma AG

Brief 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.

Study Timeline

• Study First Submitted: 2021-08-20
• Study First Submitted QC: 2021-09-20
• Study First Posted: 2021-09-28
• Study Start: 2022-03-03
• Primary Completion: 2025-02-06
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-16
• Last Update Posted: 2025-04-17
• Study Completion: 2026-01-31

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 61

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
Arm A (Basimglurant/NOE-101 to Placebo)	Basimglurant with crossover to Placebo	Basimglurant to Placebo
Arm B (Placebo to Basimglurant/NOE-101)	Placebo with crossover to Basimglurant	Placebo to Basimglurant

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
Number of patients considered treatment responders.	30 weeks
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.	30 weeks
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.	30 weeks
Safety of the study drug in children, adolescents and young adults with seizures associated with TSC.	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.
Longest seizure free interval (i.e., seizure free days).	30 weeks

Trial Locations

Locations (27)

Rainbow Children's Hospital (Site # 803); 🇮🇳 Hyderabad, Andhra Pradesh, India

David Geffen School of Medicine at UCLA (Site #: 101); 🇺🇸 Los Angeles, California, United States

Kennedy Krieger Institute (Site #: 110); 🇺🇸 Baltimore, Maryland, United States

Boston Children's Hospital (Site #: 102); 🇺🇸 Boston, Massachusetts, United States

William Beaumont Hospital - Royal Oak (Site #: 104); 🇺🇸 Royal Oak, Michigan, United States

Minnesota Epilepsy Group PA (Site #: 105); 🇺🇸 Roseville, Minnesota, United States

Boston Children's Health Physicians (BCHP) (Site #: 111); 🇺🇸 Hawthorne, New York, United States

Duke Children's Hospital and Health Center (Site #: 106); 🇺🇸 Durham, North Carolina, United States

University Hospitals Cleveland Medical Center, Rainbow Babies and Childrens Hospital (Site #: 107); 🇺🇸 Cleveland, Ohio, United States

The University of Texas Medical School at Houston (Site #: 103); 🇺🇸 Houston, Texas, United States

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

All India Institute of Medical Sciences (Site # 804); 🇮🇳 New Delhi, Delhi, India

Jaslok Hospital and Research Centre (Site # 801); 🇮🇳 Mumbai, Maharashtra, India

Deenanath Mangeshkar Hospital and Research Centre (Site # 805); 🇮🇳 Pune, Maharashtra, India

Christian Medical College (Site # 807); 🇮🇳 Vellore, Tamil Nadu, India

Hadassah Medical Center - PPDS (Site #: 503); 🇮🇱 Jerusalem, Israel

ASST Grande Ospedale Metropolitano Niguarda - Presidio Ospedaliero Ospedale Niguarda (Site # 183); 🇮🇹 Milano, Lombardia, Italy

Instytut Pomnik Centrum Zdrowia Dziecka (Site # 262); 🇵🇱 Warsaw, Mazowieckie, Poland

Hospital Universitario Germans Trias i Pujol (Site # 194); 🇪🇸 Badalona, Barcelona, Spain

Hospital Sant Joan de Deu - PIN (Site # 192); 🇪🇸 Esplugues De Llobregat, Barcelona, Spain

Hospital Regional Universitario de Malaga - Hospital General (Site # 193); 🇪🇸 Málaga, Spain

Centro de Neurología Avanzada (Site # 191); 🇪🇸 Sevilla, Spain

Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi (Site #: 905); 🇹🇷 Fatih, Istanbul, Turkey

Yeditepe University Kosuyolu Hospital (Site #: 904); 🇹🇷 Kadıköy, Istanbul, Turkey

Istanbul Egitim ve Arastirma Hastanesi (Site #: 901); 🇹🇷 Sultangazi, Istanbul, Turkey

Salford Royal Hospital - PPDS (Site # 304); 🇬🇧 Salford, Lancashire, United Kingdom

Noahs Ark Children's Hospital (Site # 306); 🇬🇧 Cardiff, South Glamorgan, United Kingdom