Last updated on February 2019

VX15/2503 Treatment for Huntington's Disease

Brief description of study

The purpose of this study is to evaluate the safety, tolerability, PK, and efficacy of VX15/2503 in subjects with late prodromal and early manifest Huntington's disease.

Detailed Study Description

VX15/2503-N-131 is a Phase 2, multi-center, randomized, double-blind, placebo controlled study of VX15/2503 in subjects with late prodromal and early manifest Huntington's disease. The primary objective is to evaluate the safety and tolerability of monthly IV administration of a single dosage of VX15/2503 (or placebo). Efficacy endpoints include determining the effect of VX15/2503 on brain volumes (MRI), FDG-PET imaging, 11C-PBR28 (TSPO) PET imaging (subset of Cohort B only) and clinical features of HD including cognition, motor function, behavior, functional abilities, global function and global measurement of change. Additional endpoints include PK / PD, immunogenicity, and exploratory biomarkers. Subjects in Cohort B that have received 12 months of VX15/2503 who volunteer will undergo a lumbar puncture at V13 to collect cerebral spinal fluid (CSF) to evaluate VX15/2503 mAb concentrations, total sSEMA4D levels, and other biomarkers in their CSF. Enrollment will involve approximately 276 individuals who are 21 years of age or older with late prodromal (CAG-age product score (CAP score) of greater than 200 and Diagnostic Confidence Level (DCL) of 2 or 3) or early manifest HD (Total Functional Capacity (TFC) greater than or equal to 11). The study will be divided into Cohort A and Cohort B. Cohort A is now complete and an unblinded analysis has been performed. Cohort A subjects were treated for 6 months with either drug or placebo (1:1) and then all subjects were treated with drug for 6 months, followed by 3 months of follow up. Treatment duration for each subject in Cohort A was 12 months. Participation in Cohort A included a Screening visit, a Baseline visit within 30 days of screening; 12 monthly treatment visits beginning at baseline and continuing through Month 12; follow-up safety phone call at one month and a follow-up safety visit three months after the final infusion. Cohort A subjects participated in the study for approximately 16 months. Based on the analysis of Cohort A, it was decided to extend the duration of treatment for a subset of subjects in Cohort B to evaluate the clinical response to VX15/2503 after 36 months. Additional enrolled subjects in Cohort B will be treated with drug or placebo (1:1). Participation in Cohort B will include a Screening visit, a Baseline visit within 30 days of screening; 18 or 36 monthly visits beginning at baseline and continuing through Month 18 or Month 36; follow-up safety phone call or visit, and for a subset of subjects, a follow-up safety visit three and six months after the final infusion. Cohort B subjects will participate in the study for approximately 19 and up to 37 months.

Clinical Study Identifier: NCT02481674

Contact Investigators or Research Sites near you

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Victor Sung, MD, PhD

University of Alabama at Birmingham
Birmingham, AL United States

Jody Corey-Bloom, MD, PhD

University of California, San Diego
La Jolla, CA United States

Lauren Seeberger, MD

University of Colorado - Denver
Aurora, CO United States

Stewart A Factor, DO

Emory University School of Medicine
Atlanta, GA United States

John Kamholz, MD

University of Iowa
Iowa City, IA United States

Kathrin LaFaver, MD

University of Louisville
Louisville, KY United States

Diana Rosas, MD, MS

Massachusetts General Hospital
Charlestown, MA United States

Praveen Dayalu, MD

University of Michigan
Ann Arbor, MI United States

Brad Racette, MD

Washington University
Saint Louis, MO United States

Karen Marder, MD, PhD

Columbia University
New York, NY United States

Richard Barbano, MD, PhD

University of Rochester
Rochester, NY United States

Burton Scott, MD, PhD

Duke University Health Center
Durham, NC United States

Francis Walker, MD

Wake Forest University
Winston-Salem, NC United States

Andrew Duker, MD

University of Cincinnati
Cincinnati, OH United States

Sandra Kostyk, MD, PhD

Ohio State University
Columbus, OH United States

Lawrence Elmer, MD, PhD

University of Toledo
Toledo, OH United States

Daniel Claassen, MD

Vanderbilt University
Nashville, TN United States

James Boyd, MD

University of Vermont
Burlington, VT United States

Karen Anderson, MD

Georgetown University
Washington, WA United States

Joanne Wojcieszek, MD

Indiana University School of Medicine
Indianapolis, IN United States

Samuel Frank, MD

Beth Israel Deaconess Medical Center
Boston, MA United States

Erin Stimming, MD

University of Texas Houston Medical School
Houston, TX United States

Claudia Testa, MD, PhD

Virginia Commonwealth University
Richmond, VA United States

Ali Samii, MD

University of Washington and VA Puget Sound Health Care System
Seattle, WA United States

Michael Geshwind, MD

University of California San Francisco
San Francisco, CA United States

Nikolaus McFarland, MD

University of Florida Gainesville
Gainesville, FL United States

Jee Bang, MD

Johns Hopkins University
Baltimore, MD United States

Ali Samii, MD

University of Washington
Seattle, WA United States

Oksana Suchowersky, MD

University of Alberta
Edmonton, AB Canada

Lynn Raymond, MD

University of British Columbia
Vancouver, BC Canada

Sylvain Chouinard

Centre hospitalier de l'Universit de Montr al (CHUM)
Montréal, QC Canada

Recruitment Status: Closed

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