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
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 IdentifierNCT04624659
SponsorForma Therapeutics, Inc.
Last Modified on24 April 2022

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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