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

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ClinicalTrials.gov Identifier: NCT02722681
Recruitment Status : Unknown
Verified March 2016 by Institute of Child Health.
Recruitment status was:  Recruiting
First Posted : March 30, 2016
Last Update Posted : March 30, 2016
Sponsor:
Collaborator:
Great Ormond Street Hospital for Children NHS Foundation Trust
Information provided by (Responsible Party):
Institute of Child Health

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.

Condition or disease Intervention/treatment
Lipoma of Spinal Cord Procedure: Blood and urine sampling Procedure: Cerebrospinal fluid sampling

Detailed Description:

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 sizes will be determined by power calculations using variance data from the patient measurements in Phase 1. Statistical analysis will be by 1‐way ANOVA, or non‐parametric equivalent, to test for significant differences between the three groups. Mass spectrometry (Figure 2) will be performed in the Institute of Child Health Centre for Proteomics, Metabolomics and Lipidomics using nano ultra performance liquid chromatography and ultra performance convergence chromatography - quadrupole time of flight mass spectrometry, a new mass spectral technology for lipidomic and metabolomic analysis. Ultra performance convergence chromatography is a chromatography technology that uses carbon dioxide present in a super critical fluid state as a mobile phase and allows the fractionation of metabolites and lipids according to their class and not hydrophobicity. It enables quantitation of all the major lipid classes present in a tissue including phospholipids, free fatty acids, esterified fatty acids, cholesterol esters and sterols. Non‐lipid molecules (e.g. choline) might also show altered abundance in lipoma patients and so a more general metabolomics analysis will also be undertaken, if time permits.


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Study Type : Observational
Estimated Enrollment : 50 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Developing a Biomarker for Monitoring Clinical Outcomes in Children With Spinal Lipoma
Study Start Date : February 2016
Estimated Primary Completion Date : December 2018
Estimated Study Completion Date : December 2018

Resource links provided by the National Library of Medicine


Group/Cohort Intervention/treatment
Symptomatic spinal lipoma patients
Spinal lipoma patients undergoing surgery due to symptomatic lipoma. Routine blood and urine samples will be taken pre-operatively, some will be kept aside for research. Cerebrospinal fluid is drained intraoperatively and usually discarded, some will be kept for research.
Procedure: Blood and urine sampling
Collection of blood and urine samples taken during usual clinical management

Procedure: Cerebrospinal fluid sampling
Collection of cerebrospinal fluid samples taken during usual clinical management

Asymptomatic spinal lipoma patients
Spinal lipoma patients who remain asymptomatic. Routine blood and urine samples will be taken as part of routine clinical care, some will be kept for research.
Procedure: Blood and urine sampling
Collection of blood and urine samples taken during usual clinical management

Non-lipoma spinal conditions
Patients undergoing spinal surgery for a non-lipoma related condition. Routine blood and urine samples will be taken pre-operatively, some will be kept aside for research. Cerebrospinal fluid is drained intra-operatively and usually discarded, some will be kept for research.
Procedure: Blood and urine sampling
Collection of blood and urine samples taken during usual clinical management

Procedure: Cerebrospinal fluid sampling
Collection of cerebrospinal fluid samples taken during usual clinical management




Primary Outcome Measures :
  1. Lipid signature within blood or urine samples of patients with symptomatic spinal lipomas [ Time Frame: 2 years ]
    Lipid profiles from urine and blood samples from spinal lipoma patients will show specific lipids present at a higher concentration in symptomatic patients when compared with asymptomatic patients.


Biospecimen Retention:   Samples With DNA
Blood samples will be stored during length of project.


Information from the National Library of Medicine

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Ages Eligible for Study:   1 Year to 16 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Children under the age of 16 attending GOSH for management of spinal lipomas and non-lipoma related spinal conditions
Criteria

Inclusion Criteria:

  • patient with proven spinal lipoma

Exclusion Criteria:

  • complex spinal lipomas related to other developmental abnormalities

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02722681


Contacts
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Contact: Victoria Jones 02072429789

Locations
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United Kingdom
Institute of Child Health Recruiting
London, United Kingdom, WC1N1EH
Contact: Victoria Jones    02072429789      
Sponsors and Collaborators
Institute of Child Health
Great Ormond Street Hospital for Children NHS Foundation Trust
Investigators
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Study Chair: Andrew Copp, PhD Institute of Child Health, UCL
Study Director: Dominic Thompson, FRCS Great Ormond Street Hospital NHS Trust
Study Director: Kevin Mills, PhD Institute of Child Health, UCL

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Responsible Party: Institute of Child Health
ClinicalTrials.gov Identifier: NCT02722681     History of Changes
Other Study ID Numbers: 13ND18
First Posted: March 30, 2016    Key Record Dates
Last Update Posted: March 30, 2016
Last Verified: March 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Additional relevant MeSH terms:
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Lipoma
Neoplasms, Adipose Tissue
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Neoplasms