Phase 2/3 Adaptive Study of VX-147 in Adult and Pediatric Participants With APOL1- Mediated Proteinuric Kidney Disease

Purpose

The purpose of this study is to evaluate the efficacy, safety, tolerability, and pharmacokinetics (PK) of VX-147 in adult and pediatric participants with apolipoprotein L1 (APOL1)-mediated proteinuric kidney disease.

Condition

  • Proteinuric Kidney Disease

Eligibility

Eligible Ages
Between 10 Years and 65 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • APOL1 genotype of G1/G1, G2/G2, or G1/G2 - Proteinuric kidney disease

Exclusion Criteria

  • Solid organ or bone marrow transplant - Uncontrolled hypertension - History of diabetes mellitus - Known underlying cause of kidney disease including but not limited to sickle cell disease Other protocol defined Inclusion/Exclusion criteria apply.

Study Design

Phase
Phase 2/Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Sequential Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Phase 2: VX-147 		Participants will be randomized to receive different dose levels of VX-147.
  • Drug: VX-147
    Tablets for oral administration.
Placebo Comparator
Phase 2: Placebo 		Participants will receive placebo matched to VX-147.
  • Drug: Placebo
    Tablets for oral administration.
Experimental
Phase 3: VX-147 		Participants will receive VX-147 with the dose to be based on the outcome of Phase 2.
  • Drug: VX-147
    Tablets for oral administration.
Placebo Comparator
Phase 3: Placebo 		Participants will receive placebo matched to VX-147.
  • Drug: Placebo
    Tablets for oral administration.

Recruiting Locations

Alabama Kidney Research
Alabaster, Alabama 35007

Cardiology, P.C.
Birmingham, Alabama 35211

Nephrology Associates
Fairhope, Alabama 36532

Nephrology Consultants, LLC
Huntsville, Alabama 35805

Renal Associates of Alabama, LLC
Montgomery, Alabama 36117

EmVenio Research
Vestavia Hills, Alabama 35216

AKDHC Medical Research Service LLC - Banner Desert Office
Mesa, Arizona 85202

Phoenix Children's Hospital, Inc.
Phoenix, Arizona 85016

Cardiology and Medicine Clinic, P.A.
Little Rock, Arkansas 72204

University of Arkansas for Medical Sciences
Little Rock, Arkansas 72205

The Medical Research Group, Inc
Fresno, California 93720

Amicis Research Center
Granada Hills, California 91344

Renal Consultants Medical Group
Granada Hills, California 91344

Eastside Clinical Research Associates
Los Angeles, California 90022

Kaiser Permanente - Los Angeles Medical Center
Los Angeles, California 90027

Cedars-Sinai Medical Center
Los Angeles, California 90048

UCI Center for Clinical Research
Orange, California 92868

Kaiser Permanente, Sacramento
Sacramento, California 95825

UCSF School of Medicine
San Francisco, California 94143

Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Torrance, California 90502

Greater Hartford Nephrology
Bloomfield, Connecticut 06002

Nephrology & Hypertension Associates, PC
Middlebury, Connecticut 06762

Yale University
New Haven, Connecticut 06510

Children's National Hospital
Washington, District of Columbia 20010

The George Washington University Medical Faculty Associates - Kidney Disease & Hypertension
Washington, District of Columbia 20037

Bay Pines Healthcare System
Bay Pines, Florida 33744

Nova Clinical Research, LLC
Bradenton, Florida 34209

Clinical Research of Brandon, LLC
Brandon, Florida 33511-5937

AMR-Miami
Coral Gables, Florida 33134

South Florida Nephrology Group PA, Research Div.
Coral Springs, Florida 33071

Delray Physician Care Center
Delray Beach, Florida 33445

North Florida/South Georgia Veterans Health System
Gainesville, Florida 32608

University of Florida, Shands Hospital
Gainesville, Florida 32610

Velocity Clinical Research - Hallandale Beach
Hallandale Beach, Florida 33009

Qway Research
Hialeah, Florida 33010

UF Health Cardiovascular Center - Jacksonville
Jacksonville, Florida 32209

South Florida Research Institute
Lauderdale Lakes, Florida 33313

South Florida Research
Miami Springs, Florida 33166

Total Research Group, LLC
Miami, Florida 33126

Cordova Research Institute
Miami, Florida 33155

Accelerated Enrollment Solutions (AES) Orlando
Orlando, Florida 32806

Elixia Central
Orlando, Florida 32806

Omega Research Orlando, LLC
Orlando, Florida 32808

Mid Florida Kidney and Hypertension Care, PL
Sanford, Florida 32771

American Clinical Trials LLC - Acworth
Acworth, Georgia 30101

Emory School of Medicine - Renal Division
Atlanta, Georgia 30308

Advanced Clinical Research of Atlanta
Atlanta, Georgia 30309

Morehouse School of Medicine, Grady Memorial Hospital
Atlanta, Georgia 30310

Children's Healthcare of Atlanta - Center for Advanced Pediatrics
Atlanta, Georgia 30322

