Assessment of Neurofilaments Levels in Premature Newborns: Study of Their Early Development

Not yet recruiting

• Conditions: Healthy Participants

• Registration Number: NCT07159555
• Lead Sponsor: Centre Hospitalier Universitaire de Liege

Brief Summary

This is a prospective observational study aiming to establish reference values of neurofilaments in late preterm newborns (34-37 weeks of gestation) and to evaluate their early postnatal evolution.

Neurofilaments are neuronal proteins released into the blood when nerve cells are damaged. They have emerged as promising biomarkers in neurodegenerative diseases, particularly in spinal muscular atrophy (SMA), where higher levels are associated with disease activity. However, very limited data exist on the normal dynamics of neurofilament levels in preterm infants.

In this study, up to 15 preterm newborns hospitalized in the neonatal unit will be enrolled (5 at 34 weeks, 5 at 35 weeks, 5 at 36 weeks of gestation). A maximum of 1 mL of additional blood will be collected only during routine clinically indicated blood draws, at birth, on day 2, and weekly until 37 weeks of gestation. Samples will be analyzed for neurofilament concentrations and compared across gestational ages, over time, and with existing data from term newborns and SMA patients.

The expected outcome is to generate normative data on neurofilament levels in late preterm infants, which will help interpret biomarker values in future studies and optimize early diagnosis and treatment strategies for neurodegenerative diseases such as SMA.

Detailed Description

Recent therapeutic advances in spinal muscular atrophy (SMA) have highlighted the importance of very early treatment, ideally before the onset of symptoms. Neurofilaments, structural proteins of neurons released into the blood when axons are damaged, have been identified as sensitive biomarkers of neurodegeneration and disease activity in SMA. Elevated neurofilament levels have been reported in affected infants, with a rapid decline following initiation of effective treatment.

Despite their growing importance as a biomarker, little is known about the normal postnatal dynamics of neurofilaments in preterm infants. Establishing normative data in this population is essential to correctly interpret biomarker levels in newborn screening programs and clinical follow-up, particularly for conditions such as SMA where treatment decisions rely on early biological signals.

Study Timeline

• Study First Submitted: 2025-08-28
• Status Verified: 2025-09
• Study Start: 2025-09-01
• Study First Submitted QC: 2025-09-05
• Last Update Submitted: 2025-09-05
• Study First Posted: 2025-09-08
• Last Update Posted: 2025-09-08
• Primary Completion: 2026-08-31
• Study Completion: 2026-12-31

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 15

Inclusion Criteria

• Gestational age between 34 and 37 weeks of amenorrhea.
• Blood test scheduled for clinical reasons.
• Signed parental consent.

Exclusion Criteria

• Known neurological disorders.
• Severe congenital malformations.
• Medical contraindication to blood sampling.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Neurofilament concentration in blood of late preterm infants	From birth to 37 weeks of gestational age (up to 3 weeks after inclusion depending on gestational age at birth).	Measurement of blood neurofilament light chain (NfL) concentrations to establish reference values and describe their early postnatal evolution in late preterm infants.

Secondary Outcome Measures

Name	Time	Method
Postnatal evolution of neurofilament concentrations	From birth up to 37 weeks of gestational age (longitudinal follow-up).	Analysis of changes in neurofilament levels over time in late preterm infants, from birth to 37 weeks of gestational age.