EQU-001 for Focal Onset Seizures

Recruiting at 19 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

27 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial tests EQU-001, a new medication, in adults aged 18-65 with epilepsy who have uncontrolled focal onset seizures. It aims to see if EQU-001 can better control seizures when added to their current treatments.

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.

What data supports the effectiveness of the drug EQU-001 for focal onset seizures?

The research highlights that placebo effects can significantly impact seizure reduction in epilepsy trials, sometimes making it difficult to distinguish the true effectiveness of a drug. However, adjustments in trial design, such as study start time and baseline population, have shown that an effective drug can achieve significant results compared to placebo, suggesting that careful trial design is crucial for demonstrating a drug's true efficacy. ^{1 2 3 4 5}

Eligibility Criteria

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

You are between 18 and 65 years old when you agree to take part in the study.

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

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

References

1. United States pubmed.ncbi.nlm.nih.gov

Confusing placebo effect with natural history in epilepsy: A big data approach. [2019]

2. United States pubmed.ncbi.nlm.nih.gov

Novel study design to assess the efficacy and tolerability of antiseizure medications for focal-onset seizures in infants and young children: A consensus document from the regulatory task force and the pediatric commission of the International League against Epilepsy (ILAE), in collaboration with the Pediatric Epilepsy Research Consortium (PERC). [2023]

3. United States pubmed.ncbi.nlm.nih.gov

Clinical features associated with placebo response in refractory focal epilepsy. [2022]

4. New Zealand pubmed.ncbi.nlm.nih.gov

Can Matching-Adjusted Indirect Comparison Methods Mitigate Placebo Response Differences Among Patient Populations in Adjunctive Trials of Brivaracetam and Levetiracetam? [2018]

5. United States pubmed.ncbi.nlm.nih.gov

Placebo and nocebo responses in drug trials of epilepsy. [2018]