Clemizole HCl for Lennox-Gastaut Syndrome

(LGS Trial)

Recruiting at 1 trial location

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

Trial Summary

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.

What is the purpose of this 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) as adjunctive therapy in children and adult participants with Lennox-Gastaut syndrome (LGS).

Research Team

Amit Ray, MD

Principal Investigator

Harmony Biosciences Management, Inc.

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+