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topiramate

Phase 3

Binge Eating | Small molecule | Psychiatry |Johnson & Johnson|Last Updated: Apr 29, 2025

Target and mechanism

ModalitySmall molecule

Also known as Topiramate and Naltrexone, topiramate, propranolol, topiramate, phenytoin

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindUNCONTROLLED
Total Trials1
Total Enrollment73

FDA Designations

No designations recorded

Clinical trial landscape

topiramate · 54 trials · 28 indications

Phase 3 32Phase 2 18Phase 1 4
NCT02201251A Study to Investigate the Safety of the Drugs Topiramate and Levetiracetam in Treating Children Recently Diagnosed With EpilepsyEpilepsy
COMPLETED63 Analytics
NCT00113815Topiramate as Adjunctive Therapy in Infants 1-24 Months for the Control of Partial Onset SeizuresPartial Seizure Disorder
COMPLETED118 Analytics
NCT00210860An Open Label Extension of a Study Comparing Topiramate and Amitriptyline in Migraine Prevention.Migraine
COMPLETED142 Analytics
NCT00210782A Comparison of the Effectiveness and Safety of Topiramate and Phenytoin in Patients With New Onset Epilepsy Requiring Rapid Initiation of Antiepileptic Drug TreatmentEpilepsy
COMPLETED262 Analytics
NCT00210873An Open Label Extension of a Study of Topiramate in Chronic Migraine.Migraine
COMPLETED200 Analytics
NCT00210821Comparing the Safety and Effectiveness of Topiramate With the Safety and Effectiveness of Amitriptyline in Preventing Migraine HeadachesMigraine
COMPLETED347 Analytics
NCT00216606The Effectiveness and Safety of Topiramate on Prevention of Chronic MigraineMigraine
COMPLETED59 Analytics
NCT00216619The Prolonged Use of Topiramate for Preventing Migraine HeadachesMigraine
COMPLETED834 Analytics
NCT00307619An Efficacy and Tolerability Study for Topiramate in Obese Patients With Binge Eating Disorder.Binge Eating
COMPLETED73 Analytics
NCT00210912A Study of the Effectiveness and Safety of Topiramate Versus Placebo for Preventing Chronic Migraine HeadachesMigraine
COMPLETED328 Analytics
PHASE3COMPLETED
A Study to Investigate the Safety of the Drugs Topiramate and Levetiracetam in Treating Children Recently Diagnosed With Epilepsy
EpilepsyUnlock trial analytics
PHASE3COMPLETED
Topiramate as Adjunctive Therapy in Infants 1-24 Months for the Control of Partial Onset Seizures
Partial Seizure DisorderUnlock trial analytics
PHASE3COMPLETED
An Open Label Extension of a Study Comparing Topiramate and Amitriptyline in Migraine Prevention.
MigraineUnlock trial analytics
PHASE3COMPLETED
A Comparison of the Effectiveness and Safety of Topiramate and Phenytoin in Patients With New Onset Epilepsy Requiring Rapid Initiation of Antiepileptic Drug Treatment
EpilepsyUnlock trial analytics
PHASE3COMPLETED
An Open Label Extension of a Study of Topiramate in Chronic Migraine.
MigraineUnlock trial analytics
PHASE3COMPLETED
Comparing the Safety and Effectiveness of Topiramate With the Safety and Effectiveness of Amitriptyline in Preventing Migraine Headaches
MigraineUnlock trial analytics
PHASE3COMPLETED
The Effectiveness and Safety of Topiramate on Prevention of Chronic Migraine
MigraineUnlock trial analytics
PHASE3COMPLETED
The Prolonged Use of Topiramate for Preventing Migraine Headaches
MigraineUnlock trial analytics
PHASE3COMPLETED
An Efficacy and Tolerability Study for Topiramate in Obese Patients With Binge Eating Disorder.
Binge EatingUnlock trial analytics
PHASE3COMPLETED
A Study of the Effectiveness and Safety of Topiramate Versus Placebo for Preventing Chronic Migraine Headaches
MigraineUnlock trial analytics

Study Endpoints

Primary Endpoints

Change From Baseline in Weight Z-score up to Month 1
Baseline up to Month 1

The Z-Score indicates how many standard deviations (SD) a participant has from the population normal values. The body weight z-scores were designed to take into account the amount of weight gain that was expected due to normal growth in children and adolescents. Body weight data were converted to Z-scores using the Statistical Analysis System (SAS) programs provided by the Centers for Disease Control (CDC) for the calculation of the 2000 CDC growth charts. The mean (SD) change in Z scores from baseline up to Month 1 for the total safety population for all age cohorts combined were presented. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean.

