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National Prospective Cohort for Monitoring Children With Severe Autoimmune Cytopenia. (BIOCEREVANCE)

27. august 2019 opdateret af: University Hospital, Bordeaux

National Prospective Cohort for Monitoring Children With Severe Autoimmune Cytopenia

This study aims to study prospectively the clinical and paraclinical evolution and prognostic factors of autoimmune haemolytic anemias, Evans syndromes and chronic immunological thrombocytopenic purpura of children in France.

Studieoversigt

Status

Afsluttet

Betingelser

Intervention / Behandling

Detaljeret beskrivelse

These autoimmune haematological diseases are rare diseases affecting the child, often very young, and serious and potentially life-threatening. International literature data are scarce, and include individual cases or small series.

They do not allow to determine an optimal therapeutic strategy in case of escape from the first-line treatments. Existing treatments (long-term corticosteroid therapy, immunoglobulins, splenectomy, immunosuppressants, chemotherapies, and more recently anti-CD20 antibodies) are inconsistently effective, and often associated with serious side effects.

The seriousness of these diseases, the therapeutic difficulties, and the absence of a targeted research project in France, led to the implementation of this study.

This study aims to study prospectively the clinical and paraclinical evolution and prognostic factors of autoimmune haemolytic anemias, Evans syndromes and chronic immunological thrombocytopenic purpura of children in France.

Undersøgelsestype

Observationel

Tilmelding (Faktiske)

122

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

Ikke ældre end 18 år (Barn, Voksen)

Tager imod sunde frivillige

Ingen

Køn, der er berettiget til at studere

Alle

Beskrivelse

Inclusion Criteria:

  • Age strictly below 18 years of age at initial diagnosis
  • Affiliate child or beneficiary of a social security scheme
  • Child residing in metropolitan France
  • Diagnosis of autoimmune haemolytic anemias, Evans syndrome and / or chronic Immune thrombocytopenic purpura
  • Free, informed, written and signed consent

Exclusion Criteria:

  • Diagnosis of constitutional haemolytic anemia
  • Diagnosis of platelet constitutional disease

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Kohorter og interventioner

Gruppe / kohorte
Intervention / Behandling
children with autoimmune haemolytic anemia
A blood sample of 2 times 2 to 5 ml additional maximum
Andet: Blood sample
A blood sample of 2 times 2 to 5 ml additional maximum
Children with Evans syndrome
A blood sample of 2 times 2 to 5 ml additional maximum
Andet: Blood sample
A blood sample of 2 times 2 to 5 ml additional maximum
Children with Immune thrombocytopenic purpura
A blood sample of 2 times 2 to 5 ml additional maximum
Andet: Blood sample
A blood sample of 2 times 2 to 5 ml additional maximum

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Complete sustainable remission (yes/no) for children with autoimmune haemolytic anemias
Tidsramme: At the screening
Absence of clinical signs of anemia (grade 0) And Hemoglobin > 11 g / dl And reticulocytes <120,000 / mm3 And haptoglobin> 10 mg / dl And bilirubin <10 mg / l or 17 μmol / l And no specific treatment for at least 12 months
At the screening
complete remission (yes/no) for children with autoimmune haemolytic anemias
Tidsramme: At the screening
Absence of clinical signs of anemia (grade 0) And Hemoglobin> 11 g / dl And reticulocytes <120,000 / mm3 Regardless of the level of haptoglobin or bilirubin And specific treatment in progress or interrupted for less than 12 months
At the screening
partial remission (yes/no) for children with autoimmune haemolytic anemias
Tidsramme: At the screening
Clinical Signs of Anemia (Grade 1 or 2) Or Hemoglobin from 7 to 11 g / dl Or reticulocytes> 120,000 / mm3 Regardless of the level of haptoglobin or bilirubin
At the screening
no response (yes/no) for children with autoimmune haemolytic anemias
Tidsramme: At the screening
Clinical Signs of Severe Anemia (Grade 3 or More) Or Hemoglobin <7 g / dl
At the screening
deceased patient (yes/no) for children with autoimmune haemolytic anemias
Tidsramme: At the screening
Death yes/no
At the screening
Complete sustainable remission (yes/no) for children with chronic immunologic thrombocytopenic purpura
Tidsramme: At the screening
Absence of clinical signs of haemorrhage (grade 0) And platelets> 100,000 / mm3 And no specific treatment for at least 12 months
At the screening
complete remission (yes/no) for children with chronic immunologic thrombocytopenic purpura
Tidsramme: At the screening
Absence of clinical signs of haemorrhage (grade 0) And platelets> 100,000 / mm3 And specific treatment in progress or interrupted for less than 12 months
At the screening
partial remission (yes/no) for children with chronic immunologic thrombocytopenic purpura
Tidsramme: At the screening
Clinical Signs of Hemorrhage (Grade 1 or 2) Or platelets between 30,000 and 100,000 / mm3
At the screening
no response (yes/no) for children with chronic immunologic thrombocytopenic purpura
Tidsramme: At the screening
Clinical Signs of Severe Hemorrhage (Grade 3 or Greater) Or Platelets <30,000 / mm3
At the screening
deceased patient (yes/no) for children with chronic immunologic thrombocytopenic purpura
Tidsramme: At the screening
Death yes/no
At the screening

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

University Hospital, Bordeaux

Efterforskere

  • Ledende efterforsker: Yves PEREL, Pr, Bordeaux University Hsopital

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Faktiske)

4. april 2007

Primær færdiggørelse (Faktiske)

8. juni 2012

Studieafslutning (Faktiske)

8. juni 2012

Datoer for studieregistrering

Først indsendt

26. august 2019

Først indsendt, der opfyldte QC-kriterier

26. august 2019

Først opslået (Faktiske)

28. august 2019

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

28. august 2019

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

27. august 2019

Sidst verificeret

1. august 2019

Mere information

Begreber relateret til denne undersøgelse

Nøgleord

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • CHUBX 2005/18

Plan for individuelle deltagerdata (IPD)

Planlægger du at dele individuelle deltagerdata (IPD)?

INGEN

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

Studerer et amerikansk FDA-reguleret lægemiddelprodukt

Ingen

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ingen

Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .

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