Basimglurant in Children, Adolescents, and Young Adults With TSC

June 12, 2024 updated by: Noema Pharma AG

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

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

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

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.

Study Type

Interventional

Enrollment (Estimated)

54

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • India
    • Andhra Pradesh
      • Hyderabad, Andhra Pradesh, India, 500034
        • Recruiting
        • Citi Neuro Centre (Site # 806)
      • Hyderabad, Andhra Pradesh, India, 500034
        • Recruiting
        • Rainbow Children's Hospital (Site # 803)
    • Delhi
      • New Delhi, Delhi, India, 110029
        • Recruiting
        • All India Institute of Medical Sciences (Site # 804)
    • Maharashtra
      • Mumbai, Maharashtra, India, 400026
        • Recruiting
        • Jaslok Hospital and Research Centre (Site # 801)
      • Pune, Maharashtra, India, 411006
        • Recruiting
        • Deenanath Mangeshkar Hospital and Research Centre (Site # 805)
    • Tamil Nadu
      • Chennai, Tamil Nadu, India, 600100
        • Withdrawn
        • Gleneagles Global Health City (Site # 802)
      • Vellore, Tamil Nadu, India, 632004
        • Recruiting
        • Christian Medical College (Site # 807)
  • Israel
      • Jerusalem, Israel, 91120
        • Completed
        • Hadassah Medical Center - PPDS (Site #: 503)
  • Italy
    • Lombardia
      • Milano, Lombardia, Italy, 20162
        • Recruiting
        • ASST Grande Ospedale Metropolitano Niguarda - Presidio Ospedaliero Ospedale Niguarda (Site # 183)
      • Milano, Lombardia, Italy, 20142
        • Active, not recruiting
        • ASST Santi Paolo e Carlo - Azienda Universitaria-Polo Universitario San Paolo (Site # 181)
    • Sicilia
      • Catania, Sicilia, Italy, 20142
        • Active, not recruiting
        • Azienda Ospedaliero Universitaria Policlinico Vittorio Emanuele (Site # 182)
    • Toscana
      • Firenze, Toscana, Italy, 50139
        • Active, not recruiting
        • Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN (Site # 184)
  • Poland
      • Kraków, Poland, 30-363
        • Active, not recruiting
        • Centrum Medyczne Plejady (Site # 263)
      • Łódź, Poland, 91-495
        • Active, not recruiting
        • AmiCare Centrum Medyczne (Site # 261)
    • Mazowieckie
      • Warsaw, Mazowieckie, Poland, 04-730
        • Recruiting
        • Instytut Pomnik Centrum Zdrowia Dziecka (Site # 262)
  • Spain
      • Málaga, Spain, 29010
        • Recruiting
        • Hospital Regional Universitario de Malaga - Hospital General (Site # 193)
      • Sevilla, Spain, 41013
        • Recruiting
        • Centro de Neurología Avanzada (Site # 191)
    • Barcelona
      • Badalona, Barcelona, Spain, 08916
        • Recruiting
        • Hospital Universitario Germans Trias i Pujol (Site # 194)
      • Esplugues De Llobregat, Barcelona, Spain, 08950
        • Recruiting
        • Hospital Sant Joan de Deu - PIN (Site # 192)
  • Turkey
    • Ankara
      • Yenimahalle, Ankara, Turkey, 06170
        • Active, not recruiting
        • Ankara Etlik Şehir Hastanesi (Site #: 903)
    • Istanbul
      • Fatih, Istanbul, Turkey, 34093
        • Recruiting
        • Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi (Site #: 905)
      • Kadıköy, Istanbul, Turkey, 34718
        • Recruiting
        • Yeditepe University Kosuyolu Hospital (Site #: 904)
      • Sultangazi, Istanbul, Turkey, 34096
        • Recruiting
        • Istanbul Egitim ve Arastirma Hastanesi (Site #: 901)
  • United Kingdom
    • Lancashire
      • Salford, Lancashire, United Kingdom, M6 8HD
        • Recruiting
        • Salford Royal Hospital - PPDS (Site # 304)
    • London, City Of
      • London, London, City Of, United Kingdom, E1 1BB
        • Active, not recruiting
        • The Royal London Hospital (Site # 302)
      • London, London, City Of, United Kingdom, SW17 0QT
        • Active, not recruiting
        • St George Hospital (Site # 303)
    • South Glamorgan
      • Cardiff, South Glamorgan, United Kingdom, CF14 4XW
        • Recruiting
        • Noahs Ark Children's Hospital (Site # 306)
      • Cardiff, South Glamorgan, United Kingdom, CF10 3AT
        • Active, not recruiting
        • Cardiff University (Site # 305)
    • West Midlands
      • Birmingham, West Midlands, United Kingdom, B15 2TH
        • Active, not recruiting
        • Queen Elizabeth Hospital Birmingham (Site # 301)
  • United States
    • California
      • Los Angeles, California, United States, 90095
        • Recruiting
        • David Geffen School of Medicine at UCLA (Site #: 101)
    • Maryland
      • Baltimore, Maryland, United States, 21205
        • Completed
        • Kennedy Krieger Institute (Site #: 110)
    • Massachusetts
      • Boston, Massachusetts, United States, 02115-5724
        • Recruiting
        • Boston Children's Hospital (Site #: 102)
    • Michigan
      • Royal Oak, Michigan, United States, 48073-6712
        • Completed
        • William Beaumont Hospital - Royal Oak (Site #: 104)
    • Minnesota
      • Roseville, Minnesota, United States, 55113-1306
        • Recruiting
        • Minnesota Epilepsy Group PA (Site #: 105)
    • New York
      • Hawthorne, New York, United States, 10532
        • Completed
        • Boston Children's Health Physicians (BCHP) (Site #: 111)
    • North Carolina
      • Durham, North Carolina, United States, 27705-4699
        • Completed
        • Duke Children's Hospital and Health Center (Site #: 106)
    • Ohio
      • Cleveland, Ohio, United States, 44106-1716
        • Completed
        • University Hospitals Cleveland Medical Center, Rainbow Babies and Childrens Hospital (Site #: 107)
    • Texas
      • Dallas, Texas, United States, 75219-3924
        • Withdrawn
        • Texas Scottish Rite Hospital For Children (Site #: 113)
      • Houston, Texas, United States, 77030-3000
        • Recruiting
        • The University of Texas Medical School at Houston (Site #: 103)
    • Washington
      • Tacoma, Washington, United States, 98405-4048
        • Withdrawn
        • Multicare Health System (Site #: 109)

