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

STATUS

Recruiting
End date

Dec 24, 2026
participants needed

344
sponsor

Forma Therapeutics, Inc.

Updated on 24 April 2022

See if I qualify

hydroxyurea

glutamine

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).

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 are two 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 the second interim analysis, and then will be tested along with evaluation of efficacy on vaso-occlusive crises at the final analysis. Following completion of 52 weeks of double-blind treatment, patients may enter a 52-week etavopivat open-label extension period.

Details

Condition	Sickle Cell Disease
Treatment	Placebo Tablets, FT-4202 Tablets, Etavopivat tablets
Clinical Study Identifier	NCT04624659
Sponsor	Forma Therapeutics, Inc.
Last Modified on	24 April 2022

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Eligibility		Yes	No	Not Sure
Inclusion Criteria
	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 ≤ 10.5 g/dL (≥ 55 and ≤ 105 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
	Patients on crizanlizumab or L-glutamine treatment at the time of consent must be on a stable dose for ≥ 12 months and must be ≥ 80% compliant with the planned regimen at the time of consent and meet the VOC eligibility criteria
Exclusion Criteria
	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)
	Known HIV positive
	Direct bilirubin > 3.0 × ULN
	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
	Prior/Concomitant Therapy
	History of overt clinical stroke within previous 2 years or any history of an
	Patients receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion)
	intracranial hemorrhage
	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
	History of deep venous thrombosis requiring systemic anti-coagulation therapy for ≥ 6 weeks, occurring within 6 months prior to Day 1 of study treatment
	Use of voxelotor within 28 days prior to starting study treatment or anticipated need for this agent 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)
	Use of an experimental selectin antagonist (eg, monoclonal antibody or small molecule) within 28 days of starting study treatment or anticipated need for such agents during the study

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