Change From Baseline in Weight Z-score up to Month 3
Baseline up to Month 3

The Z-Score indicates how many SD a participant has from the population normal values. The body weight z-scores were designed to take into account the amount of weight gain that was expected due to normal growth in children and adolescents. Body weight data were converted to Z-scores using the SAS programs provided by the CDC for the calculation of the 2000 CDC growth charts. The mean (SD) change in Z scores from baseline up to Month 3 for the total safety population for all age cohorts combined were presented. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean.

Change From Baseline in Weight Z-score up to Month 6
Baseline up to Month 6

The Z-Score indicates how many SD a participant has from the population normal values. The body weight z-scores were designed to take into account the amount of weight gain that was expected due to normal growth in children and adolescents. Body weight data were converted to Z-scores using the SAS programs provided by the CDC for the calculation of the 2000 CDC growth charts. The mean (SD) change in Z scores from baseline up to Month 6 for the total safety population for all age cohorts combined were presented. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean.

Change From Baseline in Weight Z-score up to Month 9
Baseline up to Month 9

The Z-Score indicates how many SD a participant has from the population normal values. The body weight z-scores were designed to take into account the amount of weight gain that was expected due to normal growth in children and adolescents. Body weight data were converted to Z-scores using the SAS programs provided by the CDC for the calculation of the 2000 CDC growth charts. The mean (SD) change in Z scores from baseline up yo Month 9 for the total safety population for all age cohorts combined were presented. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean.

Change From Baseline in Weight Z-score up to Month 12
Baseline up to Month 12

The Z-Score indicates how many SD a participant has from the population normal values. The body weight z-scores were designed to take into account the amount of weight gain that was expected due to normal growth in children and adolescents. Body weight data were converted to Z-scores using the SAS programs provided by the CDC for the calculation of the 2000 CDC growth charts. The mean (SD) change in Z scores from baseline to Month 12 for the total safety population for all age cohorts combined were presented. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean.

Change From Baseline in Height Z-score up to Month 1
Baseline up to Month 1

Z-Score was a statistical measure to evaluate how a single data point compares to a standard. It described whether a mean was above or below the standard and how unusual the measurement is with range from minus (-) 3 to plus (+) 3; 0 equal to (=) same mean, greater than (\>) 0 a greater mean, and less than (\<) 0 a lesser mean than the standard. Growth parameters were compared to a standard defined by CDC growth charts. The mean (SD) change in Z scores from baseline up to Month 1 for the total safety population for all age cohorts combined were presented. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean.

Change From Baseline in Height Z-score up to Month 3
Baseline up to Month 3

Z-Score was a statistical measure to evaluate how a single data point compares to a standard. It described whether a mean was above or below the standard and how unusual the measurement is with range from -3 to +3; 0 =same mean, \>0 a greater mean, and \<0 a lesser mean than the standard. Growth parameters were compared to a standard defined by CDC growth charts. The mean (SD) change in Z scores from baseline up to Month 3 for the total safety population for all age cohorts combined were presented. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean.

Change From Baseline in Height Z-score up to Month 6
Baseline up to Month 6

Z-Score was a statistical measure to evaluate how a single data point compares to a standard. It described whether a mean was above or below the standard and how unusual the measurement is with range from -3 to +3; 0 =same mean, \>0 a greater mean, and \<0 a lesser mean than the standard. Growth parameters were compared to a standard defined by CDC growth charts. The mean (SD) change in Z scores from baseline up to Month 6 for the total safety population for all age cohorts combined were presented.

Change From Baseline in Height Z-score up to Month 9
Baseline up to Month 9

Z-Score was a statistical measure to evaluate how a single data point compares to a standard. It described whether a mean was above or below the standard and how unusual the measurement is with range from -3 to +3; 0 =same mean, \>0 a greater mean, and \<0 a lesser mean than the standard. Growth parameters were compared to a standard defined by CDC growth charts. The mean (SD) change in Z scores from baseline up to Month 9 for the total safety population for all age cohorts combined were presented. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean.

Change From Baseline in Height Z-score up to Month 12
Baseline up to Month 12

Z-Score was a statistical measure to evaluate how a single data point compares to a standard. It described whether a mean was above or below the standard and how unusual the measurement is with range from -3 to +3; 0 =same mean, \>0 a greater mean, and \<0 a lesser mean than the standard. Growth parameters were compared to a standard defined by CDC growth charts. The mean (SD) change in Z scores from baseline up to Month 12 for the total safety population for all age cohorts combined were presented. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean.

