Basimglurant for Tuberous Sclerosis

No longer recruiting at 72 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new medication, basimglurant, to determine its effectiveness in controlling seizures in individuals with Tuberous Sclerosis Complex (TSC), a condition that often causes tumors in various organs and can lead to epilepsy. Researchers aim to discover if basimglurant reduces seizures in those unresponsive to other treatments. Participants will either start with basimglurant and switch to a placebo or do the reverse. Suitable candidates have a history of TSC-related seizures, currently take anti-seizure medications, and are willing to try a new treatment. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

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.

Is there any evidence suggesting that basimglurant is likely to be safe for humans?

Research shows that basimglurant, also known as NOE-101, is under study to help control seizures in people with Tuberous Sclerosis Complex (TSC). Previous studies found that basimglurant is generally well-tolerated, with only mild side effects reported and serious reactions rare. Since this trial is in Phase 2, basimglurant has already passed initial safety tests in earlier studies. Although the FDA has not yet approved basimglurant for TSC, ongoing research aims to confirm its safety and effectiveness for controlling seizures in children, adolescents, and young adults. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Tuberous Sclerosis?

Unlike the standard treatments for tuberous sclerosis, which often focus on managing symptoms with drugs like everolimus or anti-seizure medications, Basimglurant offers a new approach. This investigational drug is unique because it targets the mGluR5 receptor, which plays a role in neurological and psychiatric symptoms associated with the condition. Researchers are excited about Basimglurant because it has the potential to address underlying neural pathways, possibly offering more effective and targeted relief for patients. This could mean better management of symptoms like seizures and cognitive issues, enhancing the quality of life for those affected by tuberous sclerosis.

What evidence suggests that basimglurant might be an effective treatment for Tuberous Sclerosis?

Research suggests that basimglurant, which participants in this trial may receive, can help control seizures in people with Tuberous Sclerosis Complex (TSC). Studies have shown that it effectively manages these seizures, offering hope for better control. This treatment targets a specific part of the brain called mGluR5, believed to be involved in seizure activity. Early research indicates that basimglurant might reduce seizure frequency, potentially improving life for those with TSC. While more research is needed, the initial results are promising for those seeking new treatment options. ^{1 2 3 4 6}

Who Is on the Research Team?

Clinical Director, MD

Principal Investigator

Noema Pharma AG

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05059327

Study Details | NCT05059327 | Basimglurant (NOE-101) in ... The study intends to show that basimglurant (NOE-101) provides effective seizure control in children, adolescents and young adults with Tuberous Sclerosis ...

2. noemapharma.com noemapharma.com/2024/08/13/noema-pharma-announces-completion-of-enrollment-for-galene-phase-2b-trial-evaluating-noe-101-a-first-in-class-mglur5-nam-for-seizure-control-in-tuberous-sclerosis-complex/

Noema Pharma A double-blind, placebo-controlled trial evaluating the efficacy of NOE-101 for seizure control in tuberous sclerosis complex (TSC).

3. clinicaltrials.gov clinicaltrials.gov/study/NCT05059327?term=AREA%5BConditionSearch%5D(%22Tuberous%20Sclerosis%22)&rank=8

Study Details | Basimglurant (NOE-101) in Children, Adolescents ... Types of outcome measures include primary outcome measure and secondary outcome measure. ... A type of intervention model describing a clinical trial in which two ...

4. clinicaltrials.eu clinicaltrials.eu/trial/study-on-basimglurant-for-children-adolescents-and-young-adults-with-tuberous-sclerosis-complex-related-seizures/

Study on Basimglurant for Children, Adolescents, and ... This clinical trial investigates the effectiveness and safety of Basimglurant in managing seizures associated with Tuberous Sclerosis ...

5. synapse.patsnap.com synapse.patsnap.com/article/noema-pharma-completes-enrollment-for-galene-phase-2b-trial-on-noe-101-for-seizure-control-in-tuberous-sclerosis-complex

Noema Pharma Completes Enrollment for GALENE Phase ... This trial is a double-blind, placebo-controlled study designed to evaluate the effectiveness of NOE-101 in controlling seizures in patients ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10083014/

Treatment-Resistant Epilepsy and Tuberous Sclerosis Complex As such, this study showed that preventive vigabatrin treatment markedly improves neurodevelopmental and epilepsy outcomes at 2 years of age.