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Model Early Immunologic Stages of Pediatric Hematological Pre-lupus (PRELUDE)

25 juni 2026 bijgewerkt door: University Hospital, Bordeaux

Model Early Immunologic Stages of Pediatric Hematological Pre-lupus in Order to Prevent SLE in Children.

Immunologic thrombocytopenic purpura (ITP) in children is a pre-lupus condition if associated with the presence of anti-nuclear antibodies (ANA), providing a unique model for understanding the natural history of autoimmunity, particularly that of systemic lupus erythematosus (SLE). We will describe the shared and/or unique immunological pathways involved at diagnosis in 70 children with ITP and in 20 children with SLE, and compare them between ITP-ANA- (more often transient), ITP-ANA+ (pre-lupus condition, more often persistent) and SLE

Studie Overzicht

Toestand

Nog niet aan het werven

Conditie

Interventie / Behandeling

Gedetailleerde beschrijving

The development of an autoimmune disease (AID) requires several years. In systemic lupus erythematosus (SLE), anti-nuclear antibodies (ANA) appear several years before the onset of the disease. However, it is difficult to identify the early mechanisms of autoimmunity in humans as most patients' samples are obtained at the time of diagnosis.

We hypothesize that immune thrombocytopenic purpura (ITP) in children provides a unique model for understanding the initial mechanisms leading to autoimmunity. Children with ITP have common immune system dysfunctions leading to the production of anti-platelet antibodies. However, despite this supposedly common dysfunction, the course and degree of loss of tolerance of the immune system are very heterogeneous: the disease is transient in 75 to 80% of cases, and 15 to 20% of children with ITP have ANA and their course is usually persistent or chronic. This subgroup of children with hematological AID, ITP ANA+ meets the criteria for a broader form of AID: pre-lupus. We have recently shown that 16% of these children with pre-lupus / ITP-ANA+ progressed to complete SLE within a median of 3.8 years. Why do some children have transient or persistent ITP? limited or systemic ITP? We aim to describe the shared and/or unique immunological pathways involved at diagnosis in children with ITP ANA- (transient or persistent), with ITP ANA+ (pre-lupus condition, transient or persistent), and in patients with SLE (persistent by definition) In this exploratory, prospective, bi-centric cohort study, we will include 70 children with ITP and 20 children with SLE newly diagnosed, over 36 months with a 3-month follow-up for children with ITP-ANA- and SLE and a 48-month follow-up for children with ITP-ANA+. ANA positivity will be defined by a titer ≥ 1/160. Blood samples will be collected at inclusion and at 3 months (bio-collection), and further each 6 months for children with ITP-ANA+. ITP status will be defined at 3 months: transient or persistent. We will analyze the signaling pathways (B and T lymphocytes, cytokines, disruption of tolerance and inactivation of X) involved in these different pathophysiological distinct subgroups

Studietype

Ingrijpend

Inschrijving (Geschat)

105

Fase

  • Niet toepasbaar

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studiecontact

Studie Contact Back-up

Studie Locaties

  • Frankrijk
      • Bordeaux, Frankrijk, 33076
        • Chu de Bordeaux- Groupe Hospitalier Pellegrin - Hôpital des Enfants
        • Contact:
      • Toulouse, Frankrijk, 31026
        • CHU Toulouse - Hôpital des Enfants
        • Contact:

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

  • Kind
  • Volwassen

Accepteert gezonde vrijwilligers

Ja

Beschrijving

  • Inclusion criteria:

    • For patients :

      • Child or adolescent with newly diagnosed ITP or SLE according to the specific definitions of ITP or SLE, prior to any treatment,
      • Over 1 and under 18 years of age at diagnosis, weighing more than 7 kg.
      • Written consent from parents or guardians,
      • Patient affiliated to a social security scheme.

    • For controls :

      • Over 1 and under 18 years of age at diagnosis, weighing more than 7 kg.
      • Follow-up in the day hospital at the Bordeaux University Hospital, for a condition that does not affect the immune system
      • Matched on age,
      • Written consent from parents or guardians,
      • Patient affiliated to a social security scheme

  • Exclusion criteria:

    • For patients :

      • ITP secondary to a known cause: previous or concomitant immune deficiency, bone marrow or organ transplantation, other autoimmune disease, Evans syndrome (autoimmune hemolytic anemia or autoimmune neutropenia present at ITP diagnosis) or cancer with immunosuppressive therapy.
      • Treatment with immunomodulation or immunosuppressants (including immunoglobulins, corticoids, hydroxychloroquine), started prior to inclusion (day of sampling).
      • Pregnant women, women in labour and breastfeeding women

    • For controls :

      • Suffering from an immunological disease,
      • Infection within fifteen days prior to inclusion,
      • Immunomodulatory therapy.
      • Pregnant women, women in labour and breastfeeding women

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Primair doel: Ander
  • Toewijzing: Niet-gerandomiseerd
  • Interventioneel model: Parallelle opdracht
  • Masker: Geen (open label)

