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New hope for toddlers with sickle cell: drug may stop painful attacks

NCT ID NCT06923111

Prevention Sponsor: Nova Laboratories Limited Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study looks at whether a liquid medicine called Xromi (hydroxycarbamide) can safely prevent painful complications of sickle cell disease in children aged 9 months to 2 years. About 180 children will take part, with some receiving Xromi and a comparison group of similar children who did not get the drug. Researchers will track side effects and how well the medicine works over 2 years.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

About 180 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2025

Expected to finish

Jun 2029

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

The study population will consist of children with SCD who meet all the inclusion criteria and none of the exclusion criteria. Identification of participants will be conducted as follows: * Prospective exposure cohort: Potential participants will be identified as they attend the participating sites where eligibility criteria will be assessed prior to initiation with Xromi. * Retrospective comparator cohort: Potential participants will be selected from clinical chart review for hydroxycarbamide naive children that could be matched to prospective participants.

Ages

9 to 23 months

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

inherited hemoglobinopathy sickle cell disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Sickle cell disease

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    12 sites in 2 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Basildon University Hospital

    RECRUITING

    Basildon, United Kingdom

  • Evelina London Children's Hospital

    RECRUITING

    London, United Kingdom

  • John Radcliffe Hospital

    RECRUITING

    Oxford, United Kingdom

  • Kings College Hospital

    RECRUITING

    London, United Kingdom

  • Noah's Ark Children's Hospital for Wales

    RECRUITING

    Cardiff, United Kingdom

  • North Middlesex University Hospital

    RECRUITING

    London, United Kingdom

  • Royal Manchester Children's Hospital

    RECRUITING

    Manchester, United Kingdom

  • The Royal London Hospital

    RECRUITING

    London, United Kingdom

  • University College London Hospital

    RECRUITING

    London, United Kingdom

  • Universitätsklinikum Hamburg-Eppendorf

    RECRUITING

    Hamburg, Germany

  • Universitätsklinikum Heidelberg

    RECRUITING

    Heidelberg, Germany

  • Whittington Hospital

    RECRUITING

    London, United Kingdom

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