RAP-219 for Epilepsy

Recruiting at 6 trial locations

Overseen By Beth Bowers

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Rapport Therapeutics Inc.

Disqualifiers: Hypersensitivity to RAP-219, Pregnancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

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

Compensation: Varies

Number of Visits: Unknown

30 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test the safety and effectiveness of an experimental drug called RAP-219 for individuals with Refractory Focal Epilepsy, a type of epilepsy that doesn't respond well to standard treatments. The researchers focus on assessing how well RAP-219 can control seizures over an extended period. Those who participated in a previous study with RAP-219, have a stable responsive neurostimulation (RNS) system setting, and are generally healthy aside from epilepsy might be suitable candidates for this trial. As a Phase 2 trial, the research measures the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to important findings.

Do I need to stop my current medications to join the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor.

Is there any evidence suggesting that RAP-219 is likely to be safe for humans?

In earlier studies, RAP-219 has shown promising safety results for people with epilepsy. Research indicates that RAP-219 was well-tolerated in early trials, with participants generally experiencing positive outcomes. Specifically, it successfully reached the target areas in the brain without causing major issues, supporting its continued development for treating focal epilepsy.

Additionally, studies have reported that RAP-219 consistently provided meaningful improvement in patients, regardless of the initial severity of their condition. While this information is encouraging, ongoing research remains necessary to fully understand its long-term safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

RAP-219 is unique because it targets epilepsy with a novel mechanism of action that differentiates it from standard treatments like antiepileptic drugs (AEDs) such as levetiracetam or valproic acid, which primarily work by stabilizing neuronal activity. Researchers are excited about RAP-219 because it may offer improved seizure control by modulating specific pathways involved in epilepsy that current medications do not target. This new approach has the potential to provide better outcomes for patients who do not respond well to existing therapies.

What evidence suggests that RAP-219 might be an effective treatment for Refractory Focal Epilepsy?

Research has shown that RAP-219, the investigational treatment in this trial, can be highly effective for individuals with Refractory Focal Epilepsy. In previous studies, participants taking RAP-219 experienced a 77.8% reduction in seizures compared to their baseline. Notably, 24% of these participants remained seizure-free for eight weeks. Additionally, RAP-219 reduced seizure frequency and shortened prolonged episodes, as measured by a specialized brain device. These findings suggest RAP-219 has strong potential to help manage seizures in people with this type of epilepsy. ^{1 2 6 7 8}

Who Is on the Research Team?

Imran Quraishi, MD

Principal Investigator

Yale University

Are You a Good Fit for This Trial?

This trial is for adults with refractory focal epilepsy who completed the parent study RAP-219-FOS-201 with good tolerance. Participants should be in overall good health, have a BMI between 18 and 45 kg/m^2, stable RNS(c) system settings, and a history of following RNS(c) data procedures.

Inclusion Criteria

Aside from focal epilepsy, I am in good health.

My RNS system settings have not changed recently.

I regularly upload and share my RNS system data as required.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Open-label Treatment

Participants receive RAP-219 to evaluate long-term safety and antiseizure activity

Up to 112 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

8 weeks

What Are the Treatments Tested in This Trial?

Interventions

• RAP-219

Trial Overview

The study tests the long-term safety and effectiveness of an investigational drug called RAP-219 for patients with Refractory Focal Epilepsy. It aims to observe how well this drug can control seizures over an extended period.

How Is the Trial Designed?

Group I: RAP-219 Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Rapport Therapeutics Inc.

Lead Sponsor

Trials

1

Recruited

20+

Citations

1.

investors.rapportrx.com

investors.rapportrx.com/news-releases/news-release-details/rapport-therapeutics-announces-new-data-and-post-hoc-analysis

Release Details

Study participants achieved a 77.8% reduction in clinical seizures compared with baseline (p=0.01), with 24% achieving seizure freedom during ...

2.

investors.rapportrx.com

investors.rapportrx.com/news-releases/news-release-details/rapport-announces-positive-topline-results-phase-2a-clinical

Rapport Announces Positive Topline Results from Phase ...

In the trial, RAP-219 also demonstrated a statistically significant and clinically meaningful reduction in clinical seizures compared with ...

3.

clinicaltrials.gov

clinicaltrials.gov/study/NCT07219407

NCT07219407 | A Long-term Study of the Safety and ...

This is a clinical research study for an investigational drug called RAP-219 in patients with Refractory Focal Epilepsy. This study is being conducted to ...

4.

neurologylive.com

neurologylive.com/view/rap-219-meets-primary-end-point-phase-2a-trial-focal-onset-seizures

RAP-219 Meets Primary End Point in Phase 2a Trial for ...

Patients treated with RAP-219 had a 77.8% reduction in clinical seizures, with 24% achieving seizure freedom over 8 weeks, potentially ...

5.

aesnet.org

aesnet.org/abstractslisting/efficacy-and-tolerability-of-rap-219-a-potential-first-in-class-negative-allosteric-modulator-of-%CE%B38-transmembrane-ampa-receptor-associated-protein-impact-on-rns-long-episodes-and-focal-seizures

efficacy and tolerability of rap-219, a potential first-in-class ...

Recent analyses suggest a ≥30% reduction in long episodes (LEs) as measured by the responsive neurostimulator (RNS® System, NeuroPace) is ...

6.

investors.rapportrx.com

investors.rapportrx.com/news-releases/news-release-details/rapport-therapeutics-present-new-phase-2a-data-analysis-further

Rapport Therapeutics to Present New Phase 2a Data ...

New analysis examines RAP-219's effect during the first month of treatment and consistency of efficacy over the treatment period, effectiveness across baseline ...

7.

neurologylive.com

neurologylive.com/view/epilepsy-agent-rap-219-promising-safety-target-occupancy-across-phase-1-studies

Epilepsy Agent RAP-219 Shows Promising Safety, Target ...

RAP-219 achieved target receptor occupancy and favorable tolerability in phase 1 trials, supporting its development for focal epilepsy. The PET ...

8.

clinicaltrial.be

clinicaltrial.be/en/details/452843?per_page=20&only_recruiting=0&only_eligible=0&only_active=0

Study Evaluating the Safety and Efficacy of RAP-219 in Ad...

This is a Phase 2A, proof-of-concept, multi-center, open-label study designed to evaluate the efficacy, safety, tolerability, and PK/PD ...