<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT04450784</url>
  </required_header>
  <id_info>
    <org_study_id>CHUBX 2018/31</org_study_id>
    <nct_id>NCT04450784</nct_id>
  </id_info>
  <brief_title>ObServatory Children Acute RElated Therapy Leukemia</brief_title>
  <acronym>OSCARE</acronym>
  <official_title>ObServatory Children Acute RElated Therapy Leukemia</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>University Hospital, Bordeaux</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>University Hospital, Bordeaux</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      Acute Myeloid Leukemias (AML) of the child are a rare disease and its prognosis varies&#xD;
      according to the subgroup. Secondary AMLs remain a subgroup of poor prognosis, whose&#xD;
      cytogenetic and molecular characteristics and prognostic value differ in part from de novo&#xD;
      AMLs. The purpose of this national observatory is to record scientific and medical&#xD;
      information on cases of secondary AML that have occurred in France since 2013 in order to&#xD;
      improve the treatment of children and adolescents with this disease in the years to come.&#xD;
      This national observatory will contribute to better knowledge and progress in research into&#xD;
      these diseases.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Therapy-related myeloid neoplasms (t-MNs) are a group of hematologic diseases that arise&#xD;
      after chemotherapy and/or radiation therapy for a previous cancer or rarely autoimmune&#xD;
      diseases. t-MNs had been included in the group of AMLs and remain as a distinct category also&#xD;
      in the recent 2016 revision of the WHO classification of myeloid neoplasm and acute leukemia.&#xD;
      The latency between exposition to anticancer drugs and development of t-MN may vary from some&#xD;
      months up to 10 years, even considering the age at diagnosis of the primary malignancy, the&#xD;
      kind of cytotoxic treatment previously used, and the cumulative dose and dose intensity. The&#xD;
      prognosis of s-AML is generally considered to be poorer than that of de novo AML. The disease&#xD;
      tends to be refractory to chemotherapy, and patients' tolerance of treatment generally is&#xD;
      reduced because of prior therapies. 5-year event-free survival (EFS) and overall survival&#xD;
      (OS) rates of pediatric t-AML/t-MDS have been reported to be 14% to 30%. However, the results&#xD;
      are conflicting and overall lacking when compared with those in adults. The purpose of this&#xD;
      national observatory is to record scientific and medical information on cases of secondary&#xD;
      Acute Myeloid Leukemias that have occurred in France since 2013 in order to improve the&#xD;
      treatment of children and adolescents with this disease in the years to come. The primary&#xD;
      objective is to evaluate the association of potential prognostic factors (including&#xD;
      clinical-biological factors) with the overall survival of children and adolescents aged 0-18&#xD;
      years diagnosed with secondary AML. The secondary objectives are to test the feasibility of&#xD;
      setting up a French national database of secondary AMLs for children and adolescents as a&#xD;
      first step towards European implementation, to assess the association of potential prognostic&#xD;
      factors with event free survival and the occurrence of treatment-related toxicities, to&#xD;
      characterize the molecular abnormalities associated with secondary AMLs in children and&#xD;
      adolescents.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Not yet recruiting</overall_status>
  <start_date type="Anticipated">September 1, 2020</start_date>
  <completion_date type="Anticipated">September 1, 2024</completion_date>
  <primary_completion_date type="Anticipated">September 1, 2024</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Cohort</observational_model>
    <time_perspective>Other</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>potential clinical-biological prognostic factors - patient characterics</measure>
    <time_frame>through study completion, an average of 6 months</time_frame>
    <description>patient characteristics are sex, patient's age at the start of treatment for secondary AML, date of birth, personal history of genetic predisposition (including brittle diseases - see below), family history of cancers (1st or 2nd degree)</description>
  </primary_outcome>
  <primary_outcome>
    <measure>potential clinical-biological prognostic factors - first cancer characteristics</measure>
    <time_frame>through study completion, an average of 6 months after patient inclusion</time_frame>
    <description>First cancer characteristics are age of onset, determined from the date of diagnosis, and histology, determined by anatomo-pathological diagnosis.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>potential clinical-biological prognostic factors :first cancer treatment : types of treatments received</measure>
    <time_frame>through study completion, an average of 6 months after patient inclusion</time_frame>
    <description>type of treatment: chemotherapy (Anthracyclines, Alkylating agents) and / or radiotherapy and/or Marrow autograft or allograft</description>
  </primary_outcome>
  <primary_outcome>
