A Randomized, Placebo-controlled, Double Blind, Single Ascending and Multiple Ascending Dose Study to Assess the Safety, Pharmacokinetics and Pharmacodynamics of FT-4202 in Healthy Volunteers and Sickle Cell Disease Patients

Study point of contact

Forma Therapeutics
617-679-1970
[email protected]

Locations

19 United States sites

Age

12 to 65 Years

Phase

Phase 1

Study type

Interventional

Gender

All

Interventions

Drug

Compensation

Unknown

About the study

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.

participation requirements

– Must be between 12 and 65 years of age

– Previously diagnosed sickle cell disease (hemoglobin electrophoresis or genotype)

– Must have a minimum body weight of 40 kg (88 lbs) at the Screening Visit

– Must have the ability to understand and sign written informed consent (and assent
where applicable), which must be obtained prior to any study-related procedures being
completed

– All male and female patients of child bearing potential must agree to use medically
accepted contraceptive regimen during study participation and for 90 days after last
study drug administration

– Must be willing to abide by all study requirements and restrictions

SCD Key

participation restrictions

– Had more than 6 episodes of vaso-occlusive crisis (VOC) within the past 12 months that
required a hospital, emergency room, or clinic visit

– Had a least one episode of acute chest syndrome in the last 6 months

– Received any of the following approved therapies for use in SCD:

– Hydroxurea (HU): excluded if started HU < 90 days prior to Day 1 of study treatment - Adakveo®: excluded if received an infusion within 14 days prior to Day 1 of study treatment - Oxbryta®: excluded if received a dose within 7 days prior to start of Day 1 of study treatment - Received a red blood cell transfusion within 30 days of starting the study drug - Hemoglobin < 7.0 g/dL or > 10.5 g/dL

– Unable to take and absorb oral medications

HEALTHY VOLUNTEER

Locations

  • Little Rock, Arkansas, United States, Woodland International Research Group (SCD subjects only), 72211 [Recruiting]
  • Long Beach, California, United States, Collaborative Neuroscience Research, LLC (SCD subjects only), 90806 [Recruiting]
  • Oakland, California, United States, Pacific Research Partners (SCD subjects only), 94607 [Recruiting]
  • Oakland, California, United States, UCSF Benioff Children's Hospital Oakland (SCD subjects only), 94609 [Recruiting]
  • Miami, Florida, United States, Advanced Pharma CR, LLC (SCD subjects only), 33147 [Recruiting]
  • Atlanta, Georgia, United States, Atlanta Center for Medical Research (SCD subjects only), 30331 [Withdrawn]
  • Atlanta, Georgia, United States, Children's Healthcare of Atlanta (SCD subjects only), 30342 [Recruiting]
  • Augusta, Georgia, United States, Augusta University Medical Center (SCD subjects only), 30912 [Recruiting]
  • Chicago, Illinois, United States, University of Illinois at Chicago (SCD subjects only), 60612 [Recruiting]
  • Baltimore, Maryland, United States, University of Maryland, Greenebaum Comprehensive Cancer Center (SCD subjects only), 21201 [Recruiting]
  • New York, New York, United States, Columbia University Medical Center (SCD subjects only), 10032 [Not yet recruiting]
  • Charlotte, North Carolina, United States, Levine Cancer Institute (SCD subjects only), 28204 [Recruiting]
  • Durham, North Carolina, United States, Duke University Medical Center (SCD subjects only), 27710 [Recruiting]
  • Cincinnati, Ohio, United States, University of Cincinnati Medical Center (SCD subjects only), 45219 [Recruiting]
  • Cincinnati, Ohio, United States, Medpace Clinical Pharmacology Unit (Healthy Volunteers only), 45227 [Completed]
  • Cincinnati, Ohio, United States, Cincinnati Children's Hospital Medical Center (SCD subjects only), 45229 [Recruiting]
  • Tulsa, Oklahoma, United States, Lynn Institute of Tulsa (SCD subjects only), 74135 [Recruiting]
  • Memphis, Tennessee, United States, St. Jude Children's Research Hospital (SCD subjects only), 38105 [Recruiting]
  • Houston, Texas, United States, The University of Texas Health Science Center at Houston (SCD subjects only), 77030 [Recruiting]
Last updated 2021-02-11 Enroll Now