A Study to Evaluate the Efficacy and Safety of VX-765 in Subjects With Treatment-Resistant Partial Epilepsy
- Conditions
- Partial epilepsyMedDRA version: 14.1Level: LLTClassification code 10065336Term: Partial epilepsySystem Organ Class: 10029205 - Nervous system disordersTherapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2011-004156-19-GB
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 500
• 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 500
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
• 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
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To evaluate the efficacy, safety, and tolerability of VX-765 to treat seizures in subjects with treatment resistant partial epilepsy<br>;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<br>Treatment Period compared to the Part A Baseline Period<br>• Percent of subjects with 50% or greater reduction in weekly seizure<br>frequency (responder rate) during the Part A Late Treatment Period compared<br>to the Part A Baseline Period<br>• Safety and tolerability as assessed by vital signs, standard 12 lead<br>electrocardiograms (ECGs), laboratory assessments (serum chemistry,<br>hematology, and urinalysis), and adverse events;Timepoint(s) of evaluation of this end point: Multiple timepoints up to Study Week 37
- Secondary Outcome Measures
Name Time Method Secondary end point(s): • Percent of subjects who are seizure free during the Part A Late<br>Treatment Period<br>• Percent reduction in seizure frequency during the entire Part A<br>Treatment Period compared to the Part A Baseline Period<br>• Percent of subjects with 50% or greater reduction in seizure frequency<br>(responder rate) during the entire Part A Treatment Period compared to<br>the Part A Baseline Period<br>• Percent of subjects who are seizure-free during the entire Part A<br>Treatment Period<br>• Maximum number of consecutive days that subjects do not have<br>seizures at any time during the Part A Late Treatment Period<br>• Maximum number of consecutive days that subjects do not have<br>seizures at any time during the entire Part A Treatment Period<br>• 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