NCT04624659

A Study of Etavopivat in Adults and Adolescents With Sickle Cell Disease (HIBISCUS)

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

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 450 (estimated)
• Sponsor: Forma Therapeutics, Inc. · Industry
• Sex: All
• Age: 12 Years – 65 Years
• Healthy volunteers: Not accepted

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).

Detailed description

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.

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
DRUG	Etavopivat Tablets Low dose	200 mg once daily
DRUG	Etavopivat Tablets High dose	400 mg once daily
DRUG	Placebo Tablets	Placebo once daily
DRUG	Etavopivat Tablets	Selected dose once daily

Timeline

• Start date: 2021-01-29
• Primary completion: 2026-02-06
• Completion: 2027-03-19
• First posted: 2020-11-12
• Last updated: 2026-04-06

Locations

142 sites across 17 countries: United States, Canada, Egypt, France, Germany, Ghana, Greece, India, Italy, Kenya, Lebanon, Nigeria, Oman, Saudi Arabia, Spain, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study