Haematological Disorders Paediatrics
Conditions
Interventions
Placebo
Sponsors
Novo Nordisk
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Participants are eligible to be included in the study only if all the following criteria apply: 1. Informed consent obtained before any study-related activities. Study-related activities are any procedures that are carried out as part of the study, including activities to determine suitability for the study. 2. Male or female. 3. Age 2 to 11 years of age (both inclusive) at the time of randomisation. 4. Participant has a confirmed diagnosis of SCD: • Documentation of SCD genotype (HbSS, HbSC, HbSß0-thalassaemia, HbSß+ 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 results of Hb electrophoresis, high-performance liquid chromatography, or similar testing. Note that Hb electrophoresis is performed by the central laboratory at screening. 5. Haemoglobin = 5.5 and = 10.5 g/dL at screening. 6. At least 2 episodes and a maximum of 15 episodes of documented VOC within the 12 months prior to screening. Documentation must exist in the participant’s medical record prior to screening. Events based solely on participant/parent recall without supporting documentation should not be counted towards eligibility. 7. For participants taking 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 screening with no anticipated need for dose adjustments (other than weight-based) during the study, in the opinion of the investigator. 8. For participants taking L-glutamine, the dose must be stable (both if taken as a food supplement or as a medicinal product) for =12 months prior to screening with no anticipated need for dose adjustments (other than weight-based) during the study and they must have been = 80% compliant with the planned regimen during the 12 months prior to randomisation, in the opinion of the investigator.
Exclusion criteria
Exclusion criteria: 1. Known or suspected hypersensitivity to study intervention(s) or related products. 2. Previous randomisation in this study. 3. More than two re-screenings for this study. 4. Female who is pregnant, breast-feeding or intends to become pregnant or is of childbearing potential and not using adequate contraceptive method, as defined in Appendix 4 (Section 10.4). 5. Current participation (i.e., signed informed consent) in any other interventional clinical study. 6. Exposure to an investigational medicinal product (IMP) within 28 days or 5 half-lives of the IMP (if known), whichever is longer, before randomisation. 7. Body weight 3 × upper limit of normal (ULN). • Direct bilirubin > 2 × ULN • Liver disease with histopathological evidence or clinical diagnosis of cirrhosis or severe fibrosis. 9. Use of any herbal or dietary supplement without a stable dosing regimen maintained for at least 8 weeks prior to randomisation. Current use of herbal or dietary supplements containing known hepatotoxic ingredients is prohibited. 10. Severe renal dysfunction (estimated glomerular filtration rate < 30 mL/min/1.73 m2 or on chronic dialysis). 11. Participants with clinically significant bacterial, fungal, parasitic, or viral infection requiring systemic therapy. Participants with acute bacterial, fungal, parasitic, or viral infection requiring systemic therapy should delay screening/randomisation until active therapy has been completed. Note: Infection prophylaxis is allowed (see Section 6.8). 12. Known human immunodeficiency virus (HIV) positivity. 13. Active infection with hepatitis B virus (hepatitis B surface antigen [HBsAg] and hepatitis B core antibody [HBcAb] positive). 14. Active hepatitis C infection. 15. Documented phenylketonuria (PKU) or other clinically significant hyperphenylalaninemia, 21. Participants with iron deficiency (e.g., serum iron less than the lower limit of normal [LLN] or ferritin < 10 µg/L) at screening who are not taking or are unable to take iron supplements during the study. Participants with low serum iron levels due to chronic inflammation or other causes in which supplemental iron therapy is not indicated are allowed to be included in the study. 22. Participants with folate (or folic acid or Vitamin B9) or Vitamin B12 deficiency (e.g., folate or Vitamin B12 levels less than the LLN) at screening who are not taking or are unable to take supplements during the study. 23. Participants who are unable or unwilling to use antimalarial prophylaxis in the form of bed nets and/or effective chemoprophylaxis according to local recommendations if they live in areas of endemic malaria during participation in the study. 24. History of deep venous thrombosis requiring systemic anti-coagulation therapy for = 6 weeks, occurring within 6 months prior to screening. Participants on = 6 months of chronic or prophylactic anti-coagulation therapy are allowed. 25. Participants receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion). 26. Participants who have received an RBC transfusion for any reason 60 days prior to screening are eligible if HbA (adult Hb) is < 10% by Hb electrophoresis assessed at screening. In case of RBC transfusion during the
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Annualised rate of adjudicated VOC events with a medical contact | — |
Secondary
| Measure | Time frame |
|---|---|
| • Time to onset of first adjudicated VOC • Change in standardised T-score on the PROMIS Fatigue 10a scalea • Increase in Hb > 1 g/dL (yes/no) • Change in Hb • Change in lactate dehydrogenase • Change in absolute reticulocyte count • Change in indirect bilirubin • Number of AEs • Number of days hospitalised • Average length of stay of hospitalisations • Number of emergency room visits • Number of medical encounters • Days of missed school | — |
Countries
Kenya, Nigeria
Contacts
Public ContactMichelle Botha
Assoc Director Global Site Activation
Outcome results
None listed