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

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About this study

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.

Condition
Sickle Cell Disease
Tested
Xromi
Sponsor
Nova Laboratories Limited

Who can join

Age
0.75 to 1.92 years
Sex
All sexes
Healthy volunteers
Not accepted

Inclusion 11

  • Minimum age: 0.75 years
  • Maximum age: 1.92 years
  • Study condition: Sickle Cell Disease
  • Prospective Exposure Cohort
  • 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.
  • Known β-globin genotype.
  • Matched to an exposed participant.

Exclusion 13

  • 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
  • Use of hydroxycarbamide of any formulation before or 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.

Where

12 sites, 12 recruiting

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

and 6 more sites on ClinicalTrials.gov

Contact

Potential match only. Final eligibility is determined by the study team.