An Adaptive, Randomized, Placebo-controlled, Double-blind, Multi-center Study of Oral FT-4202, a Pyruvate Kinase Activator in Patients With Sickle Cell Disease (HIBISCUS)

About the study

This clinical trial is a Phase 2/3 study that will evaluate the efficacy and safety of
FT-4202 and test how well FT-4202 works compared to placebo to improve the amount of
hemoglobin in the blood and to reduce the number of vaso-occlusive crises (times when the
blood vessels become blocked and cause pain).

Study point of contact

Forma Therapeutics, Inc.
617-679-1970
[email protected]

Locations

23 United States sites

Age

12 to 65 Years

Phase

Phase 2/Phase 3

Study type

Interventional

Gender

All

Interventions

Drug

Compensation

Unknown

participation requirements

– Provision of consent

– Patient has a confirmed diagnosis of sickle cell disease

– At least 2 episodes of vaso-occlusive crises in the past 12 months

– Hemoglobin ≥ 5.5 and ≤ 10 g/dL (≥ 55 and ≤ 100 g/L) during screening

– Patients taking hydroxyurea, must demonstrate a stable dose for at least 90 days prior
to start of study treatment

– Female patients of childbearing potential must use highly effective methods of
contraception, male patients are willing to use barrier methods of contraception

Key

participation restrictions

Medical Conditions

– More than 10 vaso-occlusive crises within the past 12 months

– Female who is breast feeding or pregnant

– Hepatic dysfunction characterized by:

– Alanine aminotransferase (ALT) > 4.0 × upper limit of normal (ULN)

– Direct bilirubin > 3.0 × ULN

– Known HIV positive

– Active hepatitis B or hepatitis C infection

– Severe renal dysfunction or on chronic dialysis

– History of unstable or deteriorating cardiac or pulmonary disease within 6 months
prior to consent including but not limited to the following:

– Unstable angina pectoris or myocardial infarction or elective coronary
intervention

– Congestive heart failure requiring hospitalization

– Uncontrolled clinically significant arrhythmias

– Symptomatic pulmonary hypertension

– History of overt clinical stroke within previous 2 years or any history of an
intracranial hemorrhage

Prior/Concomitant Therapy

– Patients receiving regularly scheduled blood (RBC) transfusion therapy (also termed
chronic, prophylactic, or preventive transfusion)

– Receiving or use of concomitant medications that are strong inducers or
moderate/strong inhibitors of CYP3A4/5 within 2 weeks of starting study treatment or
anticipated need for such agents during the study

– Use of voxelotor within 28 days prior to starting study treatment or anticipated need
for this agent during the study

– Use of a selectin antagonist (eg, crizanlizumab or other monoclonal antibody or small
molecule) within 28 days of starting study treatment or anticipated need for such
agents during the study

– Use of erythropoietin or other hematopoietic growth factor treatment within 28 days of
starting study treatment or anticipated need for such agents during the study

– Receipt of prior cellular-based therapy (eg, hematopoietic cell transplant, gene
modification therapy)

Locations

  • Little Rock, Arkansas, United States, Woodland International Research Group, 72211 [Recruiting]
  • Long Beach, California, United States, Collaborative Neuroscience Research, LLC., 90806 [Recruiting]
  • Oakland, California, United States, Pacific Research Partners, LLC, 94607 [Recruiting]
  • Sacramento, California, United States, UC Davis Medical Center - UC Davis Comprehensive Cancer Center - Hemotology/Oncology Clinic, 95817 [Recruiting]
  • Washington, District of Columbia, United States, Howard University, 20060 [Recruiting]
  • Altamonte Springs, Florida, United States, Cornerstone Research Institute, 32701 [Recruiting]
  • Hollywood, Florida, United States, Foundation for Sickle Cell Disease Research, 33021 [Recruiting]
  • Miami, Florida, United States, Advanced Pharma CR LLC., 33147 [Recruiting]
  • Atlanta, Georgia, United States, Sonar Clinical Research, 30331 [Recruiting]
  • Atlanta, Georgia, United States, Children's Healthcare of Atlanta - Pediatric Research Center, 30342 [Recruiting]
  • Chicago, Illinois, United States, University of Illinois College of Medicine (UICM) - Chicago - Outpatient Care Center (OCC) - Pediatric Clinic, 60612 [Recruiting]
  • Baltimore, Maryland, United States, University of Maryland School of Medicine, 21201 [Recruiting]
  • Boston, Massachusetts, United States, Boston Medical Center, 02118 [Recruiting]
  • Saint Louis, Missouri, United States, Washington University School of Medicine Barnes - Jewish Hospital, 63110 [Recruiting]
  • Omaha, Nebraska, United States, University of Nebraska Medical Center, 68198 [Recruiting]
  • Bronx, New York, United States, Jacobi Medical Center, 10461 [Recruiting]
  • Brooklyn, New York, United States, Kings County Hospital, 11203 [Recruiting]
  • Jamaica, New York, United States, Queens Hospital Center, 11432 [Recruiting]
  • Chapel Hill, North Carolina, United States, UNC School of Medicine, 27514 [Recruiting]
  • Durham, North Carolina, United States, Duke University - Sickle Cell Center, 27705 [Recruiting]
  • North Canton, Ohio, United States, Neuro-Behavioral Clinical Research, 44720 [Recruiting]
  • Tulsa, Oklahoma, United States, Lynn Institute of Tulsa, 74135 [Recruiting]
  • Greenville, South Carolina, United States, Prisma Health, 29605 [Recruiting]
Last updated 2021-08-06