A Study to Evaluate the Efficacy and Safety of VX-765 in Subjects With Treatment-Resistant Partial Epilepsy

NCT01501383 | Terminated Phase 2 DMC

• 1 other identifier: VX11-765-402
• Study Type: interventional
• Target: 55
• Locations: 2 countries
• Sites: 24

Brief Summary

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

Outcome Measures

Primary Outcomes (4)

• Percent reduction in weekly seizure frequency during the Part A Late Treatment Period compared to the Part A Baseline Period

  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

  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

  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

  Up to 56 Weeks

Secondary Outcomes (13)

• Percent of subjects who are seizure-free during the Part A Late Treatment Period

  Up to 25 Weeks

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

  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

  Up to 25 Weeks

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

  13 Weeks

• Maximum number of consecutive days that subjects do not have seizures at any time during the Part A Late Treatment Period

  Up to 13 Weeks

Study Arms (6)

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

Placebo

Drug: Placebo

VX-765 Dose Part B

ACTIVE COMPARATOR

Drug: VX-765 Part B

Interventions

VX-765 Part A DRUG

Tablets of VX-765 given at different doses based on treatment group in Part A

VX-765 Dose 1 Part A; VX-765 Dose 2 Part A; VX-765 Dose 3 Part A; VX-765 Dose 4 Part A

Placebo DRUG

Matching placebo

Placebo Dose Part A

VX-765 Part B DRUG

Tablets of VX-765 given at different doses based on patients who meet the study eligibility criteria for Part B

VX-765 Dose Part B

Eligibility Criteria

• Age: 18 Years - 64 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Vertex Pharmaceuticals Incorporated: lead

Study Sites (24)

Alabama

Northport, Alabama, United States

Location

Arizona

Phoenix, Arizona, United States

Location

Arizon

Phoenix, Arizona, United States

Location

Arkansas

Little Rock, Arkansas, United States

Location

California

Loma Linda, California, United States

Location

Florida

Bradenton, Florida, United States

Location

Florida

Wellington, Florida, United States

Location

Idaho

Boise, Idaho, United States

Location

Maryland

Baltimore, Maryland, United States

Location

Michigan

Farmington Hills, Michigan, United States

Location

Minnesota

Saint Paul, Minnesota, United States

Location

New York

New York, New York, United States

Location

New York

The Bronx, New York, United States

Location

North Carolina

Asheville, North Carolina, United States

Location

North Carolina

Charlotte, North Carolina, United States

Location

Ohio

Columbus, Ohio, United States

Location

Oklahoma

Oklahoma City, Oklahoma, United States

Location

Pennsylvania

Philadelphia, Pennsylvania, United States

Location

Texas

Dallas, Texas, United States

Location

Utah

Orem, Utah, United States

Location

Virginia

Charlottesville, Virginia, United States

Location

Washington

Renton, Washington, United States

Location

Germany

Bonn, Germany

Location

Germany

Kork, Germany

Location

MeSH Terms

Conditions

Epilepsy

Condition Hierarchy (Ancestors)

Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 15, 2011
• First Posted: December 29, 2011
• Study Start: December 1, 2011
• Primary Completion: August 1, 2013
• Last Updated: September 30, 2020

Record last verified: 2020-09

Locations

DE (2); US (22)