Study of Biological Markers in Children With Sickle Cell Disease

NCT ID: NCT04839159

Last Updated: 2021-04-09

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

NA

Total Enrollment

41 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-05-10

Study Completion Date

2021-06-30

Brief Summary

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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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Detailed Description

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Conditions

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Sickle Cell Disease

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

DIAGNOSTIC

Blinding Strategy

NONE

Study Groups

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SCD Patient

Group Type EXPERIMENTAL

Blood sampling

Intervention Type OTHER

Blood sampling at the age of 6 and 12 months, 2-3-4 years

Blood sampling

Intervention Type OTHER

Blood sampling before any new sickle cell disease treatment as determined by a physician according to the standard of care to which the hospital adheres

Interventions

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Blood sampling

Blood sampling at the age of 6 and 12 months, 2-3-4 years

Intervention Type OTHER

Blood sampling

Blood sampling before any new sickle cell disease treatment as determined by a physician according to the standard of care to which the hospital adheres

Intervention Type OTHER

Eligibility Criteria

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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
Minimum Eligible Age

6 Months

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Queen Fabiola Children's University Hospital

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Bushra Zucca, MD

Role: PRINCIPAL_INVESTIGATOR

Queen Fabiola Children's University Hospital

Locations

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CHU Saint Pierre

Brussels, , Belgium

Site Status

Hôpital Universitaire Des Enfants Reine Fabiola

Brussels, , Belgium

Site Status

HIS - Site Etterbeek-Ixelles

Brussels, , Belgium

Site Status

Countries

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Belgium

References

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Ware RE, de Montalembert M, Tshilolo L, Abboud MR. Sickle cell disease. Lancet. 2017 Jul 15;390(10091):311-323. doi: 10.1016/S0140-6736(17)30193-9. Epub 2017 Feb 1.

Reference Type BACKGROUND
PMID: 28159390 (View on PubMed)

Gulbis B, Cotton F, Ferster A, Ketelslegers O, Dresse MF, Ronge-Collard E, Minon JM, Le PQ, Vertongen F. Neonatal haemoglobinopathy screening in Belgium. J Clin Pathol. 2009 Jan;62(1):49-52. doi: 10.1136/jcp.2008.060517.

Reference Type BACKGROUND
PMID: 19103861 (View on PubMed)

Ballas SK, Lieff S, Benjamin LJ, Dampier CD, Heeney MM, Hoppe C, Johnson CS, Rogers ZR, Smith-Whitley K, Wang WC, Telen MJ; Investigators, Comprehensive Sickle Cell Centers. Definitions of the phenotypic manifestations of sickle cell disease. Am J Hematol. 2010 Jan;85(1):6-13. doi: 10.1002/ajh.21550.

Reference Type BACKGROUND
PMID: 19902523 (View on PubMed)

Horan J, Lerner N. Prediction of adverse outcomes in children with sickle cell disease. N Engl J Med. 2000 May 25;342(21):1612-3. doi: 10.1056/NEJM200005253422114. No abstract available.

Reference Type BACKGROUND
PMID: 10841686 (View on PubMed)

Miller ST, Sleeper LA, Pegelow CH, Enos LE, Wang WC, Weiner SJ, Wethers DL, Smith J, Kinney TR. Prediction of adverse outcomes in children with sickle cell disease. N Engl J Med. 2000 Jan 13;342(2):83-9. doi: 10.1056/NEJM200001133420203.

Reference Type BACKGROUND
PMID: 10631276 (View on PubMed)

Quinn CT, Lee NJ, Shull EP, Ahmad N, Rogers ZR, Buchanan GR. Prediction of adverse outcomes in children with sickle cell anemia: a study of the Dallas Newborn Cohort. Blood. 2008 Jan 15;111(2):544-8. doi: 10.1182/blood-2007-07-100719. Epub 2007 Oct 1.

Reference Type BACKGROUND
PMID: 17909076 (View on PubMed)

Gladwin MT, Vichinsky E. Pulmonary complications of sickle cell disease. N Engl J Med. 2008 Nov 20;359(21):2254-65. doi: 10.1056/NEJMra0804411. No abstract available.

Reference Type BACKGROUND
PMID: 19020327 (View on PubMed)

Charrin E, Ofori-Acquah SF, Nader E, Skinner S, Connes P, Pialoux V, Joly P, Martin C. Inflammatory and oxidative stress phenotypes in transgenic sickle cell mice. Blood Cells Mol Dis. 2016 Nov;62:13-21. doi: 10.1016/j.bcmd.2016.10.020. Epub 2016 Oct 28.

Reference Type BACKGROUND
PMID: 27835777 (View on PubMed)

Sarray S, Saleh LR, Lisa Saldanha F, Al-Habboubi HH, Mahdi N, Almawi WY. Serum IL-6, IL-10, and TNFalpha levels in pediatric sickle cell disease patients during vasoocclusive crisis and steady state condition. Cytokine. 2015 Mar;72(1):43-7. doi: 10.1016/j.cyto.2014.11.030. Epub 2015 Jan 5.

Reference Type BACKGROUND
PMID: 25569375 (View on PubMed)

Hoppe C, Klitz W, Noble J, Vigil L, Vichinsky E, Styles L. Distinct HLA associations by stroke subtype in children with sickle cell anemia. Blood. 2003 Apr 1;101(7):2865-9. doi: 10.1182/blood-2002-09-2791. Epub 2002 Nov 27.

Reference Type BACKGROUND
PMID: 12517810 (View on PubMed)

Garrido VT, Proenca-Ferreira R, Dominical VM, Traina F, Bezerra MA, de Mello MR, Colella MP, Araujo AS, Saad ST, Costa FF, Conran N. Elevated plasma levels and platelet-associated expression of the pro-thrombotic and pro-inflammatory protein, TNFSF14 (LIGHT), in sickle cell disease. Br J Haematol. 2012 Sep;158(6):788-97. doi: 10.1111/j.1365-2141.2012.09218.x. Epub 2012 Jul 6.

Reference Type BACKGROUND
PMID: 22775554 (View on PubMed)

Other Identifiers

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P2012/SCD1

Identifier Type: -

Identifier Source: org_study_id

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