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.
Status: Recruiting
Location: See all (5) locations...
Intervention Type: Drug
Study Type: Observational
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
Participation Requirements
Sex: All
Minimum Age: 9 months
Maximum Age: 1
Healthy Volunteers: f
View:
⁃ 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.
Locations
Other Locations
United Kingdom
Basildon University Hospital
NOT_YET_RECRUITING
Basildon
Noah's Ark Children's Hospital for Wales
NOT_YET_RECRUITING
Cardiff
Kings College Hospital
RECRUITING
London
The Royal London Hospital
NOT_YET_RECRUITING
London
University College London Hospital
RECRUITING
London
Contact Information
Primary
Hussain Mulla, PhD
hussain.mulla@novalabs.co.uk
+44 (0)116 223 0100
Backup
Sarah Edwards, PhD
sarah.edwards@novalabs.co.uk
+44 (0)116 223 0100
Time Frame
Start Date:2025-06-09
Estimated Completion Date:2029-06
Participants
Target number of participants:180
Treatments
Prospective Exposure Cohort
Children with SCD aged 9 months to under 2 years of age who are newly prescribed Xromi®, will be identified prospectively. These participants will be followed up for 24 months from their index date, regardless of whether they continue treatment with Xromi®, discontinue all hydroxycarbamide treatment, or switch to another formulation of hydroxycarbamide. The decision to prescribe Xromi® will be made solely by the physician independently of the study, as part of standard care.~\-
Retrospective Comparator Cohort
Children with SCD and naïve to any hydroxycarbamide formulation at the index date. These participants will be identified retrospectively using the data from the last 10 years up to the date Xromi® was first used in children from 9 months to under 2 years of age at each individual site. The 24-month follow-up will be retrospective from the date they are matched to the exposed participant, irrespective of whether they start on any formulation of hydroxycarbamide during the follow-up.