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RECRUITINGOBSERVATIONAL

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

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.

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

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.

Who May Be Eligible (Plain English)

Prospective Exposure Cohort - Who May Qualify: - 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 willing to sign a consent form to participate in the study, unless there is a waiver, non-opposition, or blanket written willing to sign a consent form by the parent for research studies. - Who Should NOT Join This Trial: - 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 weakened immune system 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 - Who May Qualify: - 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 willing to sign a consent form to participate in the study, unless there is a waiver, non-opposition, or blanket written willing to sign a consent form by the parent for research studies. - Who Should NOT Join This Trial: ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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.

Treatments Being Tested

DRUG

Xromi

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

Locations (12)

Universitätsklinikum Hamburg-Eppendorf
Hamburg, Germany
Universitätsklinikum Heidelberg
Heidelberg, Germany
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
Whittington Hospital
London, United Kingdom
Royal Manchester Children's Hospital
Manchester, United Kingdom
John Radcliffe Hospital
Oxford, United Kingdom