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Tests treatment safety and results for Sickle Cell Disease
Official title A Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease
ClinicalTrials.gov ID: NCT06198712
What this study is testing
What is Etavopivat?
Etavopivat is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for sickle cell disease.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 6 to 18
You may be able to join if
- 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
- Age greater than or equal to (≥) 6 months and lesser than (\<) 18 years of age at time of enrollment, according to the enrolling cohort:
- Cohort 1: age 12 to \< 18 years (adolescents)
- Cohort 2: age 6 to \< 12 years
You likely can't join if
- 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
- Abnormal TCD in the 12 months prior to starting study treatment Prior/Concomitant Therapy
- Patients receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion)
See the full eligibility criteria
- 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
- Age greater than or equal to (≥) 6 months and lesser than (\<) 18 years of age at time of enrollment, according to the enrolling cohort:
- Cohort 1: age 12 to \< 18 years (adolescents)
- Cohort 2: age 6 to \< 12 years
- Cohort 3: age 2 to \< 6 years
- Cohort 4: age 6 months to \< 2 years
- Patient has confirmed diagnosis of SCD
- Documentation of SCD genotype (HbSS, HbSβ0-thalassemia or other sickle cell syndrome variants) based on prior history of laboratory testing. Molecular genotyping is not required. SCD genotype may be determined from the...
- 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:
- 2-15 episodes of documented VOC within the 12 months prior to screening. Documentation must exist in the patient's medical record prior to screening. Events based solely on patient recall without supporting...
- Hospitalization for any SCD-related complication in the last 12 months prior to starting study treatment
- Proteinuria, defined as an albumin:creatinine ratio (ACR) \> 100 mg/g on 2 measures (separated by ≥ 1 month) as an indicator of early renal disease
- History of a conditional TCD in the last 12 months prior to starting study treatment, but not currently being treated with chronic transfusion therapy (applicable to participants \> 2 years of age). Conditional TCD is...
- 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...
- Patients on crizanlizumab or L-glutamine 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 (ie, no changes to the dose except for changes to weight or for safety reasons)
- For patients on crizanlizumab, have been ≥ 80% compliant with the planned regimen during the 12 months prior to the time of consent
- 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...
- 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
- Abnormal TCD in the 12 months prior to starting study treatment Prior/Concomitant Therapy
- Patients receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion)
- Received any blood products within 30 days of starting study treatment
- Receiving or use of concomitant medications that are strong inducers of cytochrome P450 (CYP) 3A4/5 within 2 weeks of starting study treatment
- Use of voxelotor within 28 days prior to starting study treatment or anticipated need for this agent during the study
- Receipt of erythropoietin or other hematopoietic growth factor treatment within 28 days of starting study treatment or anticipated need for such agents during the study
- Receipt of prior cellular based therapy (eg, hematopoietic cell transplant, gene modification therapy)
The study team makes the final eligibility decision.
Where it's taking place
- Toronto, Ontario, Canada
- Paris, France
- Pierre-Bénite, France
- Roeun, France
- Kericho, Kenya
- Kisumu, Kenya
- Siaya, Kenya
- Beirut, Lebanon
- Tripoli, Lebanon
- Ituku-Ozalla, Enugu State, Nigeria
- Lagos, Nigeria
- Tarauni, Nigeria
- Ankara, Turkey (Türkiye)
- Seyhan, Turkey (Türkiye)
- London, United Kingdom
- Manchester, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 6 months to 18 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Toronto, Ontario, Canada; Paris, France; Pierre-Bénite, France; Roeun, France; Kericho, Kenya; Kisumu, Kenya and 10 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.