MedPath

A Trial of the Efficacy and Safety of CVL-865 as Adjunctive Therapy in the Treatment of Focal Onset Seizures

Phase 2
Completed
Conditions
Seizures
Interventions
Drug: Placebo
Registration Number
NCT04244175
Lead Sponsor
Cerevel Therapeutics, LLC
Brief Summary

The purpose of this study is to assess the efficacy, safety, and tolerability profile of CVL-865 as adjunctive treatment in participants with drug-resistant focal onset seizures.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
154
Inclusion Criteria
  • Participants with a diagnosis of epilepsy with focal onset, as defined in the International League Against Epilepsy (ILAE) Classification of Seizures, focal aware (except participants with only focal aware seizures without a motor component), focal impaired awareness, and focal to bilateral tonic-clonic seizures for at least 2 years prior to signing the Informed Consent Form (ICF)
  • Participants must have history of an average of 4 or more spontaneous and observable focal onset, as defined in the ILAE Classification of Seizures, focal aware (except participants with only focal aware seizures without a motor component), focal impaired awareness, and focal to bilateral tonic-clonic seizures per 28-day period for at least 3 months (84 days) prior to signing the ICF
  • Participants who have tried and failed at least 2 appropriate Anti- epileptic drugs (AEDs) in the past and also currently taking 1 to 3 permitted AEDs at a stable dose for 4 Weeks prior to the Screening Visit
  • Participants with a minimum of 8 focal onset, focal aware, focal impaired awareness, or focal to bilateral tonic-clonic seizures during the 8 week baseline period with no 21-day period free of any of these seizure types
  • Participants must have had magnetic resonance imaging or contrast enhance computed tomography scan of the brain that demonstrated no progressive structural central nervous system abnormality at the time of the diagnosis of epilepsy
  • Participants must have a body mass index (BMI) of 17.5 to 40.0 kilogram per meter square (kg/m^2) and a total body weight greater than (>) 50 kilograms (kg) [110 pounds (lbs)]
  • Women of childbearing potential must agree to use an effective method of contraception from signing of informed consent throughout the duration of the study and for 30 days post last dose
  • Male must agree to use condom during treatment and until the end of relevant systemic exposure in the male participant for 94 days following the last dose with Investigational Manufacturing Product (IMP)
Exclusion Criteria
  • Participants with (genetic) idiopathic generalized epilepsies or combined generalized and focal epilepsies, including a history of Lennox-Gastaut Syndrome
  • Participants with a history of seizures over the past 12 months that occur at such a high frequency they cannot be counted (eg, repetitive seizures, cluster seizures)
  • Participants with a history of psychogenic non-epileptic seizures within the year prior to signing the ICF
  • Participants with a history of status epilepticus within 5 years prior to signing the ICF
  • Participants with a history of neurosurgery for seizures less than 1 year prior to signing the ICF, or radiosurgery less than 2 years prior to signing the ICF
  • Participants with a current history of significant cardiovascular, pulmonary, gastrointestinal, renal, hepatic, metabolic, hematological, immunological, or neurological (excluding focal onset epilepsy) disease
  • Participants who test positive for human immunodeficiency virus (HIV), hepatitis B and/or or hepatitis C infection
  • Participants with a 12-lead ECG demonstrating : QT interval corrected for heart rate using Fridericia's formula >450 milliseconds (msec) (average of 3 ECGs obtained at the Screening Visit); QRS interval >120 msec at the Screening Visit assessed by central reader
  • Participants with abnormal laboratory test results which includes (Aspartate aminotransferase (AST) or Alanine aminotransferase (ALT) elevated to >2 × Upper limit of normal range (ULN); Total bilirubin greater than or equal to (>=)1.5 × ULN; Females: Hemoglobin <11 gram per deciliter (g/dL); Males: hemoglobin <12 g/dL; White blood cell (WBC) count <3.0 x 10 power 9 per liter (10^9/L); Neutrophil count <2.0 x 10^9/L; Platelet count <150 × 10^9/L
  • Use of prohibited medications as listed in the protocol in the absence of appropriate washout phase or the likelihood of requiring treatment during the study period with drugs not permitted by the study protocol
  • Participants taking any drug that is a sensitive P-glycoprotein (P-gp) and Breast cancer resistance protein (BCRP) substrate
  • Female participants who are breastfeeding and/or who have a positive pregnancy test result prior to receiving IMP
  • Participants who are known to be allergic or hypersensitive to the IMP or any of its components
  • Participants who have participated in any clinical trial within 60 days prior to signing the ICF or who have participated in more than 2 clinical trials within the year prior to signing the ICF
  • Participants with difficulty swallowing
  • Participants who answer "Yes" on the C-SSRS Suicidal Ideation Item 4 (Active Suicidal Ideation with Some Intent to Act, Without Specific Plan) and whose most recent episode meeting criteria for this C-SSRS Item 4 occurred within the last 6 months, OR Subjects who answer "Yes" on the C-SSRS Suicidal Ideation Item 5 (Active Suicidal Ideation with Specific Plan and Intent) and whose most recent episode meeting criteria for this CSSRS Item 5 occurred within the last 6 months OR Subjects who answer "Yes" on any of the 5 C-SSRS Suicidal Behavior Items (actual attempt, interrupted attempt, aborted attempt, preparatory acts, or behavior) and whose most recent episode meeting criteria for any of these 5 C-SSRS Suicidal Behavior Items occurred in the last 2 years

