A Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sick… (NCT06198712) | Clinical Trial Compass
RecruitingPhase 2
A Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease
Canada, France, Kenya95 participantsStarted 2023-01-12
Plain-language summary
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.
Who can participate
Age range
6 Months – 18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Patient's parent, legal guardian, or legal representative has provided documented informed consent and patients have provided age-appropriate assent
. Age greater than or equal to (≥) 6 months and lesser than (\<) 18 years of age at time of enrollment, according to the enrolling cohort:
. Patient has confirmed diagnosis of SCD
. Hemoglobin ≥ 5.5 and lesser than or equal to (≤) 10.5 grams per deciliter (g/dL)
. Pediatric patients with severe SCD, as defined by at least 1 of the following:
. For participants taking hydroxyurea (HU), the dose of HU (mg/kg) must be stable (no more than a 20% change in dosing) for at least 90 days prior to start of study treatment with no anticipated need for dose adjustments during the study, in the opinion of the Investigator
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Single-dose: maximum plasma concentration (Cmax)
Timeframe: During the 24-week primary treatment period
2
Single-dose: area under the plasma concentration time curve from dosing (time 0) to time t ((AUC)0-t)
Timeframe: During the 24-week primary treatment period
3
Single-dose: area under the plasma concentration time curve from zero to time infinity (AUC0-inf)
Timeframe: During the 24-week primary treatment period
4
Steady-state maximum plasma concentration (Cmax,ss)
Timeframe: During the 24-week primary treatment period
5
Steady-state area under the concentration time curve over the dosing interval (AUCtau,ss)
Timeframe: During the 24-week primary treatment period
6
Steady-state average plasma concentration (Cavg,ss)
Timeframe: During the 24-week primary treatment period
. Patients on crizanlizumab or L-glutamine treatment at the time of consent may be eligible if they:
. Female patients of childbearing potential who are using acceptable methods of contraception and agree not to donate ova from study start to 90 days after the last dose of study drug, and male patients who are willing to use acceptable methods of contraception and agree not to donate sperm, from study start to 90 days after the last dose of study drug.
Exclusion criteria
. 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
. Abnormal TCD in the 12 months prior to starting study treatment