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

Focal Onset Seizures Clinical Trial

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06377930
• Other study ID #: RAP-219-FOS-201
• Status: Not yet recruiting
• Phase: Phase 2
• Start date: June 1, 2024
• Est. completion date: June 1, 2025

Study information

• Verified date: April 2024
• Source: Rapport Therapeutics Inc.
• Contact: Beth Bowers
• Phone: (857) 323-9048
• Email: bbowers[@]rapportrx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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 information / eligibility

• Status: Not yet recruiting
• Enrollment: 20
• Est. completion date: June 1, 2025
• Est. primary completion date: March 1, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 65 Years

Eligibility

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

Related Conditions & MeSH terms

• Epilepsies, Partial
• Epilepsy
• Focal Onset Seizures
• Seizures

Intervention

Drug:
• RAP-219
RAP-219 oral tablet

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Rapport Therapeutics Inc.

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Efficacy in reducing frequency of RNS-recorded long episodes in participants with FOS	Change in long episode frequency on treatment compared to baseline	Screening until 5 months after enrollment
Secondary	Measurement of estimated electrographic seizure frequency from available RNS-recorded episodes	Change in 28-day estimated electrographic seizure frequency	Screening until 5 months after enrollment
Secondary	Measurement of Clinical Seizure Frequency	Measurement of 28-day clinical Seizure Frequency by paper diary	Screening until 5 months after enrollment
Secondary	Change in seizure severity	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
Secondary	Global Impression of Change	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
Secondary	Pharmacokinetic parameters	Analysis of plasma concentrations of RAP-219	Dosing until 5 months after dosing