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
PlaceboPlaceboParticipants will receive a placebo matched to CVL-865 tablets orally BID until Day 92 during the treatment period.
Low Dose: CVL-865 7.5 mgCVL-865Participants will receive CVL-865 tablets orally BID up to the maximum dose of 7.5 mg until Day 92 during the treatment period.
High Dose: CVL-865 25 mgCVL-865Participants will receive CVL-865 tablets orally twice daily (BID) up to the maximum dose of 25 milligrams (mg) until Day 92 during the treatment period.
Primary Outcome Measures
NameTimeMethod
Response Ratio (RRatio)Day 71

Response Ratio (RRatio), calculated as RRatio=(T-B)/(T+B) ×100, where T represents the focal onset seizure frequency rate per week in the Maintenance Phase and B represents the focal onset seizure frequency rate per week in the Baseline Period.

Secondary Outcome Measures
NameTimeMethod
Change from Baseline in Clinical Global Impression-Severity of Symptoms Scale (CGI-S) Score at Day 15, 43 and 71Baseline, Day 15, 43 and 71

The CGI-S is an observer-rated scale that will be used to measure symptom severity. It is a 7-point scale depicting a participants rating of overall improvement. Participants rate their change as 0 = not assessed; 1 = normal, not at all ill; 2 = borderline ill; 3 = mildly ill; 4 = moderately ill; 5 = markedly ill; 6 = severely ill; and 7 = among the most extremely ill participants.

Seizure Rate over TimeBaseline up to Day 71

Seizure frequency is defined as the total number of focal onset seizures over the treatment period of interest divided by the total number of days with no missing seizure counts in the corresponding period multiplied by 7.

Percentage of Participants with 50 Percent (%) Responder RateDay 71

Defined as the percent of participants with at least a 50% reduction in the Maintenance Phase focal onset seizure frequency rate relative to the Baseline Period.

Change From Baseline in Focal Onset Seizure Frequency per Week over the Maintenance PhaseBaseline up to Day 71

Seizure frequency is defined as the total number of focal onset seizures over the treatment period of interest divided by the total number of days with no missing seizure counts in the corresponding period multiplied by 7.

Percentage of Seizure-free ParticipantsBaseline up to Day 71

Seizure freedom is defined as the absence of all seizure regardless of seizure type.

Patient's Global Impression of Change (PGIC) Score at Days 15, 43 and 71Baseline, Day 15, 43 and 71

The self-report measure Patient's Global Impression of Change (PGIC) reflects a participant's belief about the efficacy of treatment. It is a 7-point scale depicting a participant's rating of overall improvement where 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse and 7 = very much worse.

Change From Baseline in Clinical Global Impression-Improvement Scale (CGI-I) Score at Day 15, 43 and 71Baseline, Day 15, 43 and 71

The CGI-I is an observer-rated scale that will be used to measure the participant's symptom severity compared with before initiation of treatment with IMP. It is a 7-point scale depicting a participant's change from baseline in symptom severity using the following response choices: 0 = not assessed, 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, and 7 = very much worse.

