EpilepsyPhase 2NCT01501383
A Study to Evaluate the Efficacy and Safety of VX-765 in Subjects With Treatment-Resistant Partial Epilepsy
A Phase 2 study testing VX-765 Part A and VX-765 Part B in people with Epilepsy, run by Vertex Pharmaceuticals Incorporated at 24 sites in 2 countries.
Trial summary
The purpose of this study is to evaluate the efficacy, safety and tolerability of VX-765 in subjects with treatment-resistant partial epilepsy. Read more about this study
- Who is it for?
- Ages 18 to 64 years
- Conditions the study is for
- Epilepsy
- What is being tested?
- VX-765 Part A and VX-765 Part B
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 55 participants
- Where
- 24 sitesin 2 countries
- Study dates
- Dec 2011to Aug 2013
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 64 years
- All sexes
- No healthy volunteers
- Adult
Part A and Part B Inclusion Criteria:
- Males or females aged 18 to 64 years with a body mass index 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
- Male or female subjects must agree to use acceptable contraceptive methods, as described in the protocol
- Must have a diagnosis and history of treatment-resistant partial-onset epilepsy for which they are taking 1 to 4 concomitant AEDs at the time of Screening Period
- Have had at least 1 electroencephalogram consistent with partial epilepsy
- Must have had at least 6 partial-onset seizures and a seizure-free period of no more than 3 weeks (21 days) during the Baseline Period.
- Subjects with stable medical conditions (e.g., cannot have a condition that will interfere with the conduct of the study or cause a known increase in risk of the intervention) as determined by the principal investigator
- Must be able and willing to provide written informed consent to participate
- Must be able to understand and comply with protocol requirements and instructions
Part A and Part B Exclusion Criteria:
- Subjects who are male and their female partner (if of childbearing potential) does not agree to use medically approved methods of contraception as described in the protocol for the duration od 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 who are pregnant or lactating, or who are of reproductive potential who do not agree to use medically approved birth control methods
- History of nonepileptic, transient alterations in consciousness
- History of status epilepticus in the past 12 months before the screening visit
- Subjects whose seizure frequency cannot be quantified (i.e. seizures with no discrete beginning or end, or period between seizures)
- 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.
- Have a clinically significant psychiatric illness as judged by the investigator
- Subjects who have had an active suicidal plan/intent or active suicidal thoughts, or suicide attempt as defined in the protocol
- 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 participate in Part B
- Active hepatitis B, hepatitis C, or human immunodeficiency virus
- 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 as defined in the protocol
- Using prohibited medications or treated with any systemic immunosuppressant
- Have experienced a symptomatic viral, fungal, or bacterial infection requiring systemic treatment within 7 days prior to the first dose of study drug
- A current or prior history of illness precluding them from immunomodulatory therapy (e.g., history of recurrent infections)
- Have donated any blood or have had a significant loss of blood (500 mL) as defined in the protocol
- Have 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
- 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, sponsor or designee to be inappropriate for the study
Trial locations and contacts
24 sites in 2 countries.
24 sites in 2 countries
United States22 sites
North Carolina
Asheville, North Carolina
Maryland
Baltimore, Maryland
Idaho
Boise, Idaho
Florida
Bradenton, Florida
North Carolina
Charlotte, North Carolina
Virginia
Charlottesville, Virginia
Ohio
Columbus, Ohio
Texas
Dallas, Texas
Michigan
Farmington Hills, Michigan
Arkansas
Little Rock, Arkansas
California
Loma Linda, California
New York
New York, New York
Alabama
Northport, Alabama
Oklahoma
Oklahoma City, Oklahoma
Utah
Orem, Utah
Pennsylvania
Philadelphia, Pennsylvania
Arizona
Phoenix, Arizona
Arizon
Phoenix, Arizona
Washington
Renton, Washington
Minnesota
Saint Paul, Minnesota
New York
The Bronx, New York
Florida
Wellington, Florida
About this study
Official title: A Phase 2b, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Dose-Ranging Study to Evaluate the Efficacy and Safety of VX-765 in Subjects With Treatment-Resistant Partial Epilepsy With a 24-Week Open-Label Extension
The purpose of this study is to evaluate the efficacy, safety and tolerability of VX-765 in subjects with treatment-resistant partial epilepsy.
