EQU-001 for Focal Onset Seizures

No longer recruiting at 20 trial locations

Overseen By Ya-El Mandel-Portnoy, PhD MSc

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Equilibre Biopharmaceuticals B.V.

Must be taking: Anti-seizure medications

Must not be taking: Ivermectin, Retigabine, Felbamate

Disqualifiers: Pregnancy, Psychiatric disorder, Substance use, others

Stay on Your Current Meds You can continue your current medications while participating

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

Compensation: Varies

Number of Visits: 4

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to evaluate the effectiveness and safety of a new drug, EQU-001, for individuals with focal onset seizures. These seizures begin in one area of the brain. The study will compare two doses of EQU-001 to a placebo (a pill with no active drug) to assess its effectiveness as an additional treatment for those whose seizures remain uncontrolled with current medication. Suitable candidates for this trial have experienced noticeable epilepsy-related seizures, such as shaking or loss of awareness, that have not been fully controlled by at least one anti-seizure medication in the past two years. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of participants.

Will I have to stop taking my current medications?

Participants must keep their current anti-seizure medications stable during the study. However, certain medications that may interfere with the study drug are not allowed, and some are discouraged. It's best to discuss your specific medications with the study team.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that EQU-001, the treatment under study, is safe. Studies have found that doses up to 60 mg are well-tolerated, with participants experiencing no severe side effects. The treatment also reduced the number of seizures, which is promising. Based on current evidence, EQU-001 appears to be a safe option for those considering joining the trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about EQU-001 for focal onset seizures because it offers a potential new approach to treatment. Unlike standard anti-seizure medications, which often target sodium channels or neurotransmitter levels, EQU-001 may work through a novel mechanism that's still being explored. The trial includes different dosing options, like a higher 60 mg dose and a lower 20 mg dose, which could help tailor the treatment to individual needs. This flexibility and the possibility of a new mechanism have sparked interest in its potential to provide better seizure control with fewer side effects.

What evidence suggests that EQU-001 might be an effective treatment for focal onset seizures?

Research has shown that EQU-001 may help treat focal onset seizures. In a preliminary study, 14.9% of participants remained seizure-free for at least a year, and this success rate increased to 23.6% for those who used the treatment longer. In this trial, participants will receive different doses of EQU-001, including 20 mg and 60 mg, or a placebo. EQU-001 has proven safe and well tolerated at doses up to 60 mg per day. These results suggest it could benefit people with uncontrolled focal onset seizures. ^{1 2 3 6 7}

Are You a Good Fit for This Trial?

Adults aged 18-65 with uncontrolled focal onset seizures, currently on 1-3 anti-seizure medications (ASMs), may join this trial. They must have had a stable dose for at least 4 weeks and not be part of another drug study. Women should use birth control, and participants need to keep a seizure diary. Those with certain neurological conditions, recent substance abuse, or specific medication history are excluded.

Inclusion Criteria

Willing and able to keep an accurate study diary and adhere to the protocol

Currently receiving treatment with 1-3 ASMs with stable doses for at least 4 weeks prior to screening

If participant has a vagal nerve stimulator (VNS), responsive neurostimulator (RNS) or deep brain stimulator (DBS), it must have been implanted and activated >1 year prior to screening

Exclusion Criteria

Presence of progressive neurological disorder or other progressive disorder or unstable medical condition(s) that may confound study results

You have had thoughts of hurting yourself in the past 6 months, or have tried to hurt yourself in the last 2 years, or have tried more than once in your life.

You have had problems with using drugs or alcohol in the past 2 years.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Medication Activation

Participants begin a 4-week double-blind medication activation period

4 weeks

1 visit (in-person)

Maintenance

Participants continue with a 12-week double-blind maintenance period

12 weeks

3 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• EQU-001
• Matching Placebo

Trial Overview The trial is testing EQU-001's effectiveness in reducing seizures when added to current ASM treatment compared to a placebo. Participants will receive either EQU-001 (20 mg or 60 mg) or placebo during a four-week activation period followed by twelve weeks of maintenance.

How Is the Trial Designed?

Group I: EQU-001 60 mg Experimental Treatment 1 Intervention

EQU-001 60 mg (3 x 20 mg pills)

Group II: EQU-001 20 mg Experimental Treatment 2 Interventions

EQU-001 20 mg (1 x 20 mg pill + 2 x matching placebo pills)

Group III: EQU-001 0 mg Placebo Group 1 Intervention

EQU-001 0 mg (3 x matching placebo pills)

Who Is Running the Clinical Trial?

Equilibre Biopharmaceuticals B.V.

Lead Sponsor

Trials

3

Recruited

150+

Published Research Related to This Trial

In a simulation study using data from 684,825 seizures, it was found that placebos consistently reduced seizure frequency, suggesting that some of the observed placebo effects in epilepsy trials may be due to the natural progression of the disease rather than the placebo itself.

Modifying trial design elements, such as study start time or reducing the baseline population, can significantly enhance the ability of an effective drug to show superiority over placebo, highlighting the importance of careful clinical trial design in epilepsy research.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Confusing placebo effect with natural history in epilepsy: A big data approach. Goldenholz, DM., Moss, R., Scott, J., et al.[2019]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05473442

Study of EQU-001 for Uncontrolled Focal Onset Seizures This study is a Phase 2 multinational, double-blind, placebo-controlled, randomized (1:1:1), efficacy and safety study of adjunctive EQU-001 for the treatment ...

2. neurologylive.com neurologylive.com/view/antiseizure-agent-equ-001-shows-safety-tolerability-preliminary-efficacy-phase-2-study

Antiseizure Agent EQU-001 Shows Safety, Tolerability, and ... Equilibre's antiseizure medication EQU-001 showed that the therapy was safe and well tolerated at all tested doses through 60 mg QD.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT05063877

Study Details | NCT05063877 | Safety, Tolerability, and ... This study of EQU-001 will provide safety of a range of doses, tolerability, and PK data in patients with epilepsy and aims to identify drug-specific DLTs and ...

4. centerwatch.com centerwatch.com/clinical-trials/listings/NCT05473442/study-of-equ-001-for-uncontrolled-focal-onset-seizures

Study of EQU-001 for Uncontrolled Focal Onset Seizures This study is a Phase 2 multinational, double-blind, placebo-controlled, randomized (1:1:1), efficacy and safety study of adjunctive EQU-001 ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12014913/

Interim analysis of the long‐term efficacy and safety of ... Seizure freedom for at least 12 consecutive months was achieved by 14.9% of all participants in the OLE and by 23.6% of those who had been treated for at least ...

6. clinicaltrials.eu clinicaltrials.eu/trial/study-of-equ-001-and-placebo-for-patients-with-uncontrolled-focal-seizures-in-epilepsy/

Study of EQU-001 and Placebo for Patients with ... This clinical trial tests the efficacy of EQU-001, an oral treatment, for reducing the frequency of uncontrolled focal onset seizures in ...

7. delta.larvol.com delta.larvol.com/Products/?ProductId=29dbca6c-8eff-4d77-88c8-4d5046aef7bf

EQU-001 / Equilibre Biopharma - Epilepsy EQU-001 demonstrated excellent safety profile and 60 mg daily dose of EQU-001 was able to induce clinically meaningful reductions in focal seizure frequency in ...