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

Primary Purpose

Cytopenia, Autoimmune Haemolytic Anaemia, Thrombocytopenic Purpura, Immune

Status
Completed
Phase
Locations
Study Type
Observational
Intervention
Blood sample
Sponsored by
University Hospital, Bordeaux
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an observational trial for Cytopenia focused on measuring Children

Eligibility Criteria

undefined - 18 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

    Arms of the Study

    Arm 1

    Arm 2

    Arm 3

    Arm Type

    Arm Label

    children with autoimmune haemolytic anemia

    Children with Evans syndrome

    Children with Immune thrombocytopenic purpura

    Arm Description

    A blood sample of 2 times 2 to 5 ml additional maximum

    A blood sample of 2 times 2 to 5 ml additional maximum

    A blood sample of 2 times 2 to 5 ml additional maximum

    Outcomes

    Primary Outcome Measures

    Complete sustainable remission (yes/no) for children with autoimmune haemolytic anemias
    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
    complete remission (yes/no) for children with autoimmune haemolytic anemias
    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
    partial remission (yes/no) for children with autoimmune haemolytic anemias
    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
    no response (yes/no) for children with autoimmune haemolytic anemias
    Clinical Signs of Severe Anemia (Grade 3 or More) Or Hemoglobin <7 g / dl
    deceased patient (yes/no) for children with autoimmune haemolytic anemias
    Death yes/no
    Complete sustainable remission (yes/no) for children with chronic immunologic thrombocytopenic purpura
    Absence of clinical signs of haemorrhage (grade 0) And platelets> 100,000 / mm3 And no specific treatment for at least 12 months
    complete remission (yes/no) for children with chronic immunologic thrombocytopenic purpura
    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
    partial remission (yes/no) for children with chronic immunologic thrombocytopenic purpura
    Clinical Signs of Hemorrhage (Grade 1 or 2) Or platelets between 30,000 and 100,000 / mm3
    no response (yes/no) for children with chronic immunologic thrombocytopenic purpura
    Clinical Signs of Severe Hemorrhage (Grade 3 or Greater) Or Platelets <30,000 / mm3
    deceased patient (yes/no) for children with chronic immunologic thrombocytopenic purpura
    Death yes/no

    Secondary Outcome Measures

    Full Information

    First Posted
    August 26, 2019
    Last Updated
    August 27, 2019
    Sponsor
    University Hospital, Bordeaux
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    1. Study Identification

    Unique Protocol Identification Number
    NCT04070612
    Brief Title
    National Prospective Cohort for Monitoring Children With Severe Autoimmune Cytopenia.
    Acronym
    BIOCEREVANCE
    Official Title
    National Prospective Cohort for Monitoring Children With Severe Autoimmune Cytopenia
    Study Type
    Observational

    2. Study Status

    Record Verification Date
    August 2019
    Overall Recruitment Status
    Completed
    Study Start Date
    April 4, 2007 (Actual)
    Primary Completion Date
    June 8, 2012 (Actual)
    Study Completion Date
    June 8, 2012 (Actual)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    University Hospital, Bordeaux

    4. Oversight

    Studies a U.S. FDA-regulated Drug Product
    No
    Studies a U.S. FDA-regulated Device Product
    No
    Data Monitoring Committee
    No

    5. Study Description

    Brief Summary
    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.
    Detailed Description
    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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Cytopenia, Autoimmune Haemolytic Anaemia, Thrombocytopenic Purpura, Immune
    Keywords
    Children

    7. Study Design

    Enrollment
    122 (Actual)

    8. Arms, Groups, and Interventions

    Arm Title
    children with autoimmune haemolytic anemia
    Arm Description
    A blood sample of 2 times 2 to 5 ml additional maximum
    Arm Title
    Children with Evans syndrome
    Arm Description
    A blood sample of 2 times 2 to 5 ml additional maximum
    Arm Title
    Children with Immune thrombocytopenic purpura
    Arm Description
    A blood sample of 2 times 2 to 5 ml additional maximum
    Intervention Type
    Other
    Intervention Name(s)
    Blood sample
    Intervention Description
    A blood sample of 2 times 2 to 5 ml additional maximum
    Primary Outcome Measure Information:
    Title
    Complete sustainable remission (yes/no) for children with autoimmune haemolytic anemias
    Description
    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
    Time Frame
    At the screening
    Title
    complete remission (yes/no) for children with autoimmune haemolytic anemias
    Description
    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
    Time Frame
    At the screening
    Title
    partial remission (yes/no) for children with autoimmune haemolytic anemias
    Description
    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
    Time Frame
    At the screening
    Title
    no response (yes/no) for children with autoimmune haemolytic anemias
    Description
    Clinical Signs of Severe Anemia (Grade 3 or More) Or Hemoglobin <7 g / dl
    Time Frame
    At the screening
    Title
    deceased patient (yes/no) for children with autoimmune haemolytic anemias
    Description
    Death yes/no
    Time Frame
    At the screening
    Title
    Complete sustainable remission (yes/no) for children with chronic immunologic thrombocytopenic purpura
    Description
    Absence of clinical signs of haemorrhage (grade 0) And platelets> 100,000 / mm3 And no specific treatment for at least 12 months
    Time Frame
    At the screening
    Title
    complete remission (yes/no) for children with chronic immunologic thrombocytopenic purpura
    Description
    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
    Time Frame
    At the screening
    Title
    partial remission (yes/no) for children with chronic immunologic thrombocytopenic purpura
    Description
    Clinical Signs of Hemorrhage (Grade 1 or 2) Or platelets between 30,000 and 100,000 / mm3
    Time Frame
    At the screening
    Title
    no response (yes/no) for children with chronic immunologic thrombocytopenic purpura
    Description
    Clinical Signs of Severe Hemorrhage (Grade 3 or Greater) Or Platelets <30,000 / mm3
    Time Frame
    At the screening
    Title
    deceased patient (yes/no) for children with chronic immunologic thrombocytopenic purpura
    Description
    Death yes/no
    Time Frame
    At the screening

    10. Eligibility

    Sex
    All
    Maximum Age & Unit of Time
    18 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    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
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Yves PEREL, Pr
    Organizational Affiliation
    Bordeaux University Hsopital
    Official's Role
    Principal Investigator

    12. IPD Sharing Statement

    Plan to Share IPD
    No

    Learn more about this trial

    National Prospective Cohort for Monitoring Children With Severe Autoimmune Cytopenia.

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