Status:
RECRUITING
PASS of Xromi Comparing Safety and Effectiveness in Children Under 2 Years With Sickle Cell Disease [PRECISE PASS]
Lead Sponsor:
Nova Laboratories Limited
Collaborating Sponsors:
OXON Epidemiology
Conditions:
Sickle Cell Disease
Eligibility:
All Genders
9-23 years
Brief 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 dise...
Detailed Description
The PRECISE study is a combined Post-Authorisation Safety Study (PASS) (Category 3) and a Post-Authorisation Efficacy Study that aims to provide data on the safety and effectiveness of hydroxycarbamid...
Eligibility Criteria
Inclusion
- 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.
Exclusion
Key Trial Info
Start Date :
June 9 2025
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
June 1 2029
Estimated Enrollment :
180 Patients enrolled
Trial Details
Trial ID
NCT06923111
Start Date
June 9 2025
End Date
June 1 2029
Last Update
August 6 2025
Active Locations (5)
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1
Basildon University Hospital
Basildon, Essex, United Kingdom, SS16 5NL
2
Noah's Ark Children's Hospital for Wales
Cardiff, Leicestershire, United Kingdom, CF14 4XW
3
University College London Hospital
London, North London, United Kingdom, NW1 2PG
4
The Royal London Hospital
London, Whitechapel, United Kingdom, E1 1FR