Clinical Trial: Developing a Biomarker for Monitoring Clinical Outcomes in Children With Spinal Lipoma.

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational

Official Title: Developing a Biomarker for Monitoring Clinical Outcomes in Children With Spinal Lipoma

Brief Summary: 'Spinal lipoma' is a condition, present from before birth, in which fatty tissue (lipoma) is attached to the lower end of the spinal cord, tethering it within the vertebral canal. The cord normally moves up and down with respiration, whereas tethering prevents this movement, and can lead to progressive neurological deterioration. The cord and spinal nerves become stretched and their blood supply is damaged irreversibly. Disability may include weakness or pain in the lower body, and urinary disorders in young children. Treatment is surgical, to remove the lipoma and mobilise the spinal cord, with 60 such operations performed per year at Great Ormond Street Hospital. This project aims to develop lipidomic biomarkers in order to predict which children with spinal lipoma are at highest risk of neurological deterioration, and require early surgery, while providing evidence to adopt a more conservative approach for those at lower risk.

Detailed Summary:

Clinical study to seek a metabolic biomarker(s), detectable by mass spectrometry, that can be used to 'stratify' patients with asymptomatic lipoma. In view of the often extensive nature of lipomas associated with the low spinal cord, we hypothesise that lipid components, or metabolites derived from them, may gain entry to the child's bloodstream. The more infiltrative lipomas carry a higher risk of symptomatic deterioration and, we argue, should also have a higher chance of generating a lipid 'signature' in the blood and/or urine. Phase 1 ‐ Cerebrospinal fluid, blood and urine samples will be obtained from patients with spinal lipoma undergoing surgery (n = 3 to 5). Informed consent, following appropriate ethics committee approval, will be implemented. Blood and urine will be sampled pre‐operatively, and intra‐operative cerebrospinal fluid samples will be obtained. Mass spectrometry analysis will identify lipid species present in the cerebrospinal fluid of these patients, and the extent to which these are also detectable in the patient's blood and/or urine. Any lipid species detected in both cerebrospinal fluid and blood/urine will represent potential biomarkers, and will form the focus of the next phase of the study. Phase 2 will then assess the discriminatory value of these potential biomarkers by comparing their profiles in blood and/or urine from three clinical groups: (i) Patients with spinal lipomas who have neurological symptoms/signs, and are attending hospital for surgery (i.e. similar patients as in Phase 1). Blood/urine samples will be taken preoperatively, to represent the 'high risk' group. (ii) Patients with spinal lipomas who have remained asymptomatic after several years follow‐up.

These represent the 'low risk' group; (iii) Patients with spinal conditions not involving lipoma. These represent our 'negative' control group. Group siz
Sponsor: Institute of Child Health

Current Primary Outcome: Lipid signature within blood or urine samples of patients with symptomatic spinal lipomas [ Time Frame: 2 years ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Institute of Child Health

Dates:
Date Received: March 16, 2016
Date Started: February 2016
Date Completion: December 2018
Last Updated: March 23, 2016
Last Verified: March 2016