- Conditions
- Epilepsy
- Being tested
- VX-765 Part A, Placebo, VX-765 Part B
- Medical subject headings
- Epilepsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VX-765 Part ADrug
Tablets of VX-765 given at different doses based on treatment group in Part A
PlaceboDrug
Matching placebo
VX-765 Part BDrug
Tablets of VX-765 given at different doses based on patients who meet the study eligibility criteria for Part B
| Study group | Role | What they receive |
|---|---|---|
| VX-765 Dose 1 Part A | Active comparator | Not described Receives: Drug: VX-765 Part A |
| VX-765 Dose 2 Part A | Active comparator | Not described Receives: Drug: VX-765 Part A |
| VX-765 Dose 3 Part A | Active comparator | Not described Receives: Drug: VX-765 Part A |
| VX-765 Dose 4 Part A | Active comparator | Not described Receives: Drug: VX-765 Part A |
| Placebo Dose Part A | Placebo comparator | Placebo Receives: Drug: Placebo |
| VX-765 Dose Part B | Active comparator | Not described Receives: Drug: VX-765 Part B |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent reduction in weekly seizure frequency during the Part A Late Treatment Period compared to the Part A Baseline Period
Measured: Up to 25 Weeks
Percent of subjects with 50% or greater reduction in weekly seizure frequency (responder-rate) during the Part A Late Treatment Period compared to the Part A Baseline Period
Measured: Up to 25 Weeks
Safety and tolerability as assessed by vital signs, standard 12-lead electrocardiograms (ECGs), laboratory assessments (serum chemistry, hematology, and urinalysis), and adverse events
Measured: Up to 56 Weeks
Safety and tolerability as determined by vital signs, standard 12-lead electrocardiograms (ECGs), laboratory assessments (serum chemistry, hematology, and urinalysis) and adverse events
Measured: Up to 56 Weeks
Secondary outcomes (13)
Percent of subjects who are seizure-free during the Part A Late Treatment Period
Measured: Up to 25 Weeks
Percent reduction in seizure frequency during the entire Part A Treatment Period compared to the Part A Baseline Period
Measured: Up to 25 Weeks
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
Measured: Up to 25 Weeks
Percent of subjects who are seizure-free during the entire Part A Treatment Period
Measured: 13 Weeks
Maximum number of consecutive days that subjects do not have seizures at any time during the Part A Late Treatment Period
Measured: Up to 13 Weeks
Maximum number of consecutive days that subjects do not have seizures at any time during the entire Treatment Period
Measured: 13 Weeks
Pharmacokinetics (e.g AUC, Cmax) of VX-765, VRT-043198, and concomitant antiepileptic drug (AED) levels in blood
Measured: Up to 21 Weeks
Percent reduction in weekly seizure frequency compared to the Part A Baseline Period
Measured: Up to 56 Weeks
Percent of subjects with 50% or greater reduction in weekly seizure frequency (50% responder-rate) during the entire Part B Treatment Period compared to the Part A Baseline Period
Measured: Up to 56 Weeks
Percent of subjects with 75% or greater reduction in weekly seizure frequency (75% responder-rate) during the entire Part B Treatment Period compared to the Part A Baseline Period
Measured: Up to 56 Weeks
Percent of subjects who are seizure-free during the entire Part B Treatment Period
Measured: Up to 28 Weeks
Maximum number of consecutive days that subjects do not have seizures at any time during the entire Part B Treatment Period
Measured: Up to 28 Weeks
Percent increase in weekly seizure-free days compared to the Part A Baseline Period
Measured: Up to 56 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Dec 2011 (estimated)
- Study startDec 2011
- Primary completionAug 2013
Why it stopped: Terminated by Sponsor
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for EpilepsyStudies of the same condition, open and under way.
