A comparative observational study to evaluate the safety and effectiveness of Xromi® (hydroxycarbamide oral solution 100mg/ml) for the prevention of vaso-occlusive complications of sickle cell disease in children under 2 years of age.

Research summary

We are conducting a study to gather more information on the safety and effectiveness of Xromi (hydroxyurea), a liquid medicine licensed to treat sickle cell disease (SCD) in children from 9 months of age. We aim to understand its safety profile and efficacy in preventing serious SCD complications (e.g., pain crises, acute chest syndromes, stroke) in children under 2 years when used in standard care. We will compare medical information collected during routine clinical appointments of children under 2 years who received Xromi to those who didn't receive any hydroxyurea therapy. This study won't involve additional tests, procedures, or medications beyond standard SCD care. Conducted at the European Medicines Agency's request by Nova Laboratories Limited, the study will be carried out by the children's normal SCD clinical care teams in hospitals/clinics. We aim to recruit 180 children from the UK and Germany, divided into two groups: 1. Xromi Group: Minimum 60 children currently starting or previously using Xromi while under 2 years. 2. Control Group: Minimum 120 children from historical data (up to 10 years past) who didn't receive hydroxyurea while under 2 years. Each Xromi group participant will be matched with two Control group participants from the same hospital (where possible), of the same age at visits, and with the same SCD genotype. We'll gather 24 months of patient data, starting from medication initiation for the Xromi group and a comparable historical date for the Control group. The study is expected to take approximately 4 years to recruit participants and collect data.

Principal Investigator

Dr Amrana Qureshi

Contact us

Email: rhiannon.collins@ouh.nhs.uk

IRAS number

334976