This study is a randomized, placebo-controlled, double-blind, multicenter Phase 2/3 study of patients, age 12 to 65 years, with sickle cell disease (SCD).

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 450

Inclusion Criteria

1) Provision of consent
2) Patient has a confirmed diagnosis of sickle cell disease
3) 2-15 episodes of documented vaso-occlusive crises in the past 12 months
4) Hemoglobin = 5.5 and = 10.5 g/dL (= 55 and = 105 g/L) during screening
5) Patients taking hydroxyurea, must demonstrate a stable dose for at least 90 days prior to start of study treatment
6) Female patients of childbearing potential must use acceptable methods of contraception, male patients are willing to use acceptable methods of contraception
7)Patients on crizanlizumab or L-glutamine oral powder (Endari®) treatment at the time of consent may be eligible if they:
•Have been on a stable dose for = 12 months at the time of consent (i.e., no changes to the dose except for changes to weight or for safety reasons)
•Have been = 80% compliant with the planned regimen during the 12 months prior to the time of consent
•Meet the VOC eligibility requirement in Inclusion Criterion 4

Are the trial subjects under 18? yes
Number of subjects for this age range: 40
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 408
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 2

Exclusion Criteria

Medical Conditions
1) More than 15 vaso-occlusive crises within the past 12 months prior to screening
2) Female who is breast feeding or pregnant
3) Hepatic dysfunction characterized by:
- Alanine aminotransferase (ALT) > 4.0 × upper limit of normal (ULN)
OR
- Direct bilirubin > 3.0 × ULN
4) Known HIV positive
5) Active hepatitis B or hepatitis C infection
6) Severe renal dysfunction or on chronic dialysis
7) 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
8) History of overt clinical stroke within previous 2 years or any history of an intracranial hemorrhage
9) History of deep venous thrombosis requiring systemic anti-coagulation therapy for = 6 weeks, occurring within 6 months prior to Day 1 of study treatment.
Note: patients on = 6 months of chronic or prophylactic anti-coagulation therapy are allowed on study.

Prior/Concomitant Therapy
1) Patients receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion)
2) Receiving or use of concomitant medications that are strong inducers of CYP3A4/5 within 2 weeks of starting study treatment or anticipated need for such agents during the study
3) Use of voxelotor within 28 days prior to starting study treatment or anticipated need for this agent during the study
4) Use of an experimental selectin antagonist (e.g., monoclonal antibody or small molecule) within 28 days of starting study treatment or anticipated need for such agents during the study
5) 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
6) Receipt of prior cellular-based therapy (e.g., hematopoietic cell transplant, gene modification therapy)

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

Related Trials

An Adaptive, Randomized, Placebo-controlled, Double-blind, Multi-center Study of Oral FT-4202, a Pyruvate Kinase Activator in Patients with Sickle Cell Disease (PRAISE)

Not Applicable

Forma Therapeutics Inc.

Updated 5/29/2023