Basimglurant in Children, Adolescents, and Young Adults With TSC

Tuberous Sclerosis Complex Clinical Trial

Official 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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05059327
• Other study ID #: NOE-TSC-201
• Status: Recruiting
• Phase: Phase 2
• Start date: March 3, 2021
• Est. completion date: January 31, 2026

Study information

• Verified date: April 2024
• Source: Noema Pharma AG
• Contact: Noema Pharma
• Phone: Please contact via email:
• Email: clinicaltrials[@]noemapharma.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 54
• Est. completion date: January 31, 2026
• Est. primary completion date: November 30, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 5 Years to 30 Years

Eligibility

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

Related Conditions & MeSH terms

• Sclerosis
• Tuberous Sclerosis
• Tuberous Sclerosis Complex

Intervention

Drug:
• Basimglurant with crossover to Placebo
Basimglurant with crossover to Placebo
• Placebo with crossover to Basimglurant
Placebo with crossover to Basimglurant

Locations

Country	Name	City	State
India	Gleneagles Global Health City (Site # 802)	Chennai	Tamil Nadu
India	Citi Neuro Centre (Site # 806)	Hyderabad	Andhra Pradesh
India	Rainbow Children's Hospital (Site # 803)	Hyderabad	Andhra Pradesh
India	Jaslok Hospital and Research Centre (Site # 801)	Mumbai	Maharashtra
India	All India Institute of Medical Sciences (Site # 804)	New Delhi	Delhi
India	Deenanath Mangeshkar Hospital and Research Centre (Site # 805)	Pune	Maharashtra
India	Christian Medical College (Site # 807)	Vellore	Tamil Nadu
Israel	Hadassah Medical Center - PPDS (Site #: 503)	Jerusalem
Italy	Azienda Ospedaliero Universitaria Policlinico Vittorio Emanuele (Site # 182)	Catania	Sicilia
Italy	Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN (Site # 184)	Firenze	Toscana
Italy	ASST Grande Ospedale Metropolitano Niguarda - Presidio Ospedaliero Ospedale Niguarda (Site # 183)	Milano	Lombardia
Italy	ASST Santi Paolo e Carlo - Azienda Universitaria-Polo Universitario San Paolo (Site # 181)	Milano	Lombardia
Poland	Centrum Medyczne Plejady (Site # 263)	Kraków
Poland	AmiCare Centrum Medyczne (Site # 261)	Lódz
Poland	Instytut Pomnik Centrum Zdrowia Dziecka (Site # 262)	Warsaw	Mazowieckie
Spain	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
Turkey	Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi (Site #: 905)	Fatih	Istanbul
Turkey	Yeditepe University Kosuyolu Hospital (Site #: 904)	Kadiköy	Istanbul
Turkey	Istanbul Egitim ve Arastirma Hastanesi (Site #: 901)	Sultangazi	Istanbul
Turkey	Ankara Etlik Sehir Hastanesi (Site #: 903)	Yenimahalle	Ankara
United Kingdom	Queen Elizabeth Hospital Birmingham (Site # 301)	Birmingham	West Midlands
United Kingdom	Cardiff University (Site # 305)	Cardiff	South Glamorgan
United Kingdom	Noahs Ark Children's Hospital (Site # 306)	Cardiff	South Glamorgan
United Kingdom	St George Hospital (Site # 303)	London	London, City Of
United Kingdom	The Royal London Hospital (Site # 302)	London	London, City Of
United Kingdom	Salford Royal Hospital - PPDS (Site # 304)	Salford	Lancashire
United States	Kennedy Krieger Institute (Site #: 110)	Baltimore	Maryland
United States	Boston Children's Hospital (Site #: 102)	Boston	Massachusetts
United States	University Hospitals Cleveland Medical Center, Rainbow Babies and Childrens Hospital (Site #: 107)	Cleveland	Ohio
United States	Texas Scottish Rite Hospital For Children (Site #: 113)	Dallas	Texas
United States	Duke Children's Hospital and Health Center (Site #: 106)	Durham	North Carolina
United States	Boston Children's Health Physicians (BCHP) (Site #: 111)	Hawthorne	New York
United States	The University of Texas Medical School at Houston (Site #: 103)	Houston	Texas
United States	David Geffen School of Medicine at UCLA (Site #: 101)	Los Angeles	California
United States	Minnesota Epilepsy Group PA (Site #: 105)	Roseville	Minnesota
United States	William Beaumont Hospital - Royal Oak (Site #: 104)	Royal Oak	Michigan
United States	Multicare Health System (Site #: 109)	Tacoma	Washington

Sponsors (1)

Lead Sponsor	Collaborator
Noema Pharma AG

Countries where clinical trial is conducted

United States,  India,  Israel,  Italy,  Poland,  Spain,  Turkey,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	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
Other	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
Other	Intensity of seizures detected by the wearable device evaluated as the change from Baseline compared to study treatment Periods 1 and 4.		30 weeks
Primary	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	Number of patients considered treatment responders.		30 weeks
Secondary	Longest seizure free interval (i.e., seizure free days).		30 weeks
Secondary	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
Secondary	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
Secondary	Safety of the study drug in children, adolescents and young adults with seizures associated with TSC.	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