Basimglurant for Tuberous Sclerosis

Recruiting at 65 trial locations

Overseen By Noema Pharma

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Noema Pharma AG

Must be taking: Anti-epileptic drugs

Disqualifiers: Neurologic disease, Anoxic episode, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 30 weeks

61 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop your current medications, but it mentions that participants should have stable medications for epilepsy. This suggests you may continue your current anti-epileptic drugs.

How is the drug Basimglurant different from other treatments for tuberous sclerosis?

Basimglurant is unique because it targets the mGluR5 receptor, which is involved in brain signaling, potentially offering a new approach to managing symptoms of tuberous sclerosis, unlike other treatments that focus on different pathways like mTOR inhibition. ^{1 2 3 4 5}

Research Team

Clinical Director, MD

Principal Investigator

Noema Pharma AG

Eligibility Criteria

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.

Inclusion Criteria

For female patients of childbearing potential: Willingness to use contraception

I am willing to take pregnancy tests if I can have children.

I am between 5 and 30 years old.

Exclusion Criteria

Recent anoxic episode

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

I weigh less than 15kg.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Basimglurant or placebo in a cross-over, double-blind, randomized manner for 30 weeks

30 weeks

Open-label extension

Participants who respond positively and tolerate the medicine continue with Basimglurant for 52 weeks

52 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Basimglurant

Trial Overview 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.

Participant Groups

Group I: Arm A (Basimglurant/NOE-101 to Placebo) Experimental Treatment 1 Intervention

Basimglurant to Placebo

Group II: Arm B (Placebo to Basimglurant/NOE-101) Placebo Group 1 Intervention

Placebo to Basimglurant

Who Is Running the Clinical Trial?

Noema Pharma AG

Lead Sponsor

Trials

6

Recruited

570+

References

1. England pubmed.ncbi.nlm.nih.gov

Childhood tuberous sclerosis complex in southern Sweden: a paradigm shift in diagnosis and treatment. [2023]

2. Japan pubmed.ncbi.nlm.nih.gov

[Anesthetic management of a patient with tuberous sclerosis]. [2013]

3. England pubmed.ncbi.nlm.nih.gov

Effects of bumetanide on neurodevelopmental impairments in patients with tuberous sclerosis complex: an open-label pilot study. [2021]

4. China pubmed.ncbi.nlm.nih.gov

[Efficacy and safety of rapamycin in treatment of children with epilepsy complicated with tuberous sclerosis]. [2018]

5. England pubmed.ncbi.nlm.nih.gov

Everolimus improves neuropsychiatric symptoms in a patient with tuberous sclerosis carrying a novel TSC2 mutation. [2021]