Fides Clinical Research
Atlanta, Georgia 30342

Medical College of Georgia (MCG)
Augusta, Georgia 30912-3140

Randomize Now
College Park, Georgia 30337

Clincept - Warm Springs
Columbus, Georgia 31904

Renal Associates, LLC
Columbus, Georgia 31904

Atlanta VA Healthcare System
Decatur, Georgia 30033

American Clinical Trials LLC - Douglasville
Douglasville, Georgia 30134

Southeast Kidney Associates
East Point, Georgia 30344

Javara Inc./Privia Medical Group Georgia, PLLC
Fayetteville, Georgia 30214

Georgia Nephrology
Lawrenceville, Georgia 30046

Javara Inc./Privia Medical Group Georgia, LLC
Savannah, Georgia 31406

Inova Clinical Trials and Research Center
Tyrone, Georgia 30290

Rophe Adult and Pediatric Medicine
Union City, Georgia 30291

AES Chicago (DRS)
Chicago, Illinois 60602

EmVenio Research
Chicago, Illinois 60612

NANI Research LLC
Evergreen Park, Illinois 60805

Edward Hines Jr VA Hospital
Hines, Illinois 60141

NANI Research LLC - Indiana
Fort Wayne, Indiana 46804

Indiana University
Indianapolis, Indiana 46202

University of Iowa
Iowa City, Iowa 52242

University of Kansas Medical Center
Kansas City, Kansas 66160

Renal Associates of Baton Rouge
Baton Rouge, Louisiana 70808

East Jefferson General Hospital
Metairie, Louisiana 70006

Barnum Medical Research
Natchitoches, Louisiana 71457

Louisiana State University Health Sciences Center LSUHSC
New Orleans, Louisiana 70112

University Medical Center - New Orleans
New Orleans, Louisiana 70112

Ochsner Medical Center - New Orleans
New Orleans, Louisiana 70121

Northwest Louisiana Nephrology, LLC - Shreveport
Shreveport, Louisiana 71101

Monroe Research, LLC
West Monroe, Louisiana 71291

Javara Inc./Privia Medical Group, LLC MidAtlantic - Main Research Site
Annapolis, Maryland 21401

Johns Hopkins Hospital
Baltimore, Maryland 21287

Kaiser Permanente, Mid-Atlantic
Gaithersburg, Maryland 20879

Patient First Clinical Trials
Nottingham, Maryland 21236

MD Medical Research
Oxon Hill, Maryland 20745

Javara Inc./Privia Medical Group, LLC MidAtlantic-Silver Spring, MD
Silver Spring, Maryland 20901

Tufts Medical Center
Boston, Massachusetts 02111

Boston Medical Center
Boston, Massachusetts 02118

Beth Israel Deaconess Medical Center
Boston, Massachusetts 02215

Whittier Street Health Center
Roxbury, Massachusetts 02120

Kidney Care and Transplant Services of New England
West Springfield, Massachusetts 01089

University of Michigan Health System - A. Alfred Taubman Health Care Center - Nephrology Clinic
Ann Arbor, Michigan 48109

Henry Ford Hospital
Detroit, Michigan 48202

AA Medical Research Center
Flint, Michigan 48504

Sparrow Clinical Research Institute
Lansing, Michigan 48912

Center for Advanced Kidney Research
Saint Clair Shores, Michigan 48081

Minneapolis VA Healthcare System
Minneapolis, Minnesota 55417

Nephrology Associates, P.C.
Columbus, Mississippi 39705

Olive Branch Family Medical Center
Olive Branch, Mississippi 38654

Nephrology and Hypertension Associates, LTD
Tupelo, Mississippi 38801

Washington University Saint Louis
Saint Louis, Missouri 63130

St. Louis Heart and Vascular, P.C.
Saint Louis, Missouri 63136

DaVita Clinical Research
Las Vegas, Nevada 89107

Hackensack University Medical Center
Hackensack, New Jersey 07601

Rutgers New Jersey Medical School
Newark, New Jersey 07103

New Mexico VA Healthcare System
Albuquerque, New Mexico 87108

Albert Einstein Hospital - Montefiore Medical Center
Bronx, New York 10461-1900

Montefiore Medical Center
Bronx, New York 10467

James J. Peters VA Medical Center
Bronx, New York 10468

Kings County Hospital
Brooklyn, New York 11203

SUNY Downstate Health Sciences University
Brooklyn, New York 11203

Clindove Research LLC
Brooklyn, New York 11221

Nephrology Associates, P.C. - Queens
Flushing, New York 11365

Zucker School of Medicine at Hofstra/Northwell
Great Neck, New York 11021

Clinical Research Development Associates LLC
Laurelton, New York 11413

VA New York Harbor Healthcare System
New York, New York 10010

New York University Grossman School of Medicine
New York, New York 10016

AES New York (DRS)
New York, New York 10017

The Rogosin Institute - The Rogosin Kidney Center
New York, New York 10021

Icahn School of Medicine at Mount Sinai
New York, New York 10029

Columbia University - Division of Nephrology
New York, New York 10032

NYC Health + Hospital/Harlem
New York, New York 10037

North Carolina Nephrology P.A. - Cary Office
Cary, North Carolina 27511

UNC Clinical and Translational Research Center
Chapel Hill, North Carolina 27599-7155

