The purpose of this study is to evaluate the pharmacokinetics and safety of etavopivat in paediatric participants with sickle cell disease (SCD). Participants will receive etavopivat and will be enrolled in a staggered manner, starting with the oldest age group and followed sequentially by younger cohorts after review of pharmacokinetic and safety data from the preceding cohort. All participants will undergo a 24-week primary treatment period followed by a 72-week extension treatment period to further evaluate long-term safety and pharmacokinetics of etavopivat. The total duration of the study will be approximately 96 weeks.
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 or granules 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. This study is currently undergoing a sponsor transition from Forma Therapeutics, Inc. to Novo Nordisk A/S.
Collaborator