Healthy Participants
Conditions
Keywords
Healthy Participants, Neurofilament, Premature infant
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.
Interventions
OTHERBlood 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.
Sponsors
Centre Hospitalier Régional de la Citadelle
Centre Hospitalier Universitaire de Liege
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
No minimum to 3 Weeks
Healthy volunteers
Yes
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
Countries
Belgium
Contacts
Primary ContactTamara Dangouloff, PhD
Outcome results
None listed