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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
95
Participants will receive oral tablets or granules of etavopivat once daily.
The Hospital for Sick Children
Toronto, Ontario, Canada
COMPLETEDHCL-HOPITAL LYON SUD_Institut d'Hématologie et d'Oncologie Pédiatrique (IHOPe)
Lyon, France
NOT_YET_RECRUITINGAPHP - Centre de Référence des Syndromes
Paris, France
NOT_YET_RECRUITINGCentre Hospitalier Universitaire de Rouen-Hopital Charles Nicolle
Roeun, France
NOT_YET_RECRUITINGKEMRI CRDR Siaya Clinical Research Annex, Country Referral Hospital
Siaya, Siaya County, Kenya
RECRUITINGKEMRI-Walter-Reed Kericho
Kericho, Kenya
RECRUITINGKombewa Clinical Research Centre
Kisumu, Kenya
RECRUITINGAhero Clinical Trials Unit
Kisumu, Kenya
RECRUITINGAmerican University of Beirut Medical center
Beirut, Lebanon
RECRUITINGHospital Nini
Tripoli, Lebanon
RECRUITING...and 8 more locations
Single-dose: maximum plasma concentration (Cmax)
Time frame: During the 24-week primary treatment period
Single-dose: area under the plasma concentration time curve from dosing (time 0) to time t ((AUC)0-t)
Time frame: During the 24-week primary treatment period
Single-dose: area under the plasma concentration time curve from zero to time infinity (AUC0-inf)
Time frame: During the 24-week primary treatment period
Steady-state maximum plasma concentration (Cmax,ss)
Time frame: During the 24-week primary treatment period
Steady-state area under the concentration time curve over the dosing interval (AUCtau,ss)
Time frame: During the 24-week primary treatment period
Steady-state average plasma concentration (Cavg,ss)
Time frame: During the 24-week primary treatment period
Steady-state minimum plasma concentration (Cmin,ss)
Time frame: During the 24-week primary treatment period
Incidence of adverse events (AEs), serious adverse events (SAEs), and AEs related to etavopivat
Time frame: During the 24-week primary treatment period
Number of premature discontinuations
Time frame: During the 24-week primary treatment period
Number of dose interruptions
Time frame: During the 24-week primary treatment period
Number of dose reductions
Time frame: During the 24-week primary treatment period
Incidence of AEs, SAEs, and AEs related to etavopivat
Time frame: During the 72-week treatment extension period
Number of premature discontinuations
Time frame: During the 72-week treatment extension period
Number of dose interruptions
Time frame: During the 72-week treatment extension period
Number of dose reductions
Time frame: During the 72-week treatment extension period
Hemoglobin (Hb) response rate
Time frame: Baseline, week 12 and 24
Change in Hb from baseline
Time frame: Baseline, week 12 and 24
Change from baseline in number of vaso-occlusive crises (VOCs)
Time frame: Baseline and week 24
Change from baseline in Annualized Rate of VOC
Time frame: Baseline and week 24
Change from baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) Fatigue Scale
PROMIS is a 10-item patient-reported health outcome measurement system used to evaluate quality of life in both children and adults. The assessment is based on the responses - 1. Never, 2. Almost never, 3. Sometimes, 4. Often and 5. Almost always. 'Never' response on the PROMIS fatigue scale indicates better quality of life.
Time frame: Baseline, week 12 and 24
Change from baseline in time-averaged mean of the maximum velocity (TAMMV) by transcranial Doppler ultrasonography (TCD)
Time frame: Baseline, week 24, 48, and 96
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.