    <measure>potential clinical-biological prognostic factors - first cancer treatments: response to treatment</measure>
    <time_frame>through study completion, an average of 6 months after patient inclusion</time_frame>
    <description>response measured by bone marrow assessment using morphology and minimal residual disease (MRD) assessment : no response, partial response, complete remission</description>
  </primary_outcome>
  <primary_outcome>
    <measure>potential clinical-biological prognostic factors - first cancer treatments: Number of participants with treatment-related adverse events as assessed by CTCAE v4.0</measure>
    <time_frame>through study completion, an average of 6 months after patient inclusion</time_frame>
    <description>nature, incidence and severity of adverse events (AEs)</description>
  </primary_outcome>
  <primary_outcome>
    <measure>potential clinical-biological prognostic factors - first cancer treatments: duration of treatment</measure>
    <time_frame>through study completion, an average of 6 months after patient inclusion</time_frame>
    <description>duration of treatment determined from the start and end dates</description>
  </primary_outcome>
  <primary_outcome>
    <measure>potential clinical-biological prognostic factors - first cancer treatments: cumulative dose received</measure>
    <time_frame>through study completion, an average of 6 months after patient inclusion</time_frame>
    <description>cumulative dose of each type of treatment received ( chemotherapy (Anthracyclines, Alkylating agents) and / or radiotherapy and/or Marrow autograft or allograft)</description>
  </primary_outcome>
  <primary_outcome>
    <measure>potential clinical-biological prognostic factors - secondary AML characteristics: date of diagnosis</measure>
    <time_frame>through study completion, an average of 6 months after patient inclusion</time_frame>
    <description>date of diagnosis</description>
  </primary_outcome>
  <primary_outcome>
    <measure>potential clinical-biological prognostic factors - secondary AML characteristics: median time of onset compared to the date of end of treatment for the 1st cancer</measure>
    <time_frame>through study completion, an average of 6 months after patient inclusion</time_frame>
    <description>median time of onset compared to the date of end of treatment for the 1st cancer</description>
  </primary_outcome>
  <primary_outcome>
    <measure>potential clinical-biological prognostic factors - secondary AML characteristics : hematological data assessed by morphology</measure>
    <time_frame>through study completion, an average of 6 months after patient inclusion</time_frame>
    <description>Leukocytes at diagnosis of secondary AML (G/L)</description>
  </primary_outcome>
  <primary_outcome>
    <measure>potential clinical-biological prognostic factors - secondary AML characteristics: cytogenetic data</measure>
    <time_frame>through study completion, an average of 6 months after patient inclusion</time_frame>
    <description>karyotype, exclusive and cumulative anomalies</description>
  </primary_outcome>
  <primary_outcome>
    <measure>potential clinical-biological prognostic factors - secondary AML characteristics: molecular data</measure>
    <time_frame>through study completion, an average of 6 months after patient inclusion</time_frame>
    <description>molecular data assessed by Next-Generation Sequencing panel</description>
  </primary_outcome>
  <primary_outcome>
    <measure>potential clinical-biological prognostic factors - secondary AML treatment: duration of treatment</measure>
    <time_frame>through study completion, an average of 6 months after patient inclusion</time_frame>
    <description>duration of treatment determined from the start and end dates</description>
  </primary_outcome>
  <primary_outcome>
    <measure>potential clinical-biological prognostic factors - secondary AML treatment: type of treatment</measure>
    <time_frame>through study completion, an average of 6 months after patient inclusion</time_frame>
    <description>chemotherapy received and bone marrow transplantation</description>
  </primary_outcome>
  <primary_outcome>
    <measure>potential clinical-biological prognostic factors - secondary AML treatment : response to treatment</measure>
    <time_frame>through study completion, an average of 6 months after patient inclusion</time_frame>
    <description>response measured by bone marrow assessment using morphology and minimal residual disease (MRD) assessment : no response, partial response, complete remission</description>
  </primary_outcome>
  <primary_outcome>
    <measure>potential clinical-biological prognostic factors - secondary AML treatment: Number of participants with treatment-related adverse events as assessed by CTCAE v4.0</measure>
    <time_frame>through study completion, an average of 6 months after patient inclusion</time_frame>
    <description>nature, incidence and severity of adverse events (AEs)</description>
  </primary_outcome>
  <primary_outcome>
    <measure>potential clinical-biological prognostic factors - Overall Survival (OS)</measure>
    <time_frame>at the end of the 2 years of the inclusion period</time_frame>
