PASS of Xromi Comparing Safety and Effectiveness in Children Under 2 Years With Sickle Cell Disease [PRECISE PASS]
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.
Nova Laboratories Limited
180 participants
Jun 9, 2025
OBSERVATIONAL
Conditions
Summary
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.
Eligibility
Inclusion Criteria11
- Prospective Exposure Cohort
- 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.
- 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 Criteria15
- 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
- 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.
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Interventions
Xromi is indicated for the prevention of vaso-occlusive complications of Sickle Cell Disease in patients over 9 months of age as part of standard clinical practice
Locations(12)
View Full Details on ClinicalTrials.gov
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NCT06923111