What side effects are associated with this type of intervention?

Common treatments for Tuberous Sclerosis Complex (TSC) include mTOR inhibitors like sirolimus and everolimus, which are used to manage seizures and other TSC-related symptoms. Known side effects of mTOR inhibitors include mouth ulcers, infections, hyperlipidemia, and delayed wound healing. For treatments similar to Basimglurant, which targets the mGluR5 receptor, side effects may include dizziness, fatigue, and gastrointestinal issues. It is important to consult with a healthcare provider to understand the full range of potential side effects and to tailor treatment to individual needs.

What prior FDA approvals exist?

The FDA has approved several treatments for Tuberous Sclerosis Complex (TSC), primarily focusing on mTOR inhibitors. Everolimus (marketed as Afinitor) is one such drug that has been approved for the treatment of TSC-associated partial-onset seizures and subependymal giant cell astrocytoma (SEGA). Another mTOR inhibitor, Sirolimus, has also been used off-label for TSC-related conditions. These treatments target the mTOR pathway, which is crucial in the pathophysiology of TSC. While Basimglurant, a Negative Allosteric Modulator of the mGluR5 receptor, is not yet FDA-approved, it represents a novel approach by targeting glutamatergic signaling, which is different from the mTOR pathway but still relevant to neural activity modulation in TSC.

What other types of research exist?

Promising alternatives to Basimglurant for Tuberous Sclerosis Complex (TSC) patients include Farnesyltransferase inhibitors (FTIs) such as BMS-214662 and BMS-225975, which inhibit mTOR/S6K signaling. These compounds have shown efficacy in inhibiting the growth of Tsc-null cells and may offer a novel therapeutic approach for TSC.

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Glutamate Receptor Antagonist

Basimglurant for Tuberous Sclerosis

Phase 2

Waitlist Available

Research Sponsored by Noema Pharma AG

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Age 5 to 30 years at study entry

Refractory seizure history

Must not have

Neurologic disease other than TSC

Patient weight below 15kg

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 82 weeks

Summary

This trial is testing basimglurant, a medicine that helps control seizures, in children, adolescents, and young adults with Tuberous Sclerosis Complex (TSC). Basimglurant works by blocking a specific receptor in the brain, which helps reduce seizures. The goal is to find the best dose that reduces seizures while being safe for patients.

Who is the study for?

This trial is for children, adolescents, and young adults aged 5 to 30 with Tuberous Sclerosis Complex (TSC) who have seizures not controlled by current medications. Participants must be fluent in the study staff's language, on stable epilepsy treatments, and willing to undergo pregnancy tests if applicable. Those under 15kg or with other significant medical conditions are excluded.

What is being tested?

The trial is testing Basimglurant's ability to control seizures in TSC patients. It involves a crossover design where participants receive either Basimglurant or a placebo first and then switch to the other after a certain period.

What are the potential side effects?

While specific side effects of Basimglurant aren't listed here, similar medications often cause drowsiness, dizziness, gastrointestinal issues like nausea or vomiting, mood changes, and potential allergic reactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am between 5 and 30 years old.

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My seizures do not respond to treatment.

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I am currently taking medication for epilepsy.

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My epilepsy treatment has been consistent without changes.

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I have been diagnosed with Tuberous Sclerosis Complex (TSC).

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have a neurological condition that is not tuberous sclerosis complex.

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I weigh less than 15kg.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 82 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~82 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 82 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Safety of the study drug in children, adolescents and young adults with seizures associated with TSC.

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: Arm A (Basimglurant/NOE-101 to Placebo)Experimental Treatment1 Intervention

Basimglurant to Placebo

Group II: Arm B (Placebo to Basimglurant/NOE-101)Placebo Group1 Intervention

Placebo to Basimglurant

0 / 7Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Tuberous Sclerosis Complex (TSC) include mTOR inhibitors like rapamycin and everolimus, which work by inhibiting the mTOR pathway, a key regulator of cell growth and proliferation that is often overactive in TSC. Basimglurant, a Negative Allosteric Modulator of the mGluR5 receptor, is being studied for its potential to control seizures in TSC patients by modulating glutamate signaling, which is implicated in neuronal excitability and seizure activity. These treatments are crucial for TSC patients as they target the underlying molecular pathways responsible for the growth of benign tumors and neurological symptoms, thereby improving quality of life and reducing disease burden.

Inhibition of mTOR Pathway by Rapamycin Decreases P-glycoprotein Expression and Spontaneous Seizures in Pharmacoresistant Epilepsy.