Phase 2 Sickle Cell Disease Trial, Recruiting

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• A parent, legal guardian, or legal representative has given written consent, and the child has given age-appropriate agreement to participate.
• The child is between 6 months and 17 years old at the time of enrolment, placing them in one of four age groups: 12–17 years, 6–11 years, 2–5 years, or 6 months to under 2 years.
• The child has a confirmed diagnosis of sickle cell disease (SCD), with prior lab results showing the specific type (such as HbSS or HbSβ0-thalassemia, or similar variants).
• The child's haemoglobin level is between 5.5 and 10.5 g/dL (a measure of red blood cell health).
• The child has severe SCD, meaning at least one of the following applies: 2–15 documented painful crises (VOCs) in the past 12 months recorded in medical notes; a hospital stay for any SCD-related problem in the past 12 months; two kidney protein tests showing elevated levels more than a month apart; or a borderline-abnormal brain blood flow scan (called a conditional TCD) in the past 12 months, in children over 2 years old who are not currently on regular transfusion therapy.
• If the child is taking hydroxyurea, the dose must have been stable (no more than a 20% change) for at least 90 days before starting the study, with no expected dose changes during the study.
• If the child is taking crizanlizumab or L-glutamine, they must have been on a stable dose for at least 12 months before consent (except for weight-related or safety adjustments), and crizanlizumab users must have taken at least 80% of planned doses during that time.
• Children who could become pregnant must agree to use acceptable contraception and not donate eggs from the start of the study until 90 days after the last study dose; male participants must agree to use acceptable contraception and not donate sperm during the same period.

Who may not be able to join:

• Girls or young women who are pregnant or currently breastfeeding.
• Children who had more than 15 painful crises (VOCs) requiring a hospital, emergency room, or clinic visit in the 12 months before starting the study treatment.
• Children who were hospitalised for a sickle cell crisis or similar painful event in the 14 days before starting the study treatment.
• Children who had an abnormal brain blood flow scan (TCD) in the 12 months before starting the study treatment.
• Children currently receiving regular, scheduled blood transfusions (sometimes called chronic or preventive transfusion therapy).
• Children who received any blood products within 30 days before starting the study treatment.
• Children currently taking certain medications that strongly affect how the body processes drugs (strong CYP3A4/5 inducers) within 2 weeks of starting the study treatment (confirm with trial site).
• Children who used the medication voxelotor within 28 days before starting the study treatment, or who are expected to need it during the study.
• Children who received erythropoietin or similar medications that stimulate blood cell production within 28 days before starting the study treatment, or who are expected to need them during the study.
• Children who have previously received a stem cell transplant or gene therapy.

Important: Always verify eligibility with the trial site directly before applying.