Clemizole HCl for Lennox-Gastaut Syndrome

(LGS Trial)

Recruiting at 3 trial locations

Overseen By Cindy Sandy

Age: < 65

Sex: Any

Trial Phase: Phase 3

Sponsor: Epygenix

Must not be taking: Fenfluramine, Lorcaserin

Disqualifiers: Long QT syndrome, Epilepsy surgery, others

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

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: Unknown

260 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment, clemizole HCl, to determine its safety and effectiveness for people with Lennox-Gastaut syndrome (LGS), a severe form of epilepsy that causes various seizures and developmental issues. Participants will receive either clemizole HCl or a placebo (a pill with no active medicine) to compare results. The trial suits those with major motor seizures, abnormal cognitive development, and an onset of seizures at age 11 or younger. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot use fenfluramine or lorcaserin while participating.

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

Research has shown that clemizole HCl, also known as EPX-100, has been tested for safety in people with Lennox-Gastaut syndrome (LGS). Clemizole, an older allergy medicine, has been used in other treatments before, providing some confidence about its safety.

In earlier studies, clemizole HCl was generally well-tolerated by patients. Most side effects were mild to moderate, with common ones being drowsiness and dizziness, typical for allergy medicines. Importantly, no serious side effects directly linked to clemizole appeared in the available data.

While research continues, findings so far suggest that clemizole HCl is a safe option for treating LGS. However, as with any treatment, discussing any concerns or questions with a healthcare provider is important. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Lennox-Gastaut syndrome?

Most treatments for Lennox-Gastaut Syndrome involve anti-seizure medications like valproate, lamotrigine, or topiramate, which mainly aim to control seizures. However, clemizole HCl is unique because it targets serotonin receptors in the brain, which may help in reducing seizures differently by modifying the brain's chemical signals. This novel mechanism of action offers a fresh approach compared to traditional therapies, potentially providing relief for patients who don't respond well to existing options. Researchers are excited because this could open up new avenues for managing this challenging condition, especially for those with treatment-resistant seizures.

What evidence suggests that clemizole HCl could be an effective treatment for Lennox-Gastaut syndrome?

Research shows that clemizole HCl, also known as EPX-100, might help treat Lennox-Gastaut syndrome (LGS), a severe type of epilepsy that begins in childhood. Early studies suggest that clemizole HCl can reduce seizures. This trial includes a double-blind arm where participants receive either clemizole HCl or a placebo, and an open-label arm where eligible participants continue receiving clemizole HCl. This treatment acts as an antihistamine, blocking certain brain chemicals that might cause seizures. While more research is underway, initial results are promising for people with LGS. ^{1 2 3 5 6}

Who Is on the Research Team?

Amit Ray, MD

Principal Investigator

Harmony Biosciences Management, Inc.

Are You a Good Fit for This Trial?

This trial is for males and females aged 14 or older with Lennox-Gastaut syndrome (LGS), a type of epilepsy. Participants must have had seizures starting at age 11 or younger, meet specific EEG criteria, and show abnormal cognitive development. Their treatment for epilepsy should be stable for at least 30 days before the study starts.

Inclusion Criteria

Participant/parent/legal authorized representative (LAR) willing and able to give written informed consent/assent

I am between 2 and 55 years old.

I have Lennox-Gastaut Syndrome with specific seizure types, EEG results, and cognitive issues.

Exclusion Criteria

I have used lorcaserin before or am currently using it.

I am scheduled for epilepsy surgery or had one within the last 6 months.

I do not have a history of serious heart rhythm problems.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Observational Period

Participants are observed to establish baseline measures before randomization

4 weeks

Double-Blind Period

Participants receive either clemizole HCl or placebo in a double-blind manner

16 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-Label Extension

Eligible participants may continue receiving clemizole HCl for up to 3 years

up to 156 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Clemizole HCl
• Placebo to match EPX-100

Trial Overview The study tests Clemizole HCl as an additional therapy against a placebo in people with LGS. It's a Phase 3 trial, meaning it's one of the final stages before potential approval if successful. The process is randomized and double-blind so neither participants nor researchers know who gets the real drug versus placebo.

How Is the Trial Designed?

Group I: Open-label clemizole HCl Experimental Treatment 2 Interventions

Eligible participants who complete the DB Period will have the option to continue in the OLE Period, during which they will receive clemizole HCl for up to 3 years.

Group II: Double-blind clemizole HCl Experimental Treatment 1 Intervention

Participants will receive their first dose of study drug following randomization.

Group III: Placebo Placebo Group 1 Intervention

Participants will receive their first dose of study drug following randomization.

Who Is Running the Clinical Trial?

Epygenix

Lead Sponsor

Trials

3

Recruited

150+

Harmony Biosciences Management, Inc.

Industry Sponsor

Trials

9

Recruited

2,000+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05066217?cond=%22Lennox%20Gastaut%20Syndrome%22&viewType=Table&rank=3

An Efficacy and Safety Study of Clemizole HCl in Patients ... This is a multicenter, Phase 3, randomized, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of clemizole HCl ...

2. neurology.org neurology.org/doi/10.1212/WNL.0000000000211366

A 20-Week Multicenter, Randomized, Double-Blind (DB), ... This is an upcoming Phase 3 study to determine effectiveness and safety of EPX-100 in patients with LGS.

3. neurologylive.com neurologylive.com/view/phase-3-study-test-1st-generation-antihistamine-epx-100-lennox-gastaut-syndrome

Phase 3 Study to Test 1st Generation Antihistamine EPX- ... EPX-100 is being tested for efficacy and safety in treating Lennox-Gastaut syndrome in a phase 3 study involving a 20-week period with multiple ...

4. trialx.com trialx.com/clinical-trials/listings/324189/an-efficacy-and-safety-study-of-clemizole-hcl-in-patients-with-lennox-gastaut-syndrome/

An Efficacy and Safety Study of Clemizole HCl in Patients ... This is a multicenter, Phase 3, randomized, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of clemizole HCl ...

5. withpower.com withpower.com/trial/phase-3-syndrome-1-2022-7defd

Clemizole HCl for Lennox-Gastaut Syndrome (LGS Trial) This is a multicenter, Phase 3, randomized, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of clemizole HCL (EPX-100) ...

6. ir.harmonybiosciences.com ir.harmonybiosciences.com/news-releases/news-release-details/harmony-biosciences-present-rare-epilepsy-data-36th

Harmony Biosciences to Present Rare Epilepsy Data at the ... EPX-100, clemizole hydrochloride, is under development for the treatment of Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS). EPX-100 acts ...