Change from Baseline in Quality of Life in Epilepsy -31 (QOLIE-31) Overall Score at Day 71Baseline, Day 71

The Quality of Life in Epilepsy - 31 (QOLIE-31) contains 7 multi-item scales that tap the following health concepts: emotional well-being, social functioning, energy/fatigue, cognitive functioning, seizure worry, medication effects, and overall quality of life. A QOLIE-31 overall score is obtained using a weighted average of the multi-item scale scores. The QOLIE-31 also includes a single item that assessed overall health. The QoLIE-31 score range is from 0 to 100 with a higher score indicating a better outcome for quality of life.

Change from Baseline in Health Utilities Index (HUI) Utility Score at Day 71Baseline, Day 71

The Health Utilities Index (HUI) is a rating scale used to measure general health status and health-related quality of life. In HUI, utility values range from -0.03 and -0.36 for the HUI-2 and HUI-3, respectively, to 1.00. A health utility value of 1.00 indicates perfect health while a score of 0.00 indicates death.

Number of Participants with Clinically Significant Changes in Electrocardiogram (ECGs)Baseline to Day 92 or early termination

12-lead ECGs recordings will be obtained after the participant has been supine and at rest for at least 5 minutes.

Number of Participants with Clinically Significant Changes in Vital Sign MeasurementsBaseline to Day 92 or early termination (ET)

Vital signs will be measured with the participant in a sitting/semi-recumbent position after 5 minutes rest and will include temperature, systolic and diastolic blood pressure, and heart rate.

Number of Participants with Clinically Significant Changes in Physical and Neurological Examination ResultsBaseline to Day 92 or early termination (ET)

Number of participants with clinically significant changes in physical and neurological examination results will be assessed.

Number of Participants with Positive Response to Modified Clinical Institute Withdrawal Assessment - Benzodiazepines (mCIWA-B)Day 71 up to Day 120

The modified Clinical Institute Withdrawal Assessment - Benzodiazepines (mCIWA-B) is a sensitive instrument to measure withdrawal under conditions where there is a taper of medication (rather than abrupt discontinuation). It consists of 17-items that monitor the type and severity of BZD withdrawal symptoms such as irritability, fatigue, appetite, and sleeplessness. The total score ranges from 1 to 68 with higher scores indicating more severe withdrawal.

Number of Participants With Positive Response to Columbia Suicide-Severity Rating Scale (C-SSRS)From first dose of study drug up to Day 120 (follow up period)

The C-SSRS rates an individual's degree of suicidal ideation (SI) on a scale, ranging from "wish to be dead" to "active suicidal ideation with specific plan and intent." The scale identifies SI severity and intensity, which may be indicative of an individual's intent to commit suicide. C-SSRS SI severity subscale ranges from 0 (no SI) to 5 (active SI with plan and intent).

Number of Participants With Treatment Emergent Adverse Event (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs)From first dose of study drug up to Day 120 (follow up period)

TEAEs will include abuse-related AEs and AEs related to medication handling irregularities (MHIs). Number of Participants With TEAEs and TESAEs will be assessed.

Plasma Concentrations of CVL-865Day 15, Day 43, Day 71, Day 92 and/or early termination (ET)

Plasma concentration of CVL-865 at Day 15, Day 43, Day 71, Day 92 and/or early termination (ET) will be assessed.

Trial Locations

Locations (72)