Wapens en interventies

Deelnemersgroep / Arm
Interventie / Behandeling
Ander: Controles
Procedure: Blood sampling baseline and M3
Blood sampling is a routine biological procedure performed under the same conditions as during a follow-up consultation.
Ander: Monitoring for SLE
Monitoring for Systemic Lupus Erythematosus according to SLICC 2012 classification criteria
Ander: ITP-ANA-
Procedure: Blood sampling baseline and M3
Blood sampling is a routine biological procedure performed under the same conditions as during a follow-up consultation.
Ander: Monitoring for SLE
Monitoring for Systemic Lupus Erythematosus according to SLICC 2012 classification criteria
Ander: ITP-ANA+
Procedure: Procedure/Surgery: Blood sampling baseline and M3 then each 6 months until 48 months
Blood sampling is a routine biological procedure performed under the same conditions as during a follow-up consultation.
Ander: moniktoring for SLE : baseline and M3 then each 6 months until 48 months
Monitoring for Systemic Lupus Erythematosus according to SLICC 2012 classification criteria
Ander: SLE
Procedure: Blood sampling baseline and M3
Blood sampling is a routine biological procedure performed under the same conditions as during a follow-up consultation.
Ander: Monitoring for SLE
Monitoring for Systemic Lupus Erythematosus according to SLICC 2012 classification criteria

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Characterisation of B and T lymphocyte populations
Tijdsspanne: Baseline, Month 3 visit.
Characterisation of B and T lymphocyte populations through the study of surface markers and intracellular factors. This panel will, in particular, enable the identification of effector B cells such as plasma blasts, subsets of auto-reactive 'double-negative' (DN) B cells, regulatory B cells, and follicular T helper (Tfh) and peripheral extra-follicular T helper (Tph) cells. Each population will be compared between the patients and the control group.
Baseline, Month 3 visit.
Plasma markers
Tijdsspanne: Baseline, Month 3 visit.
Soluble cytokines and factors regulating B-cell survival (BAFF, TACI, IL-6, IL-12p70, IFN-γ, IFN-β, TGF-β, and the IFN-regulated chemokine CXCL10) will be quantified in plasma using a customised multiplex Luminex assay. Interferon alpha levels will be specifically measured using ultra-sensitive SIMOA (Single Molecule Array) technology.
Baseline, Month 3 visit.
Plasma markers
Tijdsspanne: Baseline, Month 3 visit.
Circulating plasma autoantigens - soluble P-selectin and CD40L derived from platelet activation - will be measured by ELISA. The levels of circulating and mitochondrial DNA will be measured by quantitative RT-PCR in collaboration with V. Sisirak (DR CNRS, Immunoconcept). The overall activity of plasma DNases will be determined by an in vitro DNA degradation assay.
Baseline, Month 3 visit.

Secundaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Single-cell transcriptomic analysis
Tijdsspanne: Baseline, Month 3 visit , Month 6 visit , Month 12 visit, Month 18 visit, Month 24 visit , Month 30 visit, Month 36 visit, Month 42 visit and Month 48 visit.

Using single-cell RNA sequencing, a single-cell transcriptomic analysis, we will be able to quantify and analyse the transcriptomes of lymphocytes B This will enable us to characterise heterogeneous cell populations, as well as to reconstruct cell development pathways and model transcriptomic dynamics.

This technique will enable the modelling of signalling pathways involving B lymphocytes in the subgroup of ITP patients with positive AAN titres, and the development of an algorithm and a mathematical model of the progression of these cellular and molecular markers
Baseline, Month 3 visit , Month 6 visit , Month 12 visit, Month 18 visit, Month 24 visit , Month 30 visit, Month 36 visit, Month 42 visit and Month 48 visit.
Single cell epigenetic analysis
Tijdsspanne: Baseline, Month 3 visit , Month 6 visit , Month 12 visit, Month 18 visit, Month 24 visit , Month 30 visit, Month 36 visit, Month 42 visit and Month 48 visit.

Using single-cell ATAC sequencing, we will be able to analyse the regions of DNA accessible to the transcription machinery. This will enable us to identify regulatory programmes such as signalling pathways. The analysis will also reveal heterogeneity amongst B-cell subpopulations, which may respond differently to signals.

This will provide insight into the epigenetic status of genes, or at least into which factors regulate this signalling pathway and how the cell adjusts its response depending on its situation or stage of maturity, or on the signal itself.
Baseline, Month 3 visit , Month 6 visit , Month 12 visit, Month 18 visit, Month 24 visit , Month 30 visit, Month 36 visit, Month 42 visit and Month 48 visit.

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Sponsor

University Hospital, Bordeaux

Onderzoekers

  • Hoofdonderzoeker: Jérôme GRANEL, MD, University Hospital, Bordeaux

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Geschat)

1 juli 2026

Primaire voltooiing (Geschat)

1 oktober 2029

Studie voltooiing (Geschat)

1 oktober 2033

Studieregistratiedata

Eerst ingediend

25 juni 2026

Eerst ingediend dat voldeed aan de QC-criteria

25 juni 2026

Eerst geplaatst (Werkelijk)

1 juli 2026

Updates van studierecords

Laatste update geplaatst (Werkelijk)

1 juli 2026

Laatste update ingediend die voldeed aan QC-criteria

25 juni 2026

Laatst geverifieerd

1 juni 2026

Meer informatie

Termen gerelateerd aan deze studie

Trefwoorden

Aanvullende relevante MeSH-voorwaarden

Andere studie-ID-nummers

  • CHUBX 2025/052

Informatie over medicijnen en apparaten, studiedocumenten

Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel

Nee

Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct

Nee

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