Summary

Eligibility
for people ages 12-65 (full criteria)
Location
at Oakland, California and other locations
Dates
study started
completion around

Description

Summary

This clinical trial is a Phase 2/3 study that will evaluate the efficacy and safety of etavopivat and test how well etavopivat 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).

Official Title

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

Details

Etavopivat is designed to activate PKR and thereby modulate RBC metabolism by impacting two critical pathways in RBCs. The etavopivat clinical development program will investigate whether decreasing 2,3-DPG may help oxygen bind to hemoglobin (i.e. increasing oxygen affinity), and thereby increase ATP and impact RBC function. This study is a randomized, placebo-controlled, double-blind, multicenter Phase 2/3 study of patients age 12 to 65 years (inclusive), with sickle cell disease. There is one planned interim analyses in this study design. Initially, patients will be randomized at 1:1:1 to one of two dose levels of etavopivat or placebo. At the first interim analysis, one of the two etavopivat dose levels will be selected for the Phase 3 portion of the study, in which patients will be randomized at 1:1 to the selected etavopivat dose or placebo. Efficacy on hemoglobin will be evaluated at conclusion of the double-blind treatment period. Following completion of 52 weeks of double-blind treatment, patients may enter a 112-week etavopivat open-label extension period.

Keywords

Sickle Cell Disease, Sickle Cell, Anemia, Sickle Cell Anemia, Hemolytic, Hemoglobin, Vaso-occlusive Crisis, Sickle Cell Crisis, Congenital Anemia, Hemolytic Anemia, Hematologic Disease, Hemoglobinopathies, Genetic Disease, Inborn Disease, Sickle Cell Trait, Pyruvate Kinase, Etavopivat Tablets Low dose, Etavopivat Tablets High dose, Etavopivat Tablets, Open label etavopivat

Eligibility

Locations

  • UCSF Benioff Children's Hospital Oakland
    Oakland California 94609 United States
  • UCSF Oakland Benioff ChildHosp
    Oakland California 94609 United States
  • Pacific Research Partners, LLC
    Oakland California 94607 United States
  • Pacific Research Partners
    Oakland California 94607 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Forma Therapeutics, Inc.
ID
NCT04624659
Phase
Phase 3 research study
Study Type
Interventional
Participants
Expecting 450 study participants
Last Updated