This study is being done to learn about etavopivat, a once a day medicine taken by mouth in adolescents with sickle cell disease. The main goals are to study safety and how long etavopivat stays in the bloodstream, while also studying if there are benefits from taking etavopivat. Eligible participants who enter the study will start a 96-week treatment period. At the end of the 96 weeks, participants will have an end of study visit that occurs 4 weeks later. The participants will receive etavopivat every day throughout the treatment period.
Type of Participant and Disease Characteristics
Patient's parent, legal guardian, or legal representative has provided documented informed consent and patients have provided age-appropriate assent
Cohort 1: age 12 to < 18 years (adolescents)
Cohort 2: age 6 to < 12 years
Medical Conditions
Female who is breastfeeding or pregnant
More than 15 VOCs within the 12 months prior to starting study treatment that required a hospital, emergency room (ER), or clinic visit
Hospitalized for sickle cell crisis or other vaso-occlusive event occurring in the 14 days prior to starting study treatment
Single group
Etavopivat
DrugParticipants will receive oral tablets of etavopivat once daily.
Single-dose: maximum plasma concentration (Cmax)
Single-dose: area under the plasma concentration time curve from dosing (time 0) to time t ((AUC)0-t)
Single-dose: area under the plasma concentration time curve from zero to time infinity (AUC0-inf)
Steady-state maximum plasma concentration (Cmax,ss)
Incidence of AEs, SAEs, and AEs related to etavopivat
Number of premature discontinuations
Number of dose interruptions
Number of dose reductions
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Forma Therapeutics, Inc.
Lead sponsor
Novo Nordisk A/S
Collaborator
