Evaluating the safety and effectiveness of Xromi in young children with sickle cell disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational study assesses the safety and clinical effectiveness of Xromi® (hydroxycarbamide) in children aged 9 months to under 2 years with sickle cell disease. It includes a prospective cohort of patients treated with Xromi® and a matched retrospective cohort of untreated patients. Participants will be followed for 24 months to compare the incidence of adverse events and evaluate clinical outcomes. Data will be collected from routine clinical practice through chart reviews and follow-up visits.

Why it matters

Who can join: the full rules

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 it's running

• Universitätsklinikum Hamburg-Eppendorf
  Hamburg, Germany Recruiting
• Universitätsklinikum Heidelberg
  Heidelberg, Germany Recruiting
• 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

Study contacts

• Principal investigator: Sara Dr Trompeter, MD — University College London Hospitals
• Study coordinator: Hussain Mulla, PhD
• Email: hussain.mulla@novalabs.co.uk
• Phone: +44 (0)116 223 0100

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.