Change From Baseline in Bone Mineral Density (BMD) Z-score up to Month 6
Baseline up to Month 6

The BMD was measured by dual energy X-ray absorptiometry (DEXA) for the posterior-anterior lumbar spine (L1\_L4) and total body less head area. The Z-Score is the number of standard deviations a participant's BMD differs from the average BMD of their age, sex and ethnicity. Positive scores indicate BMD above the mean; positive values are "best values" and negative values are "worst values". Positive changes from baseline indicated an improvement in condition.

Change From Baseline in BMD Z-score up to Month 12
Baseline up to Month 12

The BMD was measured by DEXA for the posterior-anterior lumbar spine (L1\_L4) and total body less head area. The Z-Score is the number of standard deviations a participant's BMD differs from the average BMD of their age, sex and ethnicity. Positive scores indicate BMD above the mean; positive values are "best values" and negative values are "worst values". Positive changes from baseline indicated an improvement in condition.

Change From Baseline in Bone Mineral Content (BMC)-Z Score up to Month 6
Baseline up to Month 6

The BMC is an estimate of the amount of mineral (such as calcium) in the bone, which was assessed by DEXA scan for the posterior-anterior lumbar spine (L1\_L4) and total body less head area. Positive changes from baseline indicated an improvement in condition.

Change From Baseline in BMC-Z Score up to Month 12
Baseline up to Month 12

The BMC is an estimate of the amount of mineral (such as calcium) in the bone, which was assessed by DEXA scan for the posterior-anterior lumbar spine (L1\_L4) and total body less head area. Positive changes from baseline indicated an improvement in condition.

Video-recorded EEG was primary efficacy measure. Baseline vEEG and the vEEG at Visit 4 (Days 19 to 20; end point DB treatment phase or early withdrawal) will be read by a blinded central reader
Video-recorded EEG was primary efficacy measure. Baseline vEEG and the vEEG at Visit 4 (Days 19 to 20; end point DB treatment phase or early withdrawal) will be read by a blinded central reader
Patient diary: number, duration, severity of headaches, symptoms, other medications used; QOL: Migraine Disability Assessment, Migraine-Specific Quality of Life, Quality of Life Enjoyment & Satisfaction Questionnaire-Short Form, Weight Satisfaction Scale
The primary outcome parameter is the time to first seizure during the double blind phase of the study. The statistical evaluation will analyze if there is a significant difference in the proportion of patients being seizure free between both medications.
Patient diaries for number & severity of migraine or migrainous headaches, symptoms, & other medications used; Migraine Disability Assessment, Migraine-Specific Quality of Life, Physicians & Subjects Global Impression of Change for quality of life
Change from baseline in the average monthly migraine episode rate.
Change in migraine days, migraine periods, and migraine attacks compared between the topiramate group and placebo group at the last month of the baseline phase and the last month of the placebo-controlled phase
Change in migraine days compared between the topiramate group and placebo group at the last 4 weeks of the open-label phase and the final 4 weeks of the placebo-controlled phase
Last 4 weeks of Open Label (OL) Phase and Double Blind (DB) Phase
Weight, Body Mass Index,number of binge eating and anthropometric measurements
Change in the average number of days per month with migraine or migrainous headache by daily headache record.
Change from baseline in total Young Mania Rating Scale (YMRS) score
Baseline to Day 28 (or last available observation prior to Day 28).
Clinical Rating Scale for Tremor (TRS) score at Visit 8 (or patient's final visit) of the double-blind phase
Change from baseline to Day 21 in the total Young Mania Rating Scale (YMRS) score.
Change in the monthly (28 days) migraine period rate from the Prospective Baseline Period to the Core Double-Blind Phase.
Change in monthly (28 day) migraine period rate from the prospective baseline period to the double-blind phase.
The percent change in body weight and in sitting diastolic blood pressure from baseline/randomization (Week 0) to Week 60.
Changes in length of time between the onset and cessation of painful migraine symptoms (migraine period) from the baseline. Safety evaluations conducted throughout the study.
The percent change in body weight and change in Hemoglobin A1c from enrollment to Week 60.
Change from baseline to Day 21 for the Young Mania Rating Scale (YMRS) total score.
Reduction in the frequency of monthly migraine episodes during the entire double-blind treatment phase compared with the pretreatment phase.
The percent change in body weight and change in HbA1c from baseline (Week 0) to one year after maintenance therapy (Week 60).
The percent change in body weight from the enrollment visit to week 60.
The percent change in body weight from the baseline (randomization) to Week 60 (after one year of maintenance therapy).
Time to first seizure (partial onset or generalized tonic-clonic seizure) during the core double-blind phase (excluding taper).
Metabolic test battery including Body Mass Index (BMI), lean body mass, blood lipids and fasting glucose along with physical examinations (including body weights) will be recorded at the beginning of the trial, after 3 months and after one year
Time to exit during the double blind phase (2 partial onset seizures with or without a secondarily generalized component, a secondarily generalized tonic clonic seizure when none existed prior to this phase, or 1 episode of status epilepticus).
Percent reduction from baseline in primary generalized tonic-clonic seizure rates and percent responders (>=50% reduction in PGTC seizure rate from baseline), during the double-blind phase. Subjects' global evaluation of improvement in seizure severity.
Percent reduction in the average monthly seizure rate from baseline to end of treatment
Percent reduction from baseline in primary generalized tonic-clonic (PGTC) seizure rates in the double-blind phase.
Percent reduction from baseline in seizure rates (all types of seizures) in the double-blind phase. Percent reduction in drop attacks (tonic-clonic) and parent/guardian global evaluation at end of study
Time to patient exit (withdrawal) from the double-blind phase of the study, based on 4 pre-established exit criteria corresponding to therapeutic failure.
Cocaine-induced mood changes
measured throughout cocaine and topiramate testing sessions
Effects of topiramate on cognitive function
measured throughout cocaine and topiramate testing sessions
Drug safety
measured throughout cocaine and topiramate testing sessions
Cardiovascular response to cocaine (measured throughout cocaine and topiramate testing sessions)
measured throughout cocaine and topiramate testing sessions
Number of Participants With Adverse Events
Baseline up to 28 days after last dose of study drug

