The study intends to show that basimglurant (NOE-101) provides effective seizure control in children, adolescents and young adults with Tuberous Sclerosis Complex (TSC).
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 Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
61
Basimglurant with crossover to Placebo
Placebo with crossover to Basimglurant
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
Number of patients considered treatment responders.
Time frame: 30 weeks
Longest seizure free interval (i.e., seizure free days).
Time frame: 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
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
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.
Time frame: 82 weeks
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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
...and 17 more locations