An Observational Study to Collect Data on How Aflibercept (Eylea) Given Using a Paediatric Dosing Device is Used in Preterm Babies With Retinopathy of Prematurity in the United Kingdom (UK)

Drug Utilization Study for Eylea 40 mg/mL Using the PICLEO Paediatric Dosing Device in Preterm Infants With Retinopathy of Prematurity in the UK

This is an observational study in which only data from babies with retinopathy of prematurity (ROP) who are being treated with aflibercept (Eylea) in prefilled syringe (PFS) using a paediatric dosing device (PDD) are collected and studied.

ROP is a condition that affects the eyes of preterm babies. It occurs when the baby's retina, the part of the eye that senses light, does not develop normally. This may result in vision problems, including blindness, if left untreated. Preterm babies are born before 37 weeks of pregnancy. ROP is more likely to develop in babies who are born before 32 weeks of pregnancy or weigh less than 1.5 kilograms at birth.

Aflibercept is a drug that is injected into the eye. It works by blocking a protein called vascular endothelial growth factor (VEGF) which causes abnormal growth of blood vessels in the retina.

Aflibercept in PFS given using a PDD is approved for the treatment of babies with ROP. The prefilled syringe will be fitted with an injection needle to give aflibercept. And a PDD is a tool used to give the right amount of aflibercept to children in a safe manner.

Since there are other treatments which are commonly used for babies with ROP, the extent of use of aflibercept given using a PDD is unknown.

The main purpose of this study is to:

• find the number of preterm babies who are treated with aflibercept using a PDD in the UK
• inform whether this number is enough to perform a study to learn about the long-term safety of aflibercept given using a PDD in babies with ROP

An additional purpose of this study is to describe characteristics including age, sex, and race, and signs and symptoms of ROP observed in babies being treated with aflibercept using a PDD.

The data will come from a database called the National Neonatal Research Database. The study will cover the period from March 2024 to March 2025, if the number of babies found is enough to perform the safety study. If not, data will be collected till April 2027.

In this study only available data from preterm babies born during the study period are collected. No visits or tests are required as part of this study.

Study Overview

• Status: Recruiting
• Conditions: Preterm Infants; Retinopathy of Prematurity
• Intervention / Treatment: Drug: Aflibercept (Eylea)

• Study Type: Observational
• Enrollment (Estimated): 200

Contacts and Locations

• Study Contact: Name: Bayer Clinical Trials Contact; Phone Number: (+)1-888-84 22937; Email: clinical-trials-contact@bayer.com

Study Locations

• United Kingdom
  • Multiple Locations, United Kingdom
    • Recruiting
    • Many Locations

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

All premature infants diagnosed with ROP and treated with Eylea PFS + PDD included in the National Neonatal Research Database (NNRD) during the study period in the UK.

Description

Inclusion Criteria:

• Eligible infants within the NNRD include those who were:

  • 1. Born during the study period, i.e. from Q4/2023 following market introduction of Eylea PFS+PDD and 31st December 2026, and
  • 2. Received care in a neonatal unit that contributes data to the NNRD and the unit has agreed to participate in the study, and
  • 3. Diagnosed with ROP in any stage in at least one eye.

Exclusion Criteria:

• Infants with missing data for gestational age at birth will be excluded.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Premature infants diagnosed with ROP; Premature infants diagnosed with retinopathy of prematurity (ROP) and treated with aflibercept 0.4 mg using the Eylea 40 mg/mL prefilled syringe (PFS) in combination with the PICLEO paediatric dosing device (PDD) after marketing authorisation in UK and included in the National Neonatal Research Database (NNRD).	Drug: Aflibercept (Eylea); Eylea 40 mg/mL solution for injection in pre-filled syringe.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants treated with Eylea PFS+PDD upon completion of 1- or 3-years following market introduction	To evaluate trends in drug utilization patterns of Eylea prefilled syringe (PFS)+Paediatric Dosing Device (PDD) in the ROP patient population. Exposure to Eylea is defined as a record of Eylea PFS+PDD in the National Neonatal Research Database (NNRD) database during admission to the neonatal unity during the study period.; To inform the decision on whether the number of preterm infants with ROP exposed to Eylea PFS+PDD is sufficient (n=200) for proceeding with a cohort study to collect data on long-term safety (ocular and systemic safety including neurodevelopmental outcome).	From market introduction of Eylea PFS+PDD in UK up to 3 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Summary of patient characteristics reported as number of participants with different categories	Birth weight (in categories), sex, gestational age in weeks (in categories), ethnicity, etc.	From market introduction of Eylea PFS+PDD in UK up to 3 years
Summary of maternal factors reported as number of participants with different categories	Problems during pregnancy with mother, problems during pregnancy, mode of delivery, meconium stained liquor at delivery, etc.	From market introduction of Eylea PFS+PDD in UK up to 3 years
Summary of comorbidities reported as number of participants with different categories	Brain injury, necrotising enterocholitis, pulmonary hemorrhage, chronic lung disease, etc.	From market introduction of Eylea PFS+PDD in UK up to 3 years
Summary of clinical condition and resource utilization reported as number of participants with different categories	Respiratory support (mode of ventilation), cardiovascular (inotropic support), feeding (parenteral and enteral nutrition)	From market introduction of Eylea PFS+PDD in UK up to 3 years
Summary of clinical condition and resource utilization reported as days	Intensive care days, invasive ventilation days, parenteral nutrition days	From market introduction of Eylea PFS+PDD in UK up to 3 years
Summary of outcomes of the premature infants reported as number of participants with different categories	Survival to discharge from neonatal care (Y/N); Discharge, death, transferred to another hospital (in categories)	From market introduction of Eylea PFS+PDD in UK up to 3 years
Summary of ocular disease and treatment reported as number of participants with different categories	Number of Eylea injections in the neonatal unity, ROP surgery, ROP stage, ROP clock hours, ROP Max zone, etc.	From market introduction of Eylea PFS+PDD in UK up to 3 years

Collaborators and Investigators

• Sponsor: Bayer

Publications and helpful links

Helpful Links

• Click here for access to information about Bayer's transparency standards and Bayer studies

Study record dates

Study Major Dates

• Study Start (Actual): March 5, 2024
• Primary Completion (Estimated): March 31, 2027
• Study Completion (Estimated): April 30, 2027

Study Registration Dates

• First Submitted: February 26, 2024
• First Submitted That Met QC Criteria: March 11, 2024
• First Posted (Actual): March 18, 2024

Study Record Updates

• Last Update Posted (Actual): April 12, 2024
• Last Update Submitted That Met QC Criteria: April 11, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Keywords: ROP
• Additional Relevant MeSH Terms: Eye Diseases; Infant, Newborn, Diseases; Pregnancy Complications; Obstetric Labor Complications; Obstetric Labor, Premature; Infant, Premature, Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Retinal Diseases; Premature Birth; Retinopathy of Prematurity; Physiological Effects of Drugs; Antineoplastic Agents; Angiogenesis Inhibitors; Angiogenesis Modulating Agents; Growth Substances; Growth Inhibitors; Aflibercept
• Other Study ID Numbers: 22539

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014. Interested researchers can use www.vivli.org to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the member section of the portal.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No