    <description>Evaluated as time from diagnostic of the second AML to death from any cause or date last seen for patients who are alive at the end of the trial</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>feasibility of setting up a French national database of secondary AMLs for children and adolescents - Number of cases included over the period</measure>
    <time_frame>at the end of the 2 years of the inclusion period</time_frame>
    <description>Number of cases included over the period</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>feasibility of setting up a French national database of secondary AMLs for children and adolescents - Missing Data</measure>
    <time_frame>at the end of the 2 years of the inclusion period</time_frame>
    <description>Proportion and type of missing data in the database after the base freeze</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>feasibility of setting up a French national database of secondary AMLs for children and adolescents - Centre participation rates</measure>
    <time_frame>at the end of the 2 years of the inclusion period</time_frame>
    <description>Centre participation rates</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>association of potential prognostic factors with event free survival and the occurrence of treatment-related toxicities- Recurrence of the disease</measure>
    <time_frame>at the end of the 2 years of the inclusion period</time_frame>
    <description>Recurrence of the disease : Relapse criteria (in a patient who has had complete remission):&#xD;
≥ 5% blasts in the bone marrow (not attributable to post chemotherapy regeneration)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>association of potential prognostic factors with event free survival and the occurrence of treatment-related toxicities- Treatment-related toxicities</measure>
    <time_frame>at the end of the 2 years of the inclusion period</time_frame>
    <description>Treatment-related toxicities : Metabolic / endocrine complications in particular: growth retardation, hypothyroidism, gonadal insufficiency; Organic complications, in particular: renal failure, heart failure, respiratory failure ; Graft versus host reaction (GvH)</description>
  </secondary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Anticipated">40</enrollment>
  <condition>Leukemia, Myeloid, Acute</condition>
  <arm_group>
    <arm_group_label>Secondary Acute Myeloid Leukemias (AML) of the child</arm_group_label>
  </arm_group>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>collection of the clinical and biological characteristics of secondary AMLs in children</intervention_name>
    <description>to record scientific and medical information on cases of secondary Acute Myeloid Leukemias that have occurred in France since 2013</description>
    <arm_group_label>Secondary Acute Myeloid Leukemias (AML) of the child</arm_group_label>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        children and adolescents aged 0-18 years diagnosed with secondary AML&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Patients aged 0-18 years&#xD;
&#xD;
          -  Patient with first cancer&#xD;
&#xD;
          -  Diagnosis of secondary AML&#xD;
&#xD;
          -  Patients treated in a SFCE (Société française des cancers de l'enfant) center&#xD;
&#xD;
          -  For cases included in the prospective from March 2019 onwards: Consent of holders of&#xD;
             parental authority and consent of the child of understanding age&#xD;
&#xD;
        Exclusion Criteria: None&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>N/A</minimum_age>
    <maximum_age>18 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_contact>
    <last_name>Stéphane S DUCASSOU, MD PhD</last_name>
    <phone>05 57 82 04 38</phone>
    <email>stephane.ducassou@chu-bordeaux.fr</email>
  </overall_contact>
  <overall_contact_backup>
    <last_name>Aurore CAPELLI, PhD</last_name>
    <phone>05 57 82 08 77</phone>
    <email>aurore.capelli@chu-bordeaux.fr</email>
  </overall_contact_backup>
  <verification_date>June 2020</verification_date>
  <study_first_submitted>June 9, 2020</study_first_submitted>
  <study_first_submitted_qc>June 24, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">June 30, 2020</study_first_posted>
  <last_update_submitted>June 29, 2020</last_update_submitted>
  <last_update_submitted_qc>June 29, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">July 1, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Secondary</keyword>
  <keyword>Acute myeloid Leukemia</keyword>
  <keyword>Children</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Leukemia</mesh_term>
    <mesh_term>Leukemia, Myeloid</mesh_term>
    <mesh_term>Leukemia, Myeloid, Acute</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT04450784
org study id: CHUBX 2018/31
nct id: NCT04450784
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
Acute Myeloid Leukemias (AML) of the child are a rare disease and its prognosis varies
      according to the subgroup. Secondary AMLs remain a subgroup of poor prognosis, whose
      cytogenetic and molecular characteristics and prognostic value differ in part from de novo
      AMLs. The purpose of this national observatory is to record scientific and medical
      information on cases of secondary AML that have occurred in France since 2013 in order to
      improve the treatment of children and adolescents with this disease in the years to come.
