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.
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
30
RAP-219 oral tablet
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
Change in long episode frequency during the treatment period compared to pre-treatment baseline: Responder proportion, Percent Change Percent change
Change in long episode frequency per 28-day period during the treatment period compared to baseline
Time frame: Screening until 5 months after enrollment
Change in focal clinical seizure frequency during the treatment period compared to pre-treatment baseline: Percent Change, Responder proportion
Change in focal clinical seizure frequency per 28-day period during the treatment period compared to baseline
Time frame: Screening until 5 months after enrollment
Change in frequency of estimated electrographic seizures based on RNS® system data during the treatment period compared to pre-treatment baseline
Change in the frequency per 28-day period of estimated electrographic seizures based on RNS data during the treatment period compared to baseline
Time frame: Screening until 5 months after enrollment
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
Change in long episode-free days during the treatment period compared to pre-treatment baseline
Time frame: Screening until 5 months after enrollment
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
Change in focal clinical seizure-free days during the treatment period compared to pre-treatment baseline
Time frame: Screening until 5 months after enrollment
Change in RNS® system electrographic biomarkers other than long episodes during the treatment period compared to pre-treatment baseline
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
The University of Kansas Medical Center Epilepsy Clinic
Kansas City, Kansas, 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
...and 2 more locations
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 the Seizure Severity Response Questionnaire (SSRQ) during the treatment period compared to pre-treatment baseline
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.
Time frame: Screening until 5 months after enrollment
Count and proportion of responders on the clinical global impression of change (CGIC) scale during the treatment period compared to pre-treatment baseline
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.
Time frame: Screening until 5 months after enrollment
Plasma concentrations of RAP-219
Analysis of plasma concentrations of RAP-219
Time frame: Dosing until 5 months after dosing
Incidence of treatment-emergent adverse events (TEAEs)
Analysis of AEs during the treatment and pre-treatment baseline periods
Time frame: Dosing until 5 months after enrollment
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
Analysis of vital signs, ECGs, CBC, serum chemistries and liver function tests during the treatment and pre-treatment baseline periods
Time frame: Screening until 5 months after enrollment
Incidence of participants expressing suicidal ideation assessed with the Columbia-Suicide Severity Rating Scale (C-SSRS)
Analysis of C-SSRS entries during the treatment and pre-treatment baseline periods
Time frame: Screening until 5 months after enrollment
Cognitive function as measured using the Cogstate Digital Battery
Analysis of the Cogstate Digital Battery during the treatment and pre-treatment baseline periods
Time frame: Screening until 5 months after enrollment