A Study of FT-4202 in Adults and Adolescents With Sickle Cell Disease (HIBISCUS)

• Status: Recruiting
• Trial end date: 2026-12-01
• Target enrollment: 0
• Participant gender: All

Summary

This clinical trial is a Phase 2/3 study that will evaluate the efficacy and safety of FT-4202 and test how well FT-4202 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).

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Forma Therapeutics, Inc.

Criteria

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 g/dL (≥ 55 and ≤ 100 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

Key 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)

• Direct bilirubin > 3.0 × ULN

• Known HIV positive

• 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

• History of overt clinical stroke within previous 2 years or any history of an intracranial hemorrhage

Prior/Concomitant Therapy

• Patients receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion)

• 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

• Use of voxelotor within 28 days prior to starting study treatment or anticipated need for this agent during the study

• Use of a selectin antagonist (eg, crizanlizumab or other monoclonal antibody or small molecule) within 28 days of starting study treatment or anticipated need for such agents 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)