Clinical Trials in the UK / NCT06923111
Recruiting
Observational
PASS of Xromi Comparing Safety and Effectiveness in Children Under 2 Years With Sickle Cell Disease [PRECISE PASS]
NCT06923111 · tracked via the Priya Life Science UK tracker
Condition(s) studied
Sickle Cell Disease
Investigational drug(s) / intervention(s)
Xromi
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
Study summary
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.
Eligibility
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.
Primary outcome measure(s)
- AESI - Myelosuppression (Neutropenia) — Pre-baseline, Baseline to 24 months
Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort. Myelosuppression protocol definition of neutropenia is ANC \<1.0 x 10\^9/L - AESI - Myelosuppression (Reticulocytopenia) — Pre-baseline, Baseline to 24 months
Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort. Myelosuppression protocol definition of reticulocytopenia is ARC \<80 x 10\^9/L, unless Hb \>90 g/L, - AESI - Myelosuppression (Thrombocytopenia) — Pre-baseline, Baseline to 24 months
Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort. Myelosuppression protocol definition of thrombocytopenia defined as platelets \<80 x 10\^9/L. - AESI - Myelosuppression (Anaemia) — Pre-baseline, Baseline to 24 months
Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort. Myelosuppression protocol definition of anaemia defined as Hb \<45 g/L. - AESI - Abnormal Weight Gain — Baseline to 24 months
Weight (kg) Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort and standard care guidelines - AESI - Abnormal Weight Loss — Baseline to 24 months
Weight (kg) Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort and standard care guidelines - AESI - Increase in Hepatic Enzyme (ALT) — Baseline to 24 months
Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort and normal laboratory ranges. Defined as an increase in hepatic enzyme Alanine transaminase (ALT) increased (U/L) - AESI - Increase in Hepatic Enzyme (AST) — Baseline to 24 months
Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort and normal laboratory ranges. Defined as an increase in hepatic enzyme Aspartate transaminase (AST) increased (U/L) - AESI - Alopecia — Pre-baseline, Baseline to 24 months
Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort. Defined in study protocol as presence of alopecia (a skin and subcutaneous tissue disorder). - AESI - Other Hair Loss — Pre-baseline, Baseline to 24 months
Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort. Defined in study protocol as presence of other hair loss (a skin and subcutaneous tissue disorder). - AESI - Skin Hyperpigmentation — Pre-baseline, Baseline to 24 months
Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort. Defined in study protocol as presence of skin hyperpigmentation, including oral and nail hyperpigmentation.(a skin and subcutaneous tissue disorder). - AESI - Rash — Pre-baseline, Baseline to 24 months
Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort. Defined in study protocol as presence of a rash (a skin and subcutaneous tissue disorder). - AESI - Skin Ulcers — Pre-baseline, Baseline to 24 months
Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort. Defined in study protocol as presence of skin ulcer, including leg ulcer (a skin and subcutaneous tissue disorder). - AESI - Growth Retardation — Pre-baseline, Baseline to 24 months
height/length (cm) Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort and standard care guidelines (as a drop of 2 or more centiles on growth charts over time). - AESI - Bacterial Infection — Pre-baseline, Baseline to 24 months
Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort. Defined in the protocol as a proven or treated bacterial infection. - AESI - Viral Infection — Pre-baseline, Baseline to 24 months
Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort. Defined in the protocol as a proven or treated viral infection. - AESI - Fungal Infection — Pre-baseline, Baseline to 24 months
Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort. Defined in the protocol as a proven or treated fungal infection.
Trial sites (12)
| Facility | City | Region | Status |
|---|---|---|---|
| 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 |
Other trials for the same condition
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT06923111 on ClinicalTrials.gov ↗ ← All trials in the UK