An adverse event is any untoward medical event that occurs in a participant administered an investigational product, and it does not necessarily indicate only events with clear causal relationship with the relevant investigational product.

Mean Change From Baseline in Monthly Migraine Attacks (According to 24-Hour Rule) Through Month 6
Baseline (28 days before randomization) through Month 6

As per 24-hour rule, if symptom of pain due to migraine continues for more than 24 hours, it should be considered as 2 or more migraine attacks considering the maximum duration up to 24 hours. If the interval between latest migraine attack (ending time) and previous migraine attack (onset time) is less than 24 hours, 2 migraine attacks should be considered as 1 migraine attack. If the onset of migraine was prevented by a rescue drug, it should be considered as 1 migraine attack even if aura had started. Mean change was calculated by subtracting baseline value from the mean of 6 months value.

Reduction of alcohol-induced craving,reward,and euphoria
During testing days
Proportion of subjects who become seizure-free.
The change in percentage of heavy drinking days (5 or more standard drinking units per day for men and 4 or more standard drinking units per day for women) from baseline at 12 weeks or final visit.
Percent change in body weight from baseline to Week 16.
The rate of change in the number of binge days per week from baseline at 16 weeks or final visit.
The proportion of migraine attacks that are pain-free two hours after triptan treatment, compared to baseline.
Change in peroneal nerve conduction velocity (NCV) from baseline to the end of the double-blind phase.
The percent change in body weight from baseline (at the time of randomization) to Week 24.
Mean change in insulin sensitivity from baseline to Month 9.
The mean change from baseline to Month 6 in abdominal visceral fat as assessed by computed tomography; the safety of Topiramate for up to 12 months of continued treatment in male subjects with abdominal obesity.
To assess the safety of this novel combination of topiramate and naltrexone. To evaluate the efficacy of topiramate and naltrexone in combination for the treatment of alcoholism.
Throughout the study
Individual (each patient) and mean (each treatment) topiramate plasma concentration-time profiles.
To evaluate the potential pharmacokinetic interaction between topiramate and lithium in patients with bipolar disorders
In each sampling period, blood (lithium and topiramate concentrations) is collected pre-dose and at 0.5, 1, 1.5, 2, 3, 4, 6, 8, and 12 hours post-morning dose, and urine (lithium concentration) was collected at 0-2, 2-4, 4-8, and 8-12 hours postdose
To evaluate the potential pharmacokinetic interaction between topiramate and risperidone in patients with bipolar disorder or schizoaffective disorders.
At each sampling visit during Periods I, II and III, blood and urine collected pre-morning dose and over a period of 12 hours post-dose.

