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

NCT05059327 | Completed Phase 2 FDA Drug

• 1 other identifier: NOE-TSC-201
• Study Type: interventional
• Target: 61
• Locations: 8 countries
• Sites: 27

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).

Conditions

Tuberous Sclerosis Complex

Keywords

Basimglurant; TSC; Seizures; NOE-101

Outcome Measures

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).

  30 weeks

Secondary Outcomes (5)

• Number of patients considered treatment responders.

  30 weeks

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

  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

Other Outcomes (3)

• 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.

  30 weeks

• 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).

  30 weeks

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

  30 weeks

Study Arms (2)

Arm A (Basimglurant/NOE-101 to Placebo)

EXPERIMENTAL

Basimglurant to Placebo

Drug: Basimglurant with crossover to Placebo

Arm B (Placebo to Basimglurant/NOE-101)

PLACEBO COMPARATOR

Placebo to Basimglurant

Drug: Placebo with crossover to Basimglurant

Interventions

Basimglurant with crossover to Placebo DRUG

Basimglurant with crossover to Placebo

Arm A (Basimglurant/NOE-101 to Placebo)

Placebo with crossover to Basimglurant DRUG

Placebo with crossover to Basimglurant

Arm B (Placebo to Basimglurant/NOE-101)

Eligibility Criteria

• Age: 5 Years - 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

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

Sponsors & Collaborators

• Noema Pharma AG: lead

Study Sites (27)

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

Los Angeles, California, 90095, United States

Location

Kennedy Krieger Institute (Site #: 110)

Baltimore, Maryland, 21205, United States

Location

Boston Children's Hospital (Site #: 102)

Boston, Massachusetts, 02115-5724, United States

Location

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

Royal Oak, Michigan, 48073-6712, United States

Location

Minnesota Epilepsy Group PA (Site #: 105)

Roseville, Minnesota, 55113-1306, United States

Location

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

Hawthorne, New York, 10532, United States

Location

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

Durham, North Carolina, 27705-4699, United States

Location

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

Cleveland, Ohio, 44106-1716, United States

Location

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

Houston, Texas, 77030-3000, United States

Location

Citi Neuro Centre (Site # 806)

Hyderabad, Andhra Pradesh, 500034, India

Location

Rainbow Childrens Hospital (Site # 803)

Hyderabad, Andhra Pradesh, 500034, India

Location

Jaslok Hospital and Research Centre (Site # 801)

Mumbai, Maharashtra, 400026, India

Location

Deenanath Mangeshkar Hospital and Research Centre (Site # 805)

Pune, Maharashtra, 411006, India

Location

All India Institute of Medical Sciences (Site # 804)

New Delhi, National Capital Territory of Delhi, 110029, India

Location

Christian Medical College (Site # 807)

Vellore, Tamil Nadu, 632004, India

Location

Hadassah Medical Center - PPDS (Site #: 503)

Jerusalem, 91120, Israel

Location

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

Milan, Lombardy, 20162, Italy

Location

Instytut Pomnik Centrum Zdrowia Dziecka (Site # 262)

Warsaw, Masovian Voivodeship, 04-730, Poland

Location

Hospital Universitario Germans Trias i Pujol (Site # 194)

Badalona, Barcelona, 08916, Spain

Location

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

Esplugues de Llobregat, Barcelona, 08950, Spain

Location

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

Málaga, 29010, Spain

Location

Centro de Neurología Avanzada (Site # 191)

Seville, 41013, Spain

Location

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

Fatih, Istanbul, 34093, Turkey (Türkiye)

Location

Yeditepe University Kosuyolu Hospital (Site #: 904)

Kadıköy, Istanbul, 34718, Turkey (Türkiye)

Location

Istanbul Egitim ve Arastirma Hastanesi (Site #: 901)

Sultangazi, Istanbul, 34096, Turkey (Türkiye)

Location

Salford Royal Hospital - PPDS (Site # 304)

Salford, Lancashire, M6 8HD, United Kingdom

Location

Noahs Ark Children's Hospital (Site # 306)

Cardiff, South Glamorgan, CF14 4XW, United Kingdom

Location

MeSH Terms

Conditions

Tuberous Sclerosis; Seizures

Interventions

2-chloro-4-(1-(4-fluorophenyl)-2,5-dimethyl-1H-imidazol-4-ylethynyl)pyridine

Condition Hierarchy (Ancestors)

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

Study Officials

• Clinical Director, MD

  Noema Pharma AG

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: August 20, 2021
• First Posted: September 28, 2021
• Study Start: March 3, 2022
• Primary Completion: February 6, 2025
• Study Completion: April 28, 2025
• Last Updated: June 4, 2025

Record last verified: 2025-05

Locations

GB (2); IL (1); IT (1); US (9); TU (3); IN (6); PL (1); ES (4)