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

Forma Therapeutics, Inc.0 sites344 target enrollmentJanuary 27, 2022

Overview

No summary available.

Registry

who.int

Start Date

January 27, 2022

End Date

TBD

Last Updated

last year

Study Type

Interventional

Sex

All

Sponsor

•Participants are eligible to be included in the study only if all the following criteria apply:
•Informed Consent
•1\. Patient has provided documented informed consent or assent (the informed consent form \[ICF] must be reviewed and signed by each patient)
•2\. Age 18 to 65 years, inclusive, at time of randomization
•Type of Participant and Disease Characteristics
•3\. Patient has a confirmed diagnosis of sickle cell disease
•Documentation of SCD genotype (HbSS, HbSß0\-thalassemia or other sickle cell syndrome variants) based on prior history of laboratory testing; if unavailable, must be confirmed by laboratory testing during screening
•4\. Patient has had at least 2 episodes of VOC in the past 12 months
•For study eligibility, VOC is defined as a previously documented episode of ACS or acute painful crisis (for which there was no explanation other than VOC) which required prescription or healthcare professional\-instructed use of analgesics for moderate to severe pain (documentation must exist in the patient medical record prior to Screening)
•5\. Hemoglobin \= 5\.5 and \= 10 g/dL (\= 55 and \= 100 g/L) during screening

•Patients are excluded from the study if they meet any of the following criteria:
•Medical Conditions
•1\. More than 10 VOCs (as defined in Inclusion Criterion 4\) within the past 12 months
•2\. Hospitalized for sickle cell crisis or other vaso\-occlusive event within 14 days of signing the ICF
•3\. Female who is breast feeding or pregnant
•4\. Hepatic dysfunction characterized by:
•Alanine aminotransferase (ALT) \> 4\.0 × upper limit of normal (ULN)
•Direct bilirubin \> 3\.0 × ULN
•5\. Patients with clinically significant bacterial, fungal, parasitic, or viral infection requiring systemic therapy
•Patients with acute bacterial, fungal, parasitic, or viral infection requiring systemic therapy should delay screening/enrollment until active therapy has been completed