At a glance
- What the study gets you
- Health checks and monitoring — no treatment given
- Type of study
- Observational (no treatment given)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Xromi (drug)
- How long the study runs
- Study runs about 48 months (dates as stated)
- About the drug or intervention
- Xromi — drug: 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
- Patient visit burden
- Not specified by the sponsor
In plain English
This study, called PRECISE PASS, looks at how safe and effective Xromi (a liquid form of hydroxycarbamide) is in babies aged 9 months to under 2 years who have sickle cell disease. It compares children prescribed Xromi with similar children who have not used hydroxycarbamide. It is sponsored by Nova Laboratories Limited.
Who can take part
- Aged from 9 months to under 2 years
- Diagnosed with sickle cell disease
- Has a known beta-globin genotype (a gene test result)
- For the Xromi group: prescribed Xromi to prevent complications of sickle cell disease
- For the comparison group: matched to a child taking Xromi and has not used hydroxycarbamide
- Parent(s) or legal representative gives written informed consent (or a waiver, non-opposition, or blanket consent applies)
Who may not be able to
- Has used hydroxycarbamide in any form before the study (comparison group: before or at the start)
- Receives regular blood transfusions (every 8 weeks or more often)
- Known severe allergy to any ingredients of Xromi (Xromi group only)
- Severe liver problems (Child-Pugh class C) or severe kidney problems (creatinine clearance under 30 ml/min)
- Low blood counts: neutrophils under 1.0 x 10^9/L, reticulocytes under 80 x 10^9/L, or platelets under 80 x 10^9/L
- Taking part in another clinical study of an investigational medicine
- Taking anti-retroviral medicines for HIV
- Has an active cancer
- Xromi group only: prescribed Xromi but does not start treatment
What taking part involves
- • The Xromi group takes Xromi (a liquid hydroxycarbamide medicine) as prescribed by their doctor
- • The comparison group has not used hydroxycarbamide and is matched to a child in the Xromi group
- • The study collects information on safety and effectiveness
Time commitment: Not stated — ask the trial team about study visits, how long it lasts, and what taking part involves.
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Observing health over time
- Ages
- 9 Months to 23 Months
- Who
- All
- Number of participants
- 180
- Started
- 2025-06-09
- Last checked
- 2026-03
Plain English Summary
What is this study?
- • Testing a new treatment for sickle cell disease
- • Clinical study - 180 participants
- • 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)
Who can take part?
- • Ages 9 Months to 23 Months
- • Diagnosed with sickle cell disease
Where?
- • Basildon - Basildon University Hospital
- • Cardiff - Noah's Ark Children's Hospital for Wales
- • London - Evelina London Children's Hospital
- • London - Kings College Hospital
- • +6 more UK sites
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
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.
More detail
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.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 9 Months - 23 Months
- Who can join: All genders
What the study is looking for
- ✓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.
Who cannot take part
- ✗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.
- ✗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.
See the full criteria
Where Is This Study? (10 UK sites)
Basildon University Hospital
Basildon, United Kingdom
Noah's Ark Children's Hospital for Wales
Cardiff, United Kingdom
Evelina London Children's Hospital
London, United Kingdom
Kings College Hospital
London, United Kingdom
North Middlesex University Hospital
London, United Kingdom
The Royal London Hospital
London, United Kingdom
University College London Hospital
London, United Kingdom
Rajinder Sidhu - Associate Director, Research Governance and Operations
uclh.jro-communications@nhs.net020 3447 9825Whittington Hospital
London, United Kingdom
Royal Manchester Children's Hospital
Manchester, United Kingdom
John Radcliffe Hospital
Oxford, United Kingdom
How to Get in Touch
Hussain Mulla, PhD
Sponsor contactCONTACT
Sarah Edwards, PhD
Sponsor contactCONTACT
