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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT05059327
Other study ID # NOE-TSC-201
Secondary ID
Status Recruiting
Phase Phase 2
First received
Last updated
Start date March 3, 2022
Est. completion date January 31, 2026

Study information

Verified date June 2024
Source Noema Pharma AG
Contact Noema Pharma
Phone Please contact via email:
Email clinicaltrials@noemapharma.com
Is FDA regulated No
Health authority
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

Study Design


Related Conditions & MeSH terms


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
See also
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