      This national observatory will contribute to better knowledge and progress in research into
      these diseases.
Therapy-related myeloid neoplasms (t-MNs) are a group of hematologic diseases that arise
      after chemotherapy and/or radiation therapy for a previous cancer or rarely autoimmune
      diseases. t-MNs had been included in the group of AMLs and remain as a distinct category also
      in the recent 2016 revision of the WHO classification of myeloid neoplasm and acute leukemia.
      The latency between exposition to anticancer drugs and development of t-MN may vary from some
      months up to 10 years, even considering the age at diagnosis of the primary malignancy, the
      kind of cytotoxic treatment previously used, and the cumulative dose and dose intensity. The
      prognosis of s-AML is generally considered to be poorer than that of de novo AML. The disease
      tends to be refractory to chemotherapy, and patients' tolerance of treatment generally is
      reduced because of prior therapies. 5-year event-free survival (EFS) and overall survival
      (OS) rates of pediatric t-AML/t-MDS have been reported to be 14% to 30%. However, the results
      are conflicting and overall lacking when compared with those in adults. The purpose of this
      national observatory is to record scientific and medical information on cases of secondary
      Acute Myeloid Leukemias that have occurred in France since 2013 in order to improve the
      treatment of children and adolescents with this disease in the years to come. The primary
      objective is to evaluate the association of potential prognostic factors (including
      clinical-biological factors) with the overall survival of children and adolescents aged 0-18
      years diagnosed with secondary AML. The secondary objectives are to test the feasibility of
      setting up a French national database of secondary AMLs for children and adolescents as a
      first step towards European implementation, to assess the association of potential prognostic
      factors with event free survival and the occurrence of treatment-related toxicities, to
      characterize the molecular abnormalities associated with secondary AMLs in children and
      adolescents.
observational model: Cohort
time perspective: Other
measure: potential clinical-biological prognostic factors - patient characterics
time frame: through study completion, an average of 6 months
description: patient characteristics are sex, patient's age at the start of treatment for secondary AML, date of birth, personal history of genetic predisposition (including brittle diseases - see below), family history of cancers (1st or 2nd degree)
measure: potential clinical-biological prognostic factors - first cancer characteristics
time frame: through study completion, an average of 6 months after patient inclusion
description: First cancer characteristics are age of onset, determined from the date of diagnosis, and histology, determined by anatomo-pathological diagnosis.
measure: potential clinical-biological prognostic factors :first cancer treatment : types of treatments received
time frame: through study completion, an average of 6 months after patient inclusion
description: type of treatment: chemotherapy (Anthracyclines, Alkylating agents) and / or radiotherapy and/or Marrow autograft or allograft
measure: potential clinical-biological prognostic factors - first cancer treatments: response to treatment
time frame: through study completion, an average of 6 months after patient inclusion
description: response measured by bone marrow assessment using morphology and minimal residual disease (MRD) assessment : no response, partial response, complete remission
measure: potential clinical-biological prognostic factors - first cancer treatments: Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
time frame: through study completion, an average of 6 months after patient inclusion
description: nature, incidence and severity of adverse events (AEs)
measure: potential clinical-biological prognostic factors - first cancer treatments: duration of treatment
time frame: through study completion, an average of 6 months after patient inclusion
description: duration of treatment determined from the start and end dates
measure: potential clinical-biological prognostic factors - first cancer treatments: cumulative dose received
time frame: through study completion, an average of 6 months after patient inclusion
description: cumulative dose of each type of treatment received ( chemotherapy (Anthracyclines, Alkylating agents) and / or radiotherapy and/or Marrow autograft or allograft)
measure: potential clinical-biological prognostic factors - secondary AML characteristics: date of diagnosis
time frame: through study completion, an average of 6 months after patient inclusion
description: date of diagnosis
measure: potential clinical-biological prognostic factors - secondary AML characteristics: median time of onset compared to the date of end of treatment for the 1st cancer
