Studies Xromi in young children with sickle cell disease
Tracks safety and effect of Xromi in children with sickle cell disease.
Plain-language title generated from the registry record
Registry title: PASS of Xromi Comparing Safety and Effectiveness in Children Under 2 Years With Sickle Cell Disease [PRECISE PASS]
Key facts
- Where
- Basildon, Cardiff, London, Manchester, and Oxford, United KingdomSee the 10 sites
Where in United Kingdom
- Basildon University HospitalBasildon, United KingdomTaking people
- Noah's Ark Children's Hospital for WalesCardiff, United KingdomTaking people
- Evelina London Children's HospitalLondon, United KingdomTaking people
- Kings College HospitalLondon, United KingdomTaking people
- North Middlesex University HospitalLondon, United KingdomTaking people
- The Royal London HospitalLondon, United KingdomTaking people
- University College London HospitalLondon, United KingdomTaking people
- Whittington HospitalLondon, United KingdomTaking people
- Royal Manchester Children's HospitalManchester, United KingdomTaking people
- John Radcliffe HospitalOxford, United KingdomTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Nova Laboratories Limited
- Trial code
- NCT06923111
- Record updated
- 5 March 2026
- Original description
This post-authorisation safety and efficacy study (PRECISE PASS) evaluates the use of Xromi® (hydroxycarbamide 100 mg/mL oral solution) in children aged 9 months to under 2 years with sickle cell disease (SCD).Read full original descriptionShow less
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.
This post-authorisation safety and efficacy study (PRECISE PASS) evaluates the use of Xromi® (hydroxycarbamide 100 mg/mL oral solution) in children aged 9 months to under 2 years with sickle cell disease (SCD).
The objective is to assess the safety profile and clinical effectiveness of Xromi® under routine clinical conditions. The study includes a prospective cohort of Xromi®-treated patients and a matched retrospective comparator cohort of untreated patients. Participants will be followed for 24 months from treatment initiation or matched index date.
Prospective Exposure Cohort; Inclusion criteria:; Aged from 9 months to under 2 years at the index date; Diagnosis of SCD; Known β-globin genotype at the index date; Prescribed Xromi® for the prevention of complications of SCD; Parent(s) (or a legal representative(s)) provides written informed consent to participate in the study, unless there is a waiver, non-opposition, or blanket written informed consent by the parent for research studies; Exclusion criteria:; Previous use of hydroxycarbamide of any formulation before the index date; Receiving regular blood transfusions (occurring every 8 weeks or more frequently) at the index date; Known hypersensitivity to any of the excipients of Xromi® at the index date; Contraindications to the drug at the index date: severe hepatic impairment (Child-Pugh classification C); severe renal impairment (creatinine clearance: CrCl <30 ml/min); presence of at least one of the following: Absolute neutrophil count (ANC) < 1.0 x 10\^9/L, absolute reticulocyte count (ARC) <80 x 10\^9/L, platelets <80 x 10\^9/L; Participating in another clinical study of an investigational medicinal product (IMP) at the index date; Anti-retroviral medicinal products for human immunodeficiency virus (HIV) at the index date; Active malignancy at the index date; Participants in the prospective exposure cohort who are prescribed Xromi® but do not initiate treatment will be excluded from the dataset; Retrospective Comparator cohort; Inclusion criteria:; Aged from 9 months to under 2 years at the index date; Diagnosis of SCD; Known β-globin genotype; Matched to an exposed participant; Parent(s) (or a legal representative(s)) provides written informed consent to participate in the study, unless there is a waiver, non-opposition, or blanket written informed consent by the parent for research studies; Exclusion criteria:; Use of hydroxycarbamide of any formulation before or at the index date; Receiving regular blood transfusions (occurring every 8 weeks or more frequently) at the index date; Presence at the index date of any of the following: severe hepatic impairment (Child-Pugh classification C); severe renal impairment (CrCl <30 ml/min); presence of at least one of the following: ANC < 1.0 x 10\^9/L, ARC < 80 x 10\^9/L, platelets < 80 x 10\^9/L); Participating in another clinical study of an IMP at the index date; Anti-retroviral medicinal products for HIV at the index date; Active malignancy at the index date.