Metrolina Nephrology Associates
Charlotte, North Carolina 28208

DUHS Children's Health Center T0909
Durham, North Carolina 27710

Eastern Nephrology Associates - Greenville Office
Greenville, North Carolina 27834

Eastern Nephrology Associates - Jacksonville Office
Jacksonville, North Carolina 28546

Eastern Nephrology Associates - Kinston Office
Kinston, North Carolina 28504

Eastern Nephrology Associates - New Bern Office
New Bern, North Carolina 28562

Eastern Nephrology Associates - Wilmington Office
Wilmington, North Carolina 28401

University of Cincinnati College of Medicine
Cincinnati, Ohio 45206

University Hospitals Cleveland Medical Center
Cleveland, Ohio 44106

VA Cleveland Northeast Ohio Healthcare System
Cleveland, Ohio 44106

Cleveland Clinic
Cleveland, Ohio 44195

Ohio State University
Columbus, Ohio 43201

Thomas Jefferson University
Philadelphia, Pennsylvania 19107

Temple University Hospital
Philadelphia, Pennsylvania 19140

Einstein Medical Center (NDKD)
Philadelphia, Pennsylvania 19141

Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania 15224

Lifespan Clinical Research Center
East Providence, Rhode Island 02915

Pharmacorp Clinical Trials, Inc
Charleston, South Carolina 29412

Medical University of South Carolina - Nexus Research Clinic
Charleston, South Carolina 29425

Columbia Nephrology Associates, PA
Columbia, South Carolina 29203

Carolina Nephrology, PA
Spartanburg, South Carolina 29306

Southeast Renal Research Institute
Chattanooga, Tennessee 37404

Le Bonheur Children's Hospital - Outpatient Clinic
Memphis, Tennessee 38105

Clinical Neuroscience Solutions (CNS Healthcare) - Memphis Research Center
Memphis, Tennessee 38119

Meharry Medical College
Nashville, Tennessee 37208

Vanderbilt University Medical Center, Nephrology Clinical Trials Center
Nashville, Tennessee 37232

Baylor Scott & White Research Institute
Dallas, Texas 75204

Dallas Renal Group - 2
Dallas, Texas 75230

Dallas Renal Group - 1
Dallas, Texas 75237

Dallas Nephrology Associates - Dallas Landry Office
Dallas, Texas 75246

Southwest Family Medicine Associates (SFMA) - Dallas
Dallas, Texas 75325

UT Southwestern Medical Center
Dallas, Texas 75390

Davita Clinical Research - El Paso
El Paso, Texas 79925

Cook Children's Medical Center
Fort Worth, Texas 76104

Ronald Ralph MD PA
Houston, Texas 77054-2101

Prolato Clinical Research Center
Houston, Texas 77054

North Texas Kidney Disease Associates
Lewisville, Texas 75057

Prime Clinical Research Inc - Lewisville
Lewisville, Texas 75067

Texas Tech University Health Sciences Center
Lubbock, Texas 79430

Prime Clinical Research
Mansfield, Texas 76063

Kidney Specialists of N Houston, PLLC
Shenandoah, Texas 77384

Renal Physicians of Montgomery County, PA
The Woodlands, Texas 77384

Tranquil Clinical Research
Webster, Texas 77598

University of Virginia Health - Nephrology Clinical Research Center
Charlottesville, Virginia 22908

TKS Research, P.L.L.C.
Norfolk, Virginia 23504

York Clinical Research, LLC
Norfolk, Virginia 23504

Richmond VAMC
Richmond, Virginia 23249

Virginia Commonwealth University - Division of Nephrology
Richmond, Virginia 23298

University of Washington Medical Center
Seattle, Washington 98195

Medical College of Wisconsin
Milwaukee, Wisconsin 53226

Milwaukee Nephrologists, SC
Wauwatosa, Wisconsin 53226

Alliance, School of Medicine
Rio Piedras, Puerto Rico 00935

More Details

NCT ID
NCT05312879
Status
Recruiting
Sponsor
Vertex Pharmaceuticals Incorporated

Study Contact

Medical Information
617-341-6777
medicalinfo@vrtx.com