GENetic & Immunologic Abnomalies in Systemic Lupus Erythematosus

NCT ID: NCT01992666

Last Updated: 2025-12-19

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

COMPLETED

Clinical Phase

NA

Total Enrollment

271 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-10-31

Study Completion Date

2016-10-31

Brief Summary

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Systemic Lupus Erythematosus (SLE) is a chronic autoimmune disease for which the aetiology includes genet-ic and environmental factors. It is rare in children as compared to adults. The severity may be related to greater involvement of genetic factors in children. The impact of genetics in the development of SLE is important, and the risk of recurrence in siblings evaluated by lambda S ratio is 30 in SLE, while it is 15 for type-1 diabetes and 8 rheumatoid arthritis, thereby indicating high impact of genetics in SLE.

Recently, the group of Professor Yanick Crow in Manchester and other teams has identified new forms of lupus Mendelian genetics. The TREX1 and genes involved in the SAMHD1 frostbite lupus.

Nearly 2 % of all adult subjects with SLE have a heterozygous mutation in the TREX1 gene, which therefore represents the first genetic cause of SLE. The team of Professor Crow also identified the ACP5 gene that is responsible for SLE associated with Spondylo-epiphyseal enchondro-epiphyseal dysplasia (syndromic lupus). Other groups have identified mutations in two genes encoding a DNAse (DNAse1 and DNAse1L3) responsible for familial monogenic forms of SLE. These new genes SLE were identified through research of germ-line mutations in cases of lupus syndromic or family. In collaboration with Professor Crow, we are currently undergoing characterization of a novel gene of SLE in a family and we have identified a second locus identified in another family. The identification of these genes provides a better understanding of the mechanisms regulating immune tolerance in humans. The frequency of these genetic forms is not known. There is very little data on the immunological phenotype of these patients.

This is a clinical study to investigate the genetic and immunological abnormalities associated with pediatric SLE. The aim are to:

* study the genetics of pediatric SLE (or syndromic or family) and to search for mutations in the known genetic lupus or new genes in collaboration with Professor Yanick Crow.
* study the lymphocyte subpopulations and serum cytokines in pediatric patients with SLE (or syndromic or family) in the large Rhône- Alpes- Auvergne area.

Detailed Description

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Conditions

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Systemic Lupus Erythematosus (SLE)

Keywords

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Systemic lupus erythematosus genetic immunological pediatric mutation lupus

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

OTHER

Blinding Strategy

NONE

Study Groups

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Blood sampling

Group Type EXPERIMENTAL

Blood sampling

Intervention Type GENETIC

Immunologic and genetic analysis from a single blood sample.

Interventions

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Blood sampling

Immunologic and genetic analysis from a single blood sample.

Intervention Type GENETIC

Eligibility Criteria

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Inclusion Criteria

1. Male or female subject, major or minor of any age with SLE (defined according to the ACR criteria)

* Onset pediatric (\<18 years) OR
* Syndromic Lupus (associated with growth retardation, neurological deficit not related to lupus, frostbite, lymphoproliferation, the kidney malformations, heart, lung, brain calcifications) OR
* Lupus in context with familial consanguinity OR
* Familial cases (2 cases of SLE related first degree relative) OR related topic of the first degree to a lupus patient participant (if family lupus or related parents) OR
* mother/father's lupus patient (in cas of simplex lupus)
2. A person or beneficiary entitled to a social security scheme or similar
3. Informed consent signed by the person (or parent / holding parental authority for minors)

Exclusion Criteria

\- none
Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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Hospices Civils de Lyon

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Alexandre Belot, Dr

Role: STUDY_DIRECTOR

Hospices Civils de Lyon

Locations

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Service de médecine interne - Centre de référence des maladies rares

Lille, , France

Site Status

Service de néphrologie, endocrinologie, maladie métabolique et hématologie bénigne pédiatriques - Hôpital Jeanne de Flandre

Lille, , France

Site Status

édiatrie générale, urgences et maladies infectieuses, Hôpital Salengro

Lille, , France

Site Status

Service de Néphrologie - Hôpital Edouard Herriot

Lyon, , France

Site Status

Centre de néphrologie et de transplantation rénale - Hôpital de la conception

Marseille, , France

Site Status

Service de médecine infantile- Hôpital Nord

Marseille, , France

Site Status

Service de médecine interne - Hôpitaux privés de Metz

Metz, , France

Site Status

ervice d'urgence et post-urgences pédiatriques - CHU Arnaud de Villeneuve

Montpellier, , France

Site Status

Service médecine infantile 2

Nancy, , France

Site Status

Service de néphrologie pédiatrique - CHU de Nantes

Nantes, , France

Site Status

Service d'immunologie et rhumatologie pédiatrique - Centre de référence de maladies rhumatologiques et inflammatoires rares en pédiatrie-Hôpital Necker-Enfants malades

Paris, , France

Site Status

Service de médecine interne - Hôpital Saint Antoine

Paris, , France

Site Status

Service de pédiatrie générale - Hôpital Robert-Debré

Paris, , France

Site Status

Médecine Interne Adulte - Centre Hospitalier Lyon Sud

Pierre-Bénite, , France

Site Status

Service de Rhumatologie - Centre Hospitalier Lyon Sud

Pierre-Bénite, , France

Site Status

Service pédiatrie grands enfants-adolescents - CHU Hôpital Sud

Rennes, , France

Site Status

Hôpital Nord

Saint-Etienne, , France

Site Status

Service de Pédiatrie Générale - CHU Réunion

Saint-Pierre, , France

Site Status

Service de néphrologie - médecine interne - Hypertension pédiatrique - Hôpital des enfants

Toulouse, , France

Site Status

Service d'hématologie / oncologie pédiatrique - CHU

Angers, , France

Site Status

Néphrologie Pédiatrique - CHU Besançon

Besançon, , France

Site Status

Hôpital Femme Mère Enfant

Bron, , France

Site Status

Service de Néphrologie Pédiatrique

Clermont-Ferrand, , France

Site Status

Service de pédiatrie - CHU Fort de France

Fort de France, , France

Site Status

Service de Néphrologie et Rhumatologie Pédiatrique

Grenoble, , France

Site Status

Service de Rhumatologie Pédiatrique - Hôpital de Bicêtre

Le Kremlin-Bicêtre, , France

Site Status

Countries

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France

References

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Weill O, Decramer S, Malcus C, Kassai B, Rouvet I, Ginhoux T, Crow YJ, Rieux-Laucat F, Soulas-Sprauel P, Pagnier A, Kone-Paut I, Piram M, Galeotti C, Samaille C, Reumaux H, Lanteri A, Dubois SM, Lefebvre H, Burtey S, Maurier F, Carbasse A, Lemelle I, Meinzer U, Despert V, Flodrops H, Fabien N, Ranchin B, Hachulla E, Bader-Meunier B, Belot A. Familial and syndromic lupus share the same phenotype as other early-onset forms of lupus. Joint Bone Spine. 2017 Oct;84(5):589-593. doi: 10.1016/j.jbspin.2016.12.008. Epub 2016 Dec 28.

Reference Type RESULT
PMID: 28039062 (View on PubMed)

Other Identifiers

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2012-A01449-34

Identifier Type: OTHER

Identifier Source: secondary_id

2012.769

Identifier Type: -

Identifier Source: org_study_id