Partial epilepsy MedDRA version: 14.1 Level: LLT Classification code 10065336 Term: Partial epilepsy System Organ Class: 100000004852
Conditions
Interventions
Product Code: VX-765
Pharmaceutical Form: Tablet
INN or Proposed INN: Not Applicable
CAS Number: 273404-37-8
Current Sponsor code: VX-765
Concentration unit: mg milligram(s)
Concentration type: equal
Sponsors
Vertex Pharmaceuticals Incorporated
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Males or females aged 18 to 64 (inclusive) years with a BMI between 18 and 35 (kg/m2) • Subjects who have completed the assigned study treatment in Part A may enter Part B if eligible per protocol • Subjects must agree to use acceptable contraceptive methods as described in Section 12.7.5. of the study protocol • Must have a diagnosis and hx of treatment-resistant partial-onset epilepsy (as defined in protocol) and are taking 1 to 4 stable doses of concomitant AEDs at the time of Screening Period • Subjects must have had at least 1 EEG consistent with partial epilepsy • Must have had at least 6 partial-onset seizures and a seizure free period of no more than 3 weeks during Baseline • Subjects with stable medical conditions as determiend by Principal investigator • Must understand and comply with the protocol requirements and be willing to provide written informed consent to participate Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 63 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: • Subjects who are pregnant or lactating or do not agree to use medically approved methods of contraception as outlined in the protocol - for the duration of the study and for 90 days after last dose of study drug • Subjects who are male and have a female partner who is pregnant, nursing, or is planning to become pregnant during the study period, or within 90 days of the last dose of study drug • Subjects with a history of nonepileptic, transient alterations in consciousness (e.g., metabolic, structural, and post traumatic or pseudo seizures) • Those who have a history of status epilepticus in the past 12 months before the Screening Visit • Subjects whose seizure frequency cannot be quantified • Subjects who have a significant medical illness including kidney, liver, pulmonary, or gastrointestinal disease; or unstable or poorly controlled conditions such as hypertension, diabetes, or angina pectoris, as judged by the investigator • Subjects with clinically significant psychiatric illness or had an active suicidal plan/intent, thoughts, or attempt as defined in the study protocol • Subjects with clinically significant laboratory abnormalities during the Screening Visit/Baseline Period, as judged by the investigator • Subjects who have had serious adverse events (SAEs) thought to be related to study drug that led to discontinuation during Part A may not participcate in Part B • Subjects with active hepatitis B, hepatitis C, or human immunodeficiency virus (HIV) • Subjects with positive drug screen at screening or during the Baseline Period (excluding any allowed prescribed medications) and/or a history of alcoholism or drug addiction within past 2 years • Subjects on felbamate with fewer than 18 month of continuous felbamate exposure at the time of the Screening Visit or with significant adverse reactions to felbamate • Subjects treated with vigabatrin fewer than 2 years prior to the Screening Visit or who have a prior history of treatment with vigabatrin without a documented stable examination by an ophthalmologist or neuroophthalmologist of the visual field within the 2 years prior to the Screening Visit • Subjects using prohibited medications (Section 10.3 and Table 10 1) or treated with any systemic immunosuppressant as defined in the protocol • Subjects who experienced a symptomatic viral, fungal, or bacterial infection requiring systemic treatment within 7 days prior to the first dose of study drug • Subjects with a current or prior history of illness precluding them from immunomodulatory therapy • Subjects who have donated any blood or have had a significant loss of blood (500 mL) as defined in the protocol • Subjects who participated in any other clinical studies involving an investigational product or device and have received the last dose of the study drug associated with that clinical study within 30 days or 5 half lives (whichever is longer) of the Screening Visit • Subjects who have participated in earlier VX-765 clinical studies and received at least one dose of study drug • Subjects who have no completed the full 13-week Treatment Period in part A may not participate in Part B • Any subject judged by the investigator or sponsor (or designee) to be inappropriate for the study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the efficacy, safety, and tolerability of VX-765 to treat seizures in subjects with treatment resistant partial epilepsy ;Secondary Objective: To evaluate the pharmacokinetics (PK) of VX-765, VRT 043198 (active metabolite of VX-765), and concomitant antiepileptic drug (AED) levels in subjects with treatment resistant partial epilepsy;Primary end point(s): • Percent reduction in weekly seizure frequency during the Part A Late Treatment Period compared to the Part A Baseline Period • Percent of subjects with 50% or greater reduction in weekly seizure frequency (responder rate) during the Late Treatment Period compared to the Baseline Period • Safety and tolerability as assessed by vital signs, standard 12 lead electrocardiograms (ECGs), laboratory assessments (serum chemistry, hematology, and urinalysis), and adverse events ;Timepoint(s) of evaluation of this end point: Multiple timepoints up to Study Week 37 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Percent of subjects who are seizure free during the Part A Late Treatment Period • Percent reduction in seizure frequency during the entire Part A Treatment Period compared to the Part A Baseline Period • Percent of subjects with 50% or greater reduction in seizure frequency (responder rate) during the entire Part A Treatment Period compared to the Part A Baseline Period • Percent of subjects who are seizure-free during the entire Part A Treatment Period • Maximum number of consecutive days that subjects do not have seizures at any time during the Part A Late Treatment Period • Maximum number of consecutive days that subjects do not have seizures at any time during the entire Part A Treatment Period • PK of VX 765, VRT 043198, and concomitant AED levels in blood ;Timepoint(s) of evaluation of this end point: Multiple timepoints up to Study Week 37 | — |
Countries
Austria, Czech Republic, Finland, Germany, Hungary, United Kingdom, United States
Contacts
Public ContactMedical Information Center
Vertex Pharmaceuticals Incorporated
Outcome results
None listed