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Completed

NCT Number: NCT06377930

Study Evaluating the Safety and Efficacy of RAP-219 in Adult Participants With Refractory Focal Epilepsy

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 determine if RAP-219 works and is safe in patients with Refractory Focal Epilepsy.

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Key information

Conditions

Focal Onset Seizures Brain Diseases Central Nervous System Diseases Epilepsies, Partial Epilepsy Nervous System Diseases Neurologic Manifestations Pathological Conditions, Signs and Symptoms Seizures Signs and Symptoms

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Yale University, New Haven, Connecticut, United States

Loading trial locations.

About this study

This is a Phase 2A, proof-of-concept, multi-center, open-label study designed to evaluate the efficacy, safety, tolerability, and PK/PD relationship of RAP-219 in adult participants with refractory focal epilepsy. The activity of RAP-219 will be assessed in approximately 30 participants treated with the RNS® system.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Medically refractory focal epilepsy with an implanted responsive neurostimulator device placed correctly in a seizure onset zone that meet several device-related criteria and demonstrated history of compliance with interrogation of the RNS device and upload of data
  • If currently treated with antiseizure medications, up to a maximum of 4 concomitant mediations are allowed
  • At least 1 clinical seizure during the 8-week retrospective eligibility period
  • Participants in otherwise good health as determined by the investigator
  • Willing and able to adhere to all aspects of the protocol
  • A demonstrated history of compliance with interrogation of the RNS device and upload of data before screening

Exclusion criteria

  • Participants with generalized onset seizures in the past 10 years
  • History of status epilepticus while on antiseizure medications within 2 years of screening
  • Individuals of reproductive potential who do not agree to simultaneously use two effective birth-control methods
  • Participants who have had epilepsy surgery within the last 12 months before screening

Treatment and study plan

RAP-219

Drug

RAP-219 oral tablet

Primary outcomes

  1. Change in long episode frequency during the treatment period compared to pre-treatment baseline: Responder proportion, Percent Change Percent change

    Time frame: Screening until 5 months after enrollment

    Change in long episode frequency per 28-day period during the treatment period compared to baseline

Secondary outcomes

  1. Change in focal clinical seizure frequency during the treatment period compared to pre-treatment baseline: Percent Change, Responder proportion

    Time frame: Screening until 5 months after enrollment

    Change in focal clinical seizure frequency per 28-day period during the treatment period compared to baseline

  2. Change in frequency of estimated electrographic seizures based on RNS® system data during the treatment period compared to pre-treatment baseline

    Time frame: Screening until 5 months after enrollment

    Change in the frequency per 28-day period of estimated electrographic seizures based on RNS data during the treatment period compared to baseline

  3. Change in RNS® system long episode-free days during the treatment period compared to pre-treatment baseline: Count, Longest continuous interval, Time until pre-treatment count

    Time frame: Screening until 5 months after enrollment

    Change in long episode-free days during the treatment period compared to pre-treatment baseline

  4. Change in focal clinical seizure-free days during the treatment period compared to pre-treatment baseline: Count, Longest continuous interval, Time until pre-treatment count

    Time frame: Screening until 5 months after enrollment

    Change in focal clinical seizure-free days during the treatment period compared to pre-treatment baseline

  5. Change in RNS® system electrographic biomarkers other than long episodes during the treatment period compared to pre-treatment baseline

    Time frame: Dosing until 5 months after dosing

    Change in RNS® system electrographic biomarkers other than long episodes during the treatment period compared to pre-treatment baseline

  6. Change in the Seizure Severity Response Questionnaire (SSRQ) during the treatment period compared to pre-treatment baseline

    Time frame: Screening until 5 months after enrollment

    Measurement of seizure severity by participant rating scale of the Seizure Severity Scale. Seizure severity will be assessed by asking participants to respond to 4 questions using a 0-10 numerical rating scale and circling the number that best describes how their seizures have impacted them since their last study visit. Higher scores = higher impact.

  7. Count and proportion of responders on the clinical global impression of change (CGIC) scale during the treatment period compared to pre-treatment baseline

    Time frame: Screening until 5 months after enrollment

    Change in overall clinical status as measured by Clinical Global Impression of Change scores rated by investigator and participant. Both the participant (PGIC) and the clinician (CGIC) will rate their global impression of changes in the participant's condition throughout the study. The CGI scale measures the change in the participant's clinical status from a specific point in time, i.e., the Baseline Period and is rated on a 7-point Likert scale ranging from a score of 1 for very much improved to 7 for very much worse. Higher scores = worse outcome.

  8. Plasma concentrations of RAP-219

    Time frame: Dosing until 5 months after dosing

    Analysis of plasma concentrations of RAP-219

  9. Incidence of treatment-emergent adverse events (TEAEs)

    Time frame: Dosing until 5 months after enrollment

    Analysis of AEs during the treatment and pre-treatment baseline periods

  10. Clinically meaningful changes, if any, from baseline in vital signs, electrocardiograms (ECGs), complete blood counts, serum chemistries, or liver function tests after treatment with RAP-219

    Time frame: Screening until 5 months after enrollment

    Analysis of vital signs, ECGs, CBC, serum chemistries and liver function tests during the treatment and pre-treatment baseline periods

  11. Incidence of participants expressing suicidal ideation assessed with the Columbia-Suicide Severity Rating Scale (C-SSRS)

    Time frame: Screening until 5 months after enrollment

    Analysis of C-SSRS entries during the treatment and pre-treatment baseline periods

  12. Cognitive function as measured using the Cogstate Digital Battery

    Time frame: Screening until 5 months after enrollment

    Analysis of the Cogstate Digital Battery during the treatment and pre-treatment baseline periods

Sponsors and collaborators

Lead sponsor

Rapport Therapeutics Inc.

Industry

Registry information

Official study title

A Phase 2A, Multicenter, Open-label Study Evaluating the Safety and Efficacy of RAP-219 in Adult Participants With Refractory Focal Epilepsy

Important dates

Study start
2024
Primary completion
2025
Study completion
2025
First posted
Apr 22, 2024
Registry last updated
Feb 5, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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