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Study of Biological Markers in Children With Sickle Cell Disease

Q

Queen Fabiola Children's University Hospital

Status

Unknown

Conditions

Sickle Cell Disease

Treatments

Other: Blood sampling

Study type

Interventional

Funder types

Other

Identifiers

NCT04839159
P2012/SCD1

Details and patient eligibility

About

Sickle cell disease is associated with significant morbi-mortality hence the interest in an early and targeted care. At present, there is no plasmatic marker able to identify infants at higher risk of developping severe complications later in life. However, recent studies have demonstrated a correlation between certain complications of the disease and biomarkers of the endothelial dysfunction characterizing it.

Investigators prospectively followed a cohort of children diagnosed with SCD through the universal neonatal screening using inflammatory and haemostatic plasmatic markers to study their annual evolution. Investigators then will evaluate potential associations between these biological markers and the occurrence of SCD related complications. A secondary objective of this study is to evaluate the repercussions of therapeutic intervention on these markers.

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Enrollment

41 estimated patients

Sex

All

Ages

6+ months old

Volunteers

No Healthy Volunteers

Inclusion criteria

  • Patient aged less than 6 months
  • Sickle cell syndrome SS, Sβthal or SC confirmed by hemoglobin electrophoresis
  • Subjects legal representatives must have signed an informed consent document indicating that they understand the purpose of and procedures required for the study and are willing to let participate their child in the study

Exclusion criteria

  • Congenital abnormality other than sickle cell disease except for a glucose-6-phosphate-deshydrogenase
  • Prematurity
  • Initiation of the following therapies before enrollment: chronic transfusion regimen or bone marrow transplantation

Trial design

Primary purpose

Diagnostic

Allocation

N/A

Interventional model

Single Group Assignment

Masking

None (Open label)

41 participants in 1 patient group

SCD Patient
Experimental group
Treatment:
Other: Blood sampling
Other: Blood sampling

Trial contacts and locations

3

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Data sourced from clinicaltrials.gov

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