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. [PRECISE PASS]

Study identification

• EU PAS number:
  EUPAS1000000076
• Study ID:
  1000000076
• Official title and acronym:
  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. [PRECISE PASS]
• DARWIN EU® study:
  No
• Study countries:
  Germany

  United Kingdom
• Study description:
  This is a non-randomised, non-interventional, comparative, observational, hybrid prospective and retrospective, post-authorisation cohort study (a matched cohort design) of children with sickle cell disease (SCD) from 9 months to under 2 years of age to assess the safety and effectiveness of Xromi® (100mg/ml hydroxycarbamide) based on primary and secondary data collection.; ; This study aims to compare the safety and effectiveness of Xromi® (100mg/ml hydroxycarbamide) in children under 2 years old with SCD to a group not receiving the treatment. The study will collect data both prospectively and retrospectively and will be mainly exploratory without testing any previous hypotheses.; ; The primary goal is to compare the occurrence of specific adverse events (AESIs) between a prospectively recruited Xromi®-treatment group and a retrospective Hydroxyurea naïve (untreated) group. The study will recruit at least 60 children for the Xromi® group (Prospective Exposure Cohort) and at least 120 children for the untreated group (Retrospective Comparator Cohort) from sites in the UK and Germany.; ; The study will begin on the date of the first site given initiation greenlight and will end up to 4 years (48 months) later. Screening for the Retrospective Comparator Cohort will go back up to 10 years before Xromi® was introduced at each site following initial licensing. Recruitment for the Prospective Exposure Cohort will last 2 years (24 months), and all participants will be followed for 2 years (24 months).; ; The overall study period spans from 10 years before the study start to 4 years after the study start. The study end date will ensure that all participants in the Exposure and Comparator Cohort have completed 24-month follow-up.
• Study status:
  Planned

Research institutions and networks

Institutions

Nova Laboratories Ltd.

United Kingdom

OXON Epidemiology

Spain

United Kingdom

Study timelines

Date when funding contract was signed

Actual: 09/01/2024

Study start date

Planned: 29/04/2025

Data analysis start date

Planned: 28/02/2029

Date of interim report, if expected

Planned: 04/05/2026

Date of final study report

Planned: 03/09/2029

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Nova Laboratories Ltd. will provide funding for the duration of the study.

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  EU RMP category 3 (required)

Other study registration identification numbers and links

IRAS Number (UK): 334976