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PASS of Xromi Comparing Safety and Effectiveness in Children Under 2 Years With Sickle Cell Disease [PRECISE PASS]

Sponsor: Nova Laboratories Limited

NCT ID: NCT06923111

View on ClinicalTrials.gov ↗

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.

Sickle Cell Disease

How this trial compares with your answers

Answer 2 more questions to improve match

What we know so far

Condition· Matched your search
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Still need:

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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
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.

Where Is This Study? (10 UK sites)

Basildon University Hospital

Basildon, United Kingdom

Recruiting

Noah's Ark Children's Hospital for Wales

Cardiff, United Kingdom

Recruiting

Evelina London Children's Hospital

London, United Kingdom

Recruiting

Kings College Hospital

London, United Kingdom

Recruiting
Hospital R&D contact (matched)

Jasmine Palmer

kch-tr.research@nhs.net0203 299 1980

North Middlesex University Hospital

London, United Kingdom

Recruiting
Hospital R&D contact (matched)

Dr Deborah McCartney

deborah.mccartney2@nhs.net+44(0)208 887 2307

The Royal London Hospital

London, United Kingdom

Recruiting
Hospital R&D contact (matched)

Natasha Ajraam

rf-tr.randd@nhs.net020 375 82150

University College London Hospital

London, United Kingdom

Recruiting
Hospital R&D contact (matched)

Rajinder Sidhu - Associate Director, Research Governance and Operations

uclh.jro-communications@nhs.net020 3447 9825

Whittington Hospital

London, United Kingdom

Recruiting
Hospital R&D contact (matched)

Research Support Team

whh-tr.researchsupport@nhs.net0207 288 3585

Royal Manchester Children's Hospital

Manchester, United Kingdom

Recruiting
Hospital R&D contact (matched)

Elizabeth Mainwaring

R&D.applications@mft.nhs.uk0161 276 3340

John Radcliffe Hospital

Oxford, United Kingdom

Recruiting

How to Get in Touch

Hussain Mulla, PhD

Sponsor contact

CONTACT

+44 (0)116 223 0100 hussain.mulla@novalabs.co.uk

Sarah Edwards, PhD

Sponsor contact

CONTACT

+44 (0)116 223 0100 sarah.edwards@novalabs.co.uk
Data sourced from ClinicalTrials.gov · Last verified: 2026-03