Basimglurant in Children, Adolescents, and Young Adults With TSC
Study Purpose
The study intends to show that basimglurant provides effective seizure control in children,
adolescents and young adults with Tuberous Sclerosis Complex (TSC).
Recruitment Criteria
Accepts Healthy Volunteers
Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms
No
Study Type
An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.
An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.
Searching Both is inclusive of interventional and observational studies.
Interventional
Eligible Ages
5 Years - 30 Years
Gender
All
More Inclusion & Exclusion Criteria
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
Trial Details
Trial ID:
This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.
Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.
Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.
Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.
Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.
Phase 2
Lead Sponsor
The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.
Noema Pharma AG
Principal Investigator
The person who is responsible for the scientific and technical direction of the entire clinical study.
Clinical Director, MD
Principal Investigator Affiliation
Noema Pharma AG
Agency Class
Category of organization(s) involved as sponsor (and collaborator) supporting the trial.
Industry
Overall Status
Recruiting
Countries
India, Israel, Italy, Poland, Spain, Turkey, United Kingdom, United States
Conditions
The disease, disorder, syndrome, illness, or injury that is being studied.
Tuberous Sclerosis Complex
Additional Details
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.
Arms & Interventions
Arms
Experimental: Arm A (Basimglurant to Placebo)
Basimglurant to Placebo
Placebo Comparator: Arm B (Placebo to Basimglurant)
Placebo to Basimglurant
Interventions
Drug: - Basimglurant with crossover to Placebo
Basimglurant with crossover to Placebo
Drug: - Placebo with crossover to Basimglurant
Placebo with crossover to Basimglurant
Contact a Trial Team
If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.
Los Angeles, California
Status
Recruiting
Address
David Geffen School of Medicine at UCLA (Site #: 101)
This website uses cookies to improve your user experience. By using this site you agree to our use of cookies. Please read our Privacy Policy to learn more.
Ok
