Clinical trial NCT06377930: Study Evaluating the Safety and Efficacy of RAP-219 in Adult Participants With Refractory Focal Epilepsy
Official title: A Phase 2A, Multicenter, Open-label Study Evaluating the Safety and Efficacy of RAP-219 in Adult Participants With Refractory Focal Epilepsy
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: Focal Onset Seizures
Keywords: Epilepsy
RNS
long episode
FOS
Refractory focal epilepsy
Lead sponsor: Rapport Therapeutics Inc.
Sponsor class: INDUSTRY
First posted: 2024-04-22
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Efficacy in reducing frequency of RNS-recorded long episodes in participants with FOS
timeFrame: Screening until 5 months after enrollment
Secondary outcomes:
measure: Measurement of estimated electrographic seizure frequency from available RNS-recorded episodes
timeFrame: Screening until 5 months after enrollment
measure: Measurement of Clinical Seizure Frequency
timeFrame: Screening until 5 months after enrollment
measure: Change in seizure severity
timeFrame: Screening until 5 months after enrollment
measure: Global Impression of Change
timeFrame: Screening until 5 months after enrollment
measure: Pharmacokinetic parameters
timeFrame: Dosing until 5 months after dosing
Other outcomes:
Locations:
city: New Haven
country: United States
facility: Yale University
state: Connecticut
city: Boise
country: United States
facility: Consultants in Epilepsy and Neurology, PLLC
state: Idaho
city: Indianapolis
country: United States
facility: IU Health Neuroscience Center, Goodman Hall
state: Indiana
city: Kansas City
country: United States
facility: The University of Kansas Medical Center Epilepsy Clinic
state: Kansas
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: New York
country: United States
facility: NYU Langone Comprehensive Epilepsy Center
state: New York
city: Cleveland
country: United States
facility: Cleveland Clinic Foundation
state: Ohio
city: Philadelphia
country: United States
facility: University of Pennsylvania - Department of Neurology
state: Pennsylvania
city: Philadelphia
country: United States
facility: Thomas Jefferson University
state: Pennsylvania
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
city: Houston
country: United States
facility: University of Texas Health Science Center, Houston
state: Texas
Study officials:
affiliation: NYU Langone Comprehensive Epilepsy Center
name: Jaqueline A French, MD
role: PRINCIPAL_INVESTIGATOR
References: