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

Official title

Neurofilaments Levels in Premature Newborns

Overview

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.

Interventions

• Other Blood sampling for neurofilament analysis
  An additional blood sample (maximum 1 mL) is collected during routine clinically indicated blood draws in late preterm infants (34-37 weeks of gestation). Samples are obtained at birth, day 2, and weekly until 37 weeks of gestation if blood draws are already planned. No separate venipuncture is performed. Samples are processed and stored for subsequent neurofilament concentration analysis.

Primary outcome measures

• Neurofilament concentration in blood of late preterm infants [Time frame: From birth to 37 weeks of gestational age (up to 3 weeks after inclusion depending on gestational age at birth).]

Secondary outcome measures (1)

• Postnatal evolution of neurofilament concentrations [Time frame: From birth up to 37 weeks of gestational age (longitudinal follow-up).]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: Yes

Study design

Observational model

Cohort

Study locations

Belgium · 1 center

• Hopital Citadelle — Liège

Identifiers

NCT: NCT07159555 · Neurofilaments

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