Study Evaluating the Safety and Efficacy of RAP-219 in Adult Participants With Refractory Focal Epilepsy
- Registration Number
- NCT06377930
- Lead Sponsor
- Rapport Therapeutics Inc.
- Brief Summary
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.
- Detailed Description
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 20 participants treated with the RNS® system.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 20
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description RAP-219 RAP-219 Participants will receive 0.75 mg RAP-219 daily for 5 days followed by 1.25 mg RAP-219 daily for the remainder of the 8-week treatment period.
- Primary Outcome Measures
Name Time Method Efficacy in reducing frequency of RNS-recorded long episodes in participants with FOS Screening until 5 months after enrollment Change in long episode frequency on treatment compared to baseline
- Secondary Outcome Measures
Name Time Method Pharmacokinetic parameters Dosing until 5 months after dosing Analysis of plasma concentrations of RAP-219
Measurement of estimated electrographic seizure frequency from available RNS-recorded episodes Screening until 5 months after enrollment Change in 28-day estimated electrographic seizure frequency
Change in seizure severity 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.
Measurement of Clinical Seizure Frequency Screening until 5 months after enrollment Measurement of 28-day clinical Seizure Frequency by paper diary
Global Impression of Change 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.
Trial Locations
- Locations (14)
Emory Brain Health Center
🇺🇸Atlanta, Georgia, United States
Yale University
🇺🇸New Haven, Connecticut, United States
Consultants in Epilepsy and Neurology, PLLC
🇺🇸Boise, Idaho, United States
IU Health Neuroscience Center, Goodman Hall
🇺🇸Indianapolis, Indiana, United States
The University of Kansas Medical Center Epilepsy Clinic
🇺🇸Kansas City, Kansas, United States
Corewell Health Research Institute
🇺🇸Grand Rapids, Michigan, United States
Mayo Clinic
🇺🇸Rochester, Minnesota, United States
NYU Langone Comprehensive Epilepsy Center
🇺🇸New York, New York, United States
Cleveland Clinic Foundation
🇺🇸Cleveland, Ohio, United States
University of Pennsylvania - Department of Neurology
🇺🇸Philadelphia, Pennsylvania, United States
Thomas Jefferson University
🇺🇸Philadelphia, Pennsylvania, United States
Vanderbilt University Medical Center
🇺🇸Nashville, Tennessee, United States
Baylor College of Medicine
🇺🇸Houston, Texas, United States
University of Texas Health Science Center, Houston
🇺🇸Houston, Texas, United States