PASS of Xromi Comparing Safety and Effectiveness in Children Under 2 Years With Sickle Cell Disease [PRECISE PASS]
NCT ID: NCT06923111
Last Updated: 2025-08-06
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
180 participants
OBSERVATIONAL
2025-06-09
2029-06-30
Brief Summary
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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.
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Detailed Description
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This is a non-interventional, matched cohort study involving children with SCD aged 9 to under 24 months. The study comprises two groups:
* A prospective Xromi®-exposed cohort, enrolled at the time of treatment initiation and followed for 24 months.
* A retrospective comparator cohort, matched 2:1 by site, age, and β-globin genotype, identified from clinical records of children not treated with hydroxycarbamide at the index date.
The primary objective is to compare the incidence of adverse events of special interest (AESIs) between the two cohorts. Secondary analyses will assess the comparative effectiveness of Xromi® on clinical events, laboratory parameters, and physiological assessments. Exploratory analyses will examine treatment-related safety and effectiveness by dose, subgroups, and exposure to hydroxycarbamide during follow-up.
Data will be sourced from routine clinical practice through chart reviews and follow-up visits. No study-specific interventions will be introduced. The study is planned across specialist sites in the UK and Germany, with potential expansion to other European countries if recruitment targets require.
Conditions
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Study Design
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COHORT
OTHER
Study Groups
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Prospective Exposure Cohort
Children with SCD aged 9 months to under 2 years of age who are newly prescribed Xromi®, will be identified prospectively. These participants will be followed up for 24 months from their index date, regardless of whether they continue treatment with Xromi®, discontinue all hydroxycarbamide treatment, or switch to another formulation of hydroxycarbamide. The decision to prescribe Xromi® will be made solely by the physician independently of the study, as part of standard care.
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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
Retrospective Comparator Cohort
Children with SCD and naïve to any hydroxycarbamide formulation at the index date. These participants will be identified retrospectively using the data from the last 10 years up to the date Xromi® was first used in children from 9 months to under 2 years of age at each individual site. The 24-month follow-up will be retrospective from the date they are matched to the exposed participant, irrespective of whether they start on any formulation of hydroxycarbamide during the follow-up.
No interventions assigned to this group
Interventions
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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
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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.
* 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
* 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
* 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.
9 Months
23 Months
ALL
No
Sponsors
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OXON Epidemiology
INDUSTRY
Nova Laboratories Limited
INDUSTRY
Responsible Party
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Principal Investigators
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Hussain Dr Mulla, PhD
Role: STUDY_DIRECTOR
Nova Laboratories Ltd.
Sara Dr Trompeter, MD
Role: PRINCIPAL_INVESTIGATOR
University College London Hospitals
Locations
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Basildon University Hospital
Basildon, Essex, United Kingdom
Noah's Ark Children's Hospital for Wales
Cardiff, Leicestershire, United Kingdom
University College London Hospital
London, North London, United Kingdom
The Royal London Hospital
London, Whitechapel, United Kingdom
Kings College Hospital
London, , United Kingdom
Countries
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Central Contacts
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Facility Contacts
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Sarah Edwards, PhD
Role: primary
Sarah Edwards, PhD
Role: primary
Sarah Edwards, PhD
Role: primary
Sarah Edwards, PhD
Role: primary
Sarah Edwards, PhD
Role: primary
References
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Rankine-Mullings A, Keenan R, Chakravorty S, Inusa B, Telfer P, Velangi M, Ware RE, Moss JJ, Lloyd AL, Edwards S, Mulla H. Efficacy, safety, and pharmacokinetics of a new, ready-to-use, liquid hydroxyurea in children with sickle cell anemia. Blood Adv. 2023 Aug 22;7(16):4319-4322. doi: 10.1182/bloodadvances.2023010099. No abstract available.
Thompson BW, Miller ST, Rogers ZR, Rees RC, Ware RE, Waclawiw MA, Iyer RV, Casella JF, Luchtman-Jones L, Rana S, Thornburg CD, Kalpatthi RV, Barredo JC, Brown RC, Sarnaik S, Howard TH, Luck L, Wang WC. The pediatric hydroxyurea phase III clinical trial (BABY HUG): challenges of study design. Pediatr Blood Cancer. 2010 Feb;54(2):250-5. doi: 10.1002/pbc.22269.
Other Identifiers
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EUPAS1000000076
Identifier Type: OTHER
Identifier Source: secondary_id
CHIL 62837
Identifier Type: OTHER
Identifier Source: secondary_id
334976
Identifier Type: OTHER
Identifier Source: secondary_id
NOVDD-001
Identifier Type: -
Identifier Source: org_study_id
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