NCT06923111

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.

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 180 (estimated)
• Sponsor: Nova Laboratories Limited · Industry
• Sex: All
• Age: 9 Months – 23 Months
• Healthy volunteers: Not accepted

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.

Detailed description

The PRECISE study is a combined Post-Authorisation Safety Study (PASS) (Category 3) and a Post-Authorisation Efficacy Study that aims to provide data on the safety and effectiveness of hydroxycarbamide 100mg/ml oral solution (Xromi ®) administered prospectively to children under 2 years of age, over a follow-up period of 24 months compared to matched retrospective comparators who were treatment naïve. This is a non-interventional, matched cohort study involving children with SCD aged 9 to under 24 months. The study comprises two groups: * A prospective Xromi®-exposed cohort, enrolled at the time of treatment initiation and followed for 24 months. * A retrospective comparator cohort, matched 2:1 by site, age, and β-globin genotype, identified from clinical records of children not treated with hydroxycarbamide at the index date. The primary objective is to compare the incidence of adverse events of special interest (AESIs) between the two cohorts. Secondary analyses will assess the comparative effectiveness of Xromi® on clinical events, laboratory parameters, and physiological assessments. Exploratory analyses will examine treatment-related safety and effectiveness by dose, subgroups, and exposure to hydroxycarbamide during follow-up. Data will be sourced from routine clinical practice through chart reviews and follow-up visits. No study-specific interventions will be introduced. The study is planned across specialist sites in the UK and Germany, with potential expansion to other European countries if recruitment targets require.

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
DRUG	Xromi	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

Timeline

• Start date: 2025-06-09
• Primary completion: 2029-06-01
• Completion: 2029-06-01
• First posted: 2025-04-11
• Last updated: 2026-03-05

Locations

12 sites across 2 countries: Germany, United Kingdom

Regulatory

• FDA-regulated drug study