- More trials run by Vertex Pharmaceuticals IncorporatedThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 55 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01501383
- Sponsor's protocol ID
- VX11-765-402
- Status last verified
- Sep 2020
- First submitted
- 15 Dec 2011
- Last updated
- 30 Sep 2020
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 30 Sep 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2b, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Dose-Ranging Study to Evaluate the Efficacy and Safety of VX-765 in Subjects With Treatment-Resistant Partial Epilepsy With a 24-Week Open-Label Extension
- ClinicalTrials.gov ID
- NCT01501383
- Acronym / sponsor ID
- VX11-765-402
- Conditions
- Epilepsy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Terminated by Sponsor
- Enrolment
- 55 participants (actual)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Study start
- Dec 2011
- Primary completion
- Aug 2013 (actual)
- First posted
- 29 Dec 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to evaluate the efficacy, safety and tolerability of VX-765 in subjects with treatment-resistant partial epilepsy.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| VX-765 Dose 1 Part A | Active comparator | Drug: VX-765 Part A |
| VX-765 Dose 2 Part A | Active comparator | Drug: VX-765 Part A |
| VX-765 Dose 3 Part A | Active comparator | Drug: VX-765 Part A |
| VX-765 Dose 4 Part A | Active comparator | Drug: VX-765 Part A |
| Placebo Dose Part A | Placebo comparator | Drug: PlaceboPlacebo |
| VX-765 Dose Part B | Active comparator | Drug: VX-765 Part B |
Who may qualify
- Ages
- 18 to 64 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VX-765 Part ATablets of VX-765 given at different doses based on treatment group in Part A | Drug | VX-765 Dose 1 Part A, VX-765 Dose 2 Part A, VX-765 Dose 3 Part A, VX-765 Dose 4 Part A |
| PlaceboMatching placebo | Drug | Placebo Dose Part A |
| VX-765 Part BTablets of VX-765 given at different doses based on patients who meet the study eligibility criteria for Part B | Drug | VX-765 Dose Part B |
Primary outcome
Percent reduction in weekly seizure frequency during the Part A Late Treatment Period compared to the Part A Baseline Period
Time frame: Up to 25 Weeks
Percent of subjects with 50% or greater reduction in weekly seizure frequency (responder-rate) during the Part A Late Treatment Period compared to the Part A Baseline Period
Time frame: Up to 25 Weeks
Safety and tolerability as assessed by vital signs, standard 12-lead electrocardiograms (ECGs), laboratory assessments (serum chemistry, hematology, and urinalysis), and adverse events
Time frame: Up to 56 Weeks
Safety and tolerability as determined by vital signs, standard 12-lead electrocardiograms (ECGs), laboratory assessments (serum chemistry, hematology, and urinalysis) and adverse events
Time frame: Up to 56 Weeks
13 secondary outcomes
Percent of subjects who are seizure-free during the Part A Late Treatment Period
Time frame: Up to 25 Weeks
Percent reduction in seizure frequency during the entire Part A Treatment Period compared to the Part A Baseline Period
Time frame: Up to 25 Weeks
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
Time frame: Up to 25 Weeks
Percent of subjects who are seizure-free during the entire Part A Treatment Period
Time frame: 13 Weeks
Maximum number of consecutive days that subjects do not have seizures at any time during the Part A Late Treatment Period
Time frame: Up to 13 Weeks
Maximum number of consecutive days that subjects do not have seizures at any time during the entire Treatment Period
Time frame: 13 Weeks
Pharmacokinetics (e.g AUC, Cmax) of VX-765, VRT-043198, and concomitant antiepileptic drug (AED) levels in blood
Time frame: Up to 21 Weeks
Percent reduction in weekly seizure frequency compared to the Part A Baseline Period
Time frame: Up to 56 Weeks
Percent of subjects with 50% or greater reduction in weekly seizure frequency (50% responder-rate) during the entire Part B Treatment Period compared to the Part A Baseline Period
Time frame: Up to 56 Weeks
Percent of subjects with 75% or greater reduction in weekly seizure frequency (75% responder-rate) during the entire Part B Treatment Period compared to the Part A Baseline Period
Time frame: Up to 56 Weeks
Percent of subjects who are seizure-free during the entire Part B Treatment Period
Time frame: Up to 28 Weeks
Maximum number of consecutive days that subjects do not have seizures at any time during the entire Part B Treatment Period
Time frame: Up to 28 Weeks
Percent increase in weekly seizure-free days compared to the Part A Baseline Period
Time frame: Up to 56 Weeks
Results and safety
The study was stopped early (reason given: Terminated by Sponsor). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Sep 2020.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
24 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing VX-765 Part A and VX-765 Part B in people with Epilepsy, run by Vertex Pharmaceuticals Incorporated at 24 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 30 Sep 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 24 sites in United States, Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Terminated by Sponsor). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percent reduction in weekly seizure frequency during the Part A Late Treatment Period compared to the Part A Baseline Period (time frame: Up to 25 Weeks). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Sep 2020.