Miami, Florida

🇺🇸

Miami, Florida, United States

Philadelphia, Pennsylvania

🇺🇸

Philadelphia, Pennsylvania, United States

Downey, California

🇺🇸

Downey, California, United States

Altamonte Springs, Florida

🇺🇸

Altamonte Springs, Florida, United States

Gulf Breeze, Florida

🇺🇸

Gulf Breeze, Florida, United States

Jacksonville, Florida

🇺🇸

Jacksonville, Florida, United States

Suwanee, Georgia,

🇺🇸

Suwanee, Georgia, United States

Scarborough, Maine

🇺🇸

Scarborough, Maine, United States

Boston, Massachusetts

🇺🇸

Boston, Massachusetts, United States

Hackensack, New Jersey

🇺🇸

Hackensack, New Jersey, United States

Rochester, New York

🇺🇸

Rochester, New York, United States

Toledo, Ohio

🇺🇸

Toledo, Ohio, United States

Syracuse, New York

🇺🇸

Syracuse, New York, United States

Columbus, Ohio

🇺🇸

Columbus, Ohio, United States

Camperdown, New South Wales

🇦🇺

Camperdown, New South Wales, Australia

Randwick, New South Wales

🇦🇺

Randwick, New South Wales, Australia

Westmead, New South Wales

🇦🇺

Westmead, New South Wales, Australia

Herston, Queensland

🇦🇺

Herston, Queensland, Australia

South Brisbane, Queensland

🇦🇺

South Brisbane, Queensland, Australia

Fitzroy, Victoria

🇦🇺

Fitzroy, Victoria, Australia

Melbourne, Victoria

🇦🇺

Melbourne, Victoria, Australia

Heidelberg, Victoria

🇦🇺

Heidelberg, Victoria, Australia

Gwangjin-gu, Seoul

🇰🇷

Gwangju, Seoul, Korea, Republic of

Bydgoszcz, Kujawsko-Pomorskie

🇵🇱

Bydgoszcz, Kujawsko-Pomorskie, Poland

Irwon-Ro Gangnam-gu., Seoul

🇰🇷

Irwon-dong, Seoul, Korea, Republic of

Parkville, Victoria

🇦🇺

Parkville, Victoria, Australia

Lodz

🇵🇱

Łódź, Lodz, Poland

Kraków, Malopolskie

🇵🇱

Kraków, Malopolskie, Poland

Warszawa, Mazowieckie

🇵🇱

Warszawa, Mazowieckie, Poland

Wojnicz, Lskie

🇵🇱

Wojnicz, Wojnicz Lskie, Poland

Warszawa

🇵🇱

Warszawa, Poland

Kragujevac, Sumadija

🇷🇸

Kragujevac, Sumadija, Serbia

Clinic of Neurology, Belgrade

🇷🇸

Belgrade, Serbia

Niš

🇷🇸

Niš, Serbia

Barcelona, Catalunya

🇪🇸

Barcelona, Catalonia, Spain

Malaga,

🇪🇸

Málaga, Andalusia, Spain

Terrassa

🇪🇸

Terrassa, Catalonia, Spain

Uzhgorod

🇺🇦

Úzhgorod, Uzhgorod, Ukraine

Kyiv

🇺🇦

Kyiv, Ukraine

Lviv

🇺🇦

Lviv, Ukraine

Loma Linda, California

🇺🇸

Loma Linda, California, United States

Valencia, California

🇺🇸

Valencia, California, United States

Chesterfield, Missouri

🇺🇸

Chesterfield, Missouri, United States

Lexington, Kentucky

🇺🇸

Lexington, Kentucky, United States

Mineola, New York

🇺🇸

Mineola, New York, United States

Saint Louis, Missouri

🇺🇸

Saint Louis, Missouri, United States

Nashville, Tennessee

🇺🇸

Nashville, Tennessee, United States

Barcelona

🇪🇸

Barcelona, Spain

New York

🇺🇸

New York, New York, United States

Valencia

🇪🇸

Valencia, Spain

Belgrade

🇷🇸

Belgrade, Serbia

Navarra

🇪🇸

Navarro, Navarra, Spain

Madrid

🇪🇸

Madrid, Spain

Sevilla

🇪🇸

Sevilla, Spain

Białystok

🇵🇱

Białystok, Poland

Charleston, South Carolina

🇺🇸

Charleston, South Carolina, United States

New Haven, Connecticut

🇺🇸

New Haven, Connecticut, United States

Port Orange, Florida

🇺🇸

Port Orange, Florida, United States

Port Charlotte, Florida

🇺🇸

Port Charlotte, Florida, United States

Homestead, Florida

🇺🇸

Homestead, Florida, United States

Miami Lakes, Florida

🇺🇸

Miami Lakes, Florida, United States

Gdańsk, Pomorskie

🇵🇱

Gdańsk, Pomorskie, Poland

Baltimore, Maryland

🇺🇸

Baltimore, Maryland, United States

Neurology Department, Kragujevac

🇷🇸

Kragujevac, Sumadija, Serbia

Salt Lake City, Utah

🇺🇸

Salt Lake City, Utah, United States

Little Rock, Arkansas

🇺🇸

Little Rock, Arkansas, United States

Orlando, Florida

🇺🇸

Orlando, Florida, United States

Lublin

🇵🇱

Lublin, Poland

Tampa, Florida

🇺🇸

Tampa, Florida, United States

Bethesda, Maryland

🇺🇸

Bethesda, Maryland, United States

Oklahoma City, Oklahoma

🇺🇸

Oklahoma City, Oklahoma, United States

Honolulu, Hawaii

🇺🇸

Honolulu, Hawaii, United States

© Copyright 2025. All Rights Reserved by MedPath