Carisbamate Safety Study in Adult and Pediatric Subjects With Lennox-Gastaut Syndrome

Phase 1, Open-Label Study of Carisbamate in Adult and Pediatric Subjects With Lennox-Gastaut Syndrome

Open-label extension study from YKP509C001 to evaluate the safety and tolerability of carisbamate in subjects with Lennox-Gastaut Syndrome (LGS).

Study Overview

• Status: Completed
• Conditions: Lennox Gastaut Syndrome
• Intervention / Treatment: Drug: Carisbamate

Detailed Description

Subjects who successfully completed the YKP509C001 study and could benefit from continued exposure to carisbamate.

This is an open-label (OL), multi-center study of carisbamate in subjects with LGS, with safety assessments from Baseline (Visit 1) through the early termination (ET)/end of study (EOS) visit.

• Study Type: Interventional
• Enrollment (Actual): 15
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Maryland
    • Baltimore, Maryland, United States, 21210
      • Johns Hopkins Hospital
  • New Hampshire
    • Lebanon, New Hampshire, United States, 03756
      • Dartmouth-Hitchcock Medical Center
  • Oregon
    • Portland, Oregon, United States, 97239
      • Oregon Health and Science University
  • Utah
    • Salt Lake City, Utah, United States, 84113
      • The University of Utah School of Medicine - Primary Children's Hospital (Primary Children's Medical Center)
  • Washington
    • Renton, Washington, United States, 98055
      • UW Valley Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 8 months and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Subjects who completed the YKP509C001 study
• Investigator believes subject could benefit from continued exposure to study drug
• Subjects must continue to meet all of the inclusion criteria from the YKP509C001 study

Exclusion Criteria:

• Subjects must continue to not meet any of the exclusion criteria from the YKP509C001 study
• There are no additional exclusion criteria in this study

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Basic Science
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cohort I; Subjects ≥ 18 years of age These subjects will reach maximum stable dose and continue onto YKP509C002.	Drug: Carisbamate; An oral liquid formulation (20 mg/mL) of carisbamate (S-carisbamate); Other Names: YKP509
Experimental: Cohort II; Subjects 12 to <18 years of age These subjects will reach maximum stable dose and continue onto YKP509C002.	Drug: Carisbamate; An oral liquid formulation (20 mg/mL) of carisbamate (S-carisbamate); Other Names: YKP509
Experimental: Cohort III; Subjects 6 to <12 years of age These subjects will reach maximum stable dose and continue onto YKP509C002.	Drug: Carisbamate; An oral liquid formulation (20 mg/mL) of carisbamate (S-carisbamate); Other Names: YKP509
Experimental: Cohort IV; Subjects 2 to <6 years of age These subjects will reach maximum stable dose and continue onto YKP509C002.	Drug: Carisbamate; An oral liquid formulation (20 mg/mL) of carisbamate (S-carisbamate); Other Names: YKP509

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Concomitant medication	Safety	Up to 20 months
12-lead electrocardiograms (ECGs)	Safety	Up to 20 months
Physical examinations	Safety	Up to 20 months
Seizure Frequency	An assessment of seizure frequency will be made using a subject/caregiver seizure diary with seizure type and number of daily seizures recorded since the prior visit.	Up to 20 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety- adverse events	Adverse events assessment for seriousness (yes, no) severity (mild, moderate, severe), affect on carisbamate dosing (increase,reduced, interrupted, withdrawn, no change) and outcome (recovered/resolved, recovered/resolved with sequelae, recovering/resolving, not recovered/not resolved, fatal or unknown	The duration of this OL study will be until carisbamate bas been approved for treatment of LGS and is available by prescription, or development of carisbamate for LGS has stopped, whichever is first. This could occur up to 36 months.

Collaborators and Investigators

• Sponsor: SK Life Science, Inc.
• Investigators: Study Director: Marc Kamin, MD, SK Life Science, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): May 3, 2019
• Primary Completion (Actual): December 14, 2022
• Study Completion (Actual): December 14, 2022

Study Registration Dates

• First Submitted: May 30, 2019
• First Submitted That Met QC Criteria: August 19, 2019
• First Posted (Actual): August 20, 2019

Study Record Updates

• Last Update Posted (Actual): April 4, 2024
• Last Update Submitted That Met QC Criteria: April 2, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Epileptic Syndromes; Disease; Genetic Diseases, Inborn; Epilepsy; Syndrome; Lennox Gastaut Syndrome
• Other Study ID Numbers: YKP509C002

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No