Clinical Trial
Study Details
Participants will receive FT-4202/placebo and monitored for side effects while undergoing pharmacokinetics and pharmacodynamic studies
FT-4202 is an oral small-molecule agonist of pyruvate kinase red blood cell isozyme (PKR) being developed for the treatment of hemolytic anemias. This initial study will characterize the safety, tolerability and the pharmacokinetics/pharmacodynamics (PK/PD) of a single ascending dose and multiple ascending doses of FT-4202 in the context of Phase 1 studies in healthy volunteers and sickle cell disease patients. The effects of food on the absorption of FT-4202 will also be evaluated in healthy volunteers.
Study Overview
This is a first-in-human (FIH), Phase 1 study of FT-4202 that will characterize the safety, PK and PD of FT-4202 after a single dose and after repeated dosing first in healthy adult volunteers and then in adolescents or adults with sickle cell disease (SCD). Initially, a dose range of FT-4202 in single ascending dose (SAD) escalation cohorts will be explored in healthy subjects. Enrollment of healthy subjects into 2-week multiple ascending dose (MAD) escalation cohorts will be initiated once the safety and PK from at least two SAD cohorts is available to inform the doses for the 2-week MAD portion of the study. The MAD cohorts will then run in parallel to the single dose cohorts. A single dose cohort of healthy subjects is planned to understand food effects (FE) on the PK of FT-4202. After the SAD and FE studies in healthy subjects are completed, the safety, PK, and PD of a single dose of FT-4202 that was found to be safe in healthy subjects will then be evaluated in SCD subjects. Multiple dose studies in SCD subjects will then be initiated upon completion of MAD studies in healthy volunteers.
Contacts and Locations
Woodland International Research Group (SCD subjects only)
Little Rock, Arkansas 72211
United States
Collaborative Neuroscience Research, LLC (SCD subjects only)
Long Beach, California 90806
United States
Pacific Research Partners (SCD subjects only)
Oakland, California 94607
United States
UCSF Benioff Children's Hospital Oakland (SCD subjects only)
Oakland, California 94609
United States
Advanced Pharma CR, LLC (SCD subjects only)
Miami, Florida 33147
United States
Children's Healthcare of Atlanta (SCD subjects only)
Atlanta, Georgia 30342
United States
Augusta University Medical Center (SCD subjects only)
Augusta, Georgia 30912
United States
University of Illinois at Chicago (SCD subjects only)
Chicago, Illinois 60612
United States
University of Maryland, Greenebaum Comprehensive Cancer Center (SCD subjects only)
Baltimore, Maryland 21201
United States
Columbia University Medical Center (SCD subjects only)
New York, New York 10032
United States
Levine Cancer Institute (SCD subjects only)
Charlotte, North Carolina 28204
United States
Duke University Medical Center (SCD subjects only)
Durham, North Carolina 27710
United States
University of Cincinnati Medical Center (SCD subjects only)
Cincinnati, Ohio 45219
United States
Medpace Clinical Pharmacology Unit (Healthy Volunteers only)
Cincinnati, Ohio 45227
United States
Cincinnati Children's Hospital Medical Center (SCD subjects only)
Cincinnati, Ohio 45229
United States
Lynn Institute of Tulsa (SCD subjects only)
Tulsa, Oklahoma 74135
United States
St. Jude Children's Research Hospital (SCD subjects only)
Memphis, Tennessee 38105
United States
The University of Texas Health Science Center at Houston (SCD subjects only)
Houston, Texas 77030
United States