time frame: through study completion, an average of 6 months after patient inclusion
description: median time of onset compared to the date of end of treatment for the 1st cancer
measure: potential clinical-biological prognostic factors - secondary AML characteristics : hematological data assessed by morphology
time frame: through study completion, an average of 6 months after patient inclusion
description: Leukocytes at diagnosis of secondary AML (G/L)
measure: potential clinical-biological prognostic factors - secondary AML characteristics: cytogenetic data
time frame: through study completion, an average of 6 months after patient inclusion
description: karyotype, exclusive and cumulative anomalies
measure: potential clinical-biological prognostic factors - secondary AML characteristics: molecular data
time frame: through study completion, an average of 6 months after patient inclusion
description: molecular data assessed by Next-Generation Sequencing panel
measure: potential clinical-biological prognostic factors - secondary AML treatment: duration of treatment
time frame: through study completion, an average of 6 months after patient inclusion
description: duration of treatment determined from the start and end dates
measure: potential clinical-biological prognostic factors - secondary AML treatment: type of treatment
time frame: through study completion, an average of 6 months after patient inclusion
description: chemotherapy received and bone marrow transplantation
measure: potential clinical-biological prognostic factors - secondary AML treatment : response to treatment
time frame: through study completion, an average of 6 months after patient inclusion
description: response measured by bone marrow assessment using morphology and minimal residual disease (MRD) assessment : no response, partial response, complete remission
measure: potential clinical-biological prognostic factors - secondary AML treatment: Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
time frame: through study completion, an average of 6 months after patient inclusion
description: nature, incidence and severity of adverse events (AEs)
measure: potential clinical-biological prognostic factors - Overall Survival (OS)
time frame: at the end of the 2 years of the inclusion period
description: Evaluated as time from diagnostic of the second AML to death from any cause or date last seen for patients who are alive at the end of the trial
measure: feasibility of setting up a French national database of secondary AMLs for children and adolescents - Number of cases included over the period
time frame: at the end of the 2 years of the inclusion period
description: Number of cases included over the period
measure: feasibility of setting up a French national database of secondary AMLs for children and adolescents - Missing Data
time frame: at the end of the 2 years of the inclusion period
description: Proportion and type of missing data in the database after the base freeze
measure: feasibility of setting up a French national database of secondary AMLs for children and adolescents - Centre participation rates
time frame: at the end of the 2 years of the inclusion period
description: Centre participation rates
measure: association of potential prognostic factors with event free survival and the occurrence of treatment-related toxicities- Recurrence of the disease
time frame: at the end of the 2 years of the inclusion period
description: Recurrence of the disease : Relapse criteria (in a patient who has had complete remission):
≥ 5% blasts in the bone marrow (not attributable to post chemotherapy regeneration)
measure: association of potential prognostic factors with event free survival and the occurrence of treatment-related toxicities- Treatment-related toxicities
time frame: at the end of the 2 years of the inclusion period
description: Treatment-related toxicities : Metabolic / endocrine complications in particular: growth retardation, hypothyroidism, gonadal insufficiency; Organic complications, in particular: renal failure, heart failure, respiratory failure ; Graft versus host reaction (GvH)
arm group label: Secondary Acute Myeloid Leukemias (AML) of the child
intervention type: Other
intervention name: collection of the clinical and biological characteristics of secondary AMLs in children
description: to record scientific and medical information on cases of secondary Acute Myeloid Leukemias that have occurred in France since 2013
arm group label: Secondary Acute Myeloid Leukemias (AML) of the child
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: N/A
maximum age: 18 Years
healthy volunteers: No
last name: Stéphane S DUCASSOU, MD PhD
phone: 05 57 82 04 38
email: stephane.ducassou@chu-bordeaux.fr
last name: Aurore CAPELLI, PhD
phone: 05 57 82 08 77
email: aurore.capelli@chu-bordeaux.fr
responsible party type: Sponsor
mesh term: Leukemia
mesh term: Leukemia, Myeloid
mesh term: Leukemia, Myeloid, Acute
sharing ipd: No