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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.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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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

Other trials in United States

Germany2 sites
  • Germany

    Bonn

  • Germany

    Kork

Other trials in Germany

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 groups in NCT01501383
Study groupRoleWhat they receive
VX-765 Dose 1 Part AActive comparatorNot described
Receives: Drug: VX-765 Part A
VX-765 Dose 2 Part AActive comparatorNot described
Receives: Drug: VX-765 Part A
VX-765 Dose 3 Part AActive comparatorNot described
Receives: Drug: VX-765 Part A
VX-765 Dose 4 Part AActive comparatorNot described
Receives: Drug: VX-765 Part A
Placebo Dose Part APlacebo comparatorPlacebo
Receives: Drug: Placebo
VX-765 Dose Part BActive comparatorNot described
Receives: Drug: VX-765 Part B

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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

  2. 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

  3. 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

  4. 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)
  1. Percent of subjects who are seizure-free during the Part A Late Treatment Period

    Measured: Up to 25 Weeks

  2. Percent reduction in seizure frequency during the entire Part A Treatment Period compared to the Part A Baseline Period

    Measured: Up to 25 Weeks

  3. 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

  4. Percent of subjects who are seizure-free during the entire Part A Treatment Period

    Measured: 13 Weeks

  5. 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

  6. Maximum number of consecutive days that subjects do not have seizures at any time during the entire Treatment Period

    Measured: 13 Weeks

  7. Pharmacokinetics (e.g AUC, Cmax) of VX-765, VRT-043198, and concomitant antiepileptic drug (AED) levels in blood

    Measured: Up to 21 Weeks

  8. Percent reduction in weekly seizure frequency compared to the Part A Baseline Period

    Measured: Up to 56 Weeks

  9. 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

  10. 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

  11. Percent of subjects who are seizure-free during the entire Part B Treatment Period

    Measured: Up to 28 Weeks

  12. 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

  13. 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.

  1. First posted29 Dec 2011 (estimated)
  2. Study startDec 2011
  3. Primary completionAug 2013

Why it stopped: Terminated by Sponsor

Resources

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)
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

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.

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