Secondary Endpoints

Number of Participants With Treatment-emergent Adverse Events (TEAE)
Up to Day 390
Percentage of Participants With Kidney Stones
Up to Day 390
Percent treatment responders >=50% reduction seizure rate. ·Percentage change in seizure rate recorded on subject take-home records. Percentage change in seizure rates for all seizure types recorded on VEEG.
Baseline to endpoint of double blind phase
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Study Design & Arms

AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeHEALTH_SERVICES_RESEARCH

Treatment Arms

ArmTypeDescription
TopiramateEXPERIMENTALTopiramate weight based dosing for participants 2 to less than (\<) 10 years of age not to exceed 350 mg/day (milligram per day), as tolerated; not to exceed 400 mg/day in participants 10-15 years of age, as tolerated.
LevetiracetamACTIVE_COMPARATORLevetiracetam weight based dosing for all participants 2-15 years of age, not to exceed 60 milligram per kilogram per day (mg/kg/day), as tolerated. The maximum recommended daily dosage is 3,000 milligram (mg).
003EXPERIMENTALtopiramate 25 mg/kg/day
002EXPERIMENTALtopiramate 15 mg/kg/day
001EXPERIMENTALtopiramate 5 mg/kg/day
004EXPERIMENTALplacebo placebo
Open Label PhaseEXPERIMENTALTopiramate treatment started with one tablet per day, taken in the evening, for the first 7 days of the OL phase. Each tablet contained 25 mg topiramate. After one week, the dose was raised to two tablets per day: one tablet was taken in the morning, the other in the evening. Until Week 26
Double Blind and Roll Out PhaseEXPERIMENTALthe trial medication consisted of topiramate 25 mg tablets or matching placebo tablets which were identical in appearance, taste and smell. DB randomisation phase (after the 26-weeks OL phase) were randomly allocated (1:1) to one of the two treatment groups (topiramate or placebo). The randomisation took place at Visit 6 (Week 26).
PlaceboPLACEBO_COMPARATOR -
Topiramate (JNS019) 50 mgEXPERIMENTALIn titration period, topiramate 25 milligram (mg) tablet will be given once daily in evening orally for 7 days; then topiramate 25 mg tablet twice daily orally from Day 8 to Day 14; then topiramate 25 mg tablet twice daily along with matching placebo tablet once daily in the evening orally from Day 15 to Day 21; then topiramate 25 mg tablet along with matching placebo tablet twice daily orally from Day 22 to Day 28 and will be continued further for 18 weeks in the fixed dose period.
Topiramate 100 mgEXPERIMENTALIn titration period, topiramate 25 mg tablet will be given once daily in the evening orally for 7 days; then topiramate 25 mg tablet twice daily orally from Day 8 to Day 14; then topiramate 25 mg tablet twice daily (1 tablet in the morning and 2 tablets in the evening) orally from Day 15 to Day 21; then 2 topiramate 25 mg tablets twice daily orally from Day 22 to Day 28 and will be continued further for 18 weeks in the fixed dose period.
Topiramate + NaltrexoneEXPERIMENTALCombination of Topiramate and Naltrexone

Interventions

NameTypeDescription
TopiramateDRUGTopiramate weight based dosing for participants 2 to \<10 years of age not to exceed 350 mg/day, as tolerated; not to exceed 400 mg/day in participants 10-15 years of age, as tolerated.
LevetiracetamDRUGLevetiracetam weight based dosing for all participants 2-15 years of age, not to exceed 60 mg/kg/day, as tolerated. The maximum recommended daily dosage is 3,000 mg.
placeboDRUGplacebo
topiramate, phenytoinDRUG -
Topiramate - PlaceboDRUGthe trial medication consisted of topiramate 25 mg tablets or matching placebo tablets which were identical in appearance, taste and smell. DB randomisation phase (after the 26-weeks OL phase) were randomly allocated (1:1) to one of the two treatment groups (topiramate or placebo). The randomisation took place at Visit 6 (Week 26)
topiramate, propranololDRUG -
Topiramate and NaltrexoneDRUGTopiramate 200 mg and Naltrexone 50 mg
risperidoneDRUGTwice daily, individualized dosing to stabilization at 1-6 mg/day.
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Eligibility Criteria

Age Range2 Years to 15 Years
SexALL
Healthy VolunteersNo
Study Sites41

Inclusion Criteria: * Participant with a clinical diagnosis of new-onset or recent-onset epilepsy characterized by partial-onset seizures (POS) (with or without secondary generalization) or primary generalized tonic-clonic seizures (PGTCS) in accordance with criteria of the International League Aga...

Countries:United StatesArgentinaAustraliaAustriaBelgiumCanadaFranceGermanyHungaryPhilippinesPolandRussiaSouth AfricaTaiwanChileFinlandIndiaIsraelMexicoNetherlandsNew ZealandNorwaySouth KoreaSpainThailandUkraineBulgariaCzechiaDenmarkGreeceIrelandPortugalSaudi ArabiaSloveniaSwitzerlandTurkey (Türkiye)United KingdomJapan
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