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

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.

This study is testing if the medication Xromi is safe and effective for young children with sickle cell disease.

Quick facts

• Study type: Observational
• Enrollment: 180 (estimated)
• Ages: 9 Months to 23 Months
• Sex: All
• Sponsor: Nova Laboratories Limited (industry)
• Locations: 12 sites (Hamburg and 11 other locations)
• Trial ID: NCT06923111 on ClinicalTrials.gov

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.

Who should consider this trial

Good fit: Ideal candidates are children aged 9 months to under 2 years diagnosed with sickle cell disease who are prescribed Xromi®.

Not a fit: Patients who have previously used hydroxycarbamide or are receiving regular blood transfusions may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide valuable insights into the safety and effectiveness of Xromi® for young children with sickle cell disease.

How similar studies have performed: Other studies have shown success with similar approaches in evaluating treatments for sickle cell disease, but this specific formulation and age group may be novel.

Eligibility 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 this trial is running

Hamburg and 11 other locations

• 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)
• North Middlesex University Hospital — London, United Kingdom (RECRUITING)
• The Royal London Hospital — London, United Kingdom (RECRUITING)
• University College London Hospital — London, United Kingdom (RECRUITING)
• Whittington Hospital — London, United Kingdom (RECRUITING)
• Royal Manchester Children's Hospital — Manchester, United Kingdom (RECRUITING)
• John Radcliffe Hospital — Oxford, United Kingdom (RECRUITING)

Study contacts

• Principal investigator: Sara Dr Trompeter, MD — University College London Hospitals
• Study coordinator: Hussain Mulla, PhD

Conditions: Sickle Cell Disease, Hydroxyurea, liquid formulation, HbS Disease, Hemoglobin S Disease, Sickle Cell Anemia, Sickle Cell Disorders, Sickling Disorder Due to Hemoglobin S