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

5 years to 30 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

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:

    1. Willingness to undergo serum or urinary pregnancy testing at screening and during the trial period.
    2. 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Crossover Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Arm A (Basimglurant to Placebo)
Basimglurant to Placebo
Drug: Basimglurant with crossover to Placebo
Basimglurant with crossover to Placebo
Placebo Comparator: Arm B (Placebo to Basimglurant)
Placebo to Basimglurant
Drug: Placebo with crossover to Basimglurant
Placebo with crossover to Basimglurant

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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
30 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of patients considered treatment responders.
Time Frame: 30 weeks
30 weeks
Longest seizure free interval (i.e., seizure free days).
Time Frame: 30 weeks
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.
Time Frame: 30 weeks
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.
Time Frame: 30 weeks
30 weeks
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.
82 weeks

Other Outcome Measures

Outcome Measure
Time Frame
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
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).
Time Frame: 30 weeks
30 weeks
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
30 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Noema Pharma AG

Investigators

  • Study Director: Clinical Director, MD, Noema Pharma AG

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 3, 2022

Primary Completion (Estimated)

November 30, 2024

Study Completion (Estimated)

January 31, 2026

Study Registration Dates

First Submitted

August 20, 2021

First Submitted That Met QC Criteria

September 20, 2021

First Posted (Actual)

September 28, 2021

Study Record Updates

Last Update Posted (Actual)

June 13, 2024

Last Update Submitted That Met QC Criteria

June 12, 2024

Last Verified

June 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NOE-TSC-201

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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