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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| RAP-219 | Experimental | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| RAP-219 | Drug | RAP-219 oral tablet |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 | Screening until 5 months after enrollment |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | 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 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Jaqueline A French, MD | NYU Langone Comprehensive Epilepsy Center | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Yale University | New Haven | Connecticut | 06519 | United States | ||
| Consultants in Epilepsy and Neurology, PLLC |
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Change in the frequency per 28-day period of estimated electrographic seizures based on RNS data during the treatment period compared to baseline |
| 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 | 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 | 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 | Change in RNS® system electrographic biomarkers other than long episodes during the treatment period compared to pre-treatment baseline | 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. | 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. | Screening until 5 months after enrollment |
| Plasma concentrations of RAP-219 | Analysis of plasma concentrations of RAP-219 | Dosing until 5 months after dosing |
| Incidence of treatment-emergent adverse events (TEAEs) | Analysis of AEs during the treatment and pre-treatment baseline periods | 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 | 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 | 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 | Screening until 5 months after enrollment |
| Boise |
| Idaho |
| 83702 |
| United States |
| IU Health Neuroscience Center, Goodman Hall | Indianapolis | Indiana | 46202 | United States |
| The University of Kansas Medical Center Epilepsy Clinic | Kansas City | Kansas | 66160 | United States |
| Mayo Clinic | Rochester | Minnesota | 55905 | United States |
| NYU Langone Comprehensive Epilepsy Center | New York | New York | 10016 | United States |
| Cleveland Clinic Foundation | Cleveland | Ohio | 44195 | United States |
| University of Pennsylvania - Department of Neurology | Philadelphia | Pennsylvania | 19104 | United States |
| Thomas Jefferson University | Philadelphia | Pennsylvania | 19107 | United States |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | United States |
| Baylor College of Medicine | Houston | Texas | 770300 | United States |
| University of Texas Health Science Center, Houston | Houston | Texas | 77030 | United States |
| ID | Term |
|---|---|
| D012640 | Seizures |
| D004827 | Epilepsy |
| D004828 | Epilepsies, Partial |
| ID | Term |
|---|---|
| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
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