<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/NCT04477681</url>
  </required_header>
  <id_info>
    <org_study_id>2019-A01317-50</org_study_id>
    <secondary_id>2019/2848</secondary_id>
    <nct_id>NCT04477681</nct_id>
  </id_info>
  <brief_title>Securing Access to Innovative Molecules in Oncology and Hematology for Children, Adolescents and Young Adults</brief_title>
  <acronym>SACHA</acronym>
  <official_title>Securing Access to Innovative Molecules in Oncology and Hematology for Children, Adolescents and Young Adults in Therapeutic Failure or Relapse and Not Eligible for a Clinical Trial: a Project of the SFCE</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Gustave Roussy, Cancer Campus, Grand Paris</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Gustave Roussy, Cancer Campus, Grand Paris</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>
      It involves collecting safety and efficacy data, under the actual conditions of use of&#xD;
      medicines in children and adolescents, using a validated tool (Ennov EDC) and relying on the&#xD;
      network of Interregional pediatric oncology appeal organizations (OIR) identified by INCa&#xD;
      since 2010 and responsible for the organization of Pluridisciplinary Pediatric Interregional&#xD;
      (RCPPI) and National Consultative Meetings which discuss each case of relapse in order to&#xD;
      define the best therapeutic options.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      The objective is to aim for completeness throughout the national territory. This is why this&#xD;
      project will be carried out in close collaboration with the RIOs and RCPPI and that of the 31&#xD;
      centers of the SFCE which bring together more than 400 doctors pediatric oncologists and&#xD;
      hematologists on the French territory&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">April 9, 2020</start_date>
  <completion_date type="Anticipated">April 2026</completion_date>
  <primary_completion_date type="Anticipated">April 2025</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>Prospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Collect access data</measure>
    <time_frame>up to 1 year</time_frame>
    <description>Collect access data of children, adolescents and young adults in therapeutic failures and not eligible for a clinical trial with innovative molecules, whether they are targeted therapies, immunotherapies or chemotherapies</description>
  </primary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Anticipated">750</enrollment>
  <condition>Pediatric Cancer</condition>
  <arm_group>
    <arm_group_label>Patients in therapeutic failure or relapse</arm_group_label>
    <description>Any child, adolescent or young adult, treated for a pediatric tumor or leukemia, in therapeutic failure or relapse without standard treatment option, not eligible / refusal of inclusion in a clinical study open on the territory and treated with an innovative drug within the framework of an ATU or outside AMM, in one of the centers of the SFCE (Société Française Cancer Enfant)</description>
  </arm_group>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Data collection</intervention_name>
    <description>In particular, the patient's demographic data, medical history, previous and concomitant treatments, data on the treatment of interest (legal framework, ATU / off-label), dosage, start date, etc.), clinical data will be collected. biological (including molecular profiling of the tumor if available) and radiological follow-up, information on adverse effects. The data to be filled in is that of the CERFA pharmacovigilance form of the ANSM, but additional fields can be created if necessary.</description>
    <arm_group_label>Patients in therapeutic failure or relapse</arm_group_label>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        Any child, adolescent or young adult, treated for a pediatric tumor or leukemia, in&#xD;
        therapeutic failure or relapse without standard treatment option, not eligible / refusal of&#xD;
        inclusion in a clinical study open on the territory and treated with an innovative drug&#xD;
        within the framework of an ATU or outside AMM, in one of the centers of the SFCE (Société&#xD;
        Française Cancer Enfant).&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Age ≤ 25 years old at the time of inclusion in the study&#xD;
&#xD;
          -  Patient with a pediatric tumor or leukemia in therapeutic failure or relapse without&#xD;
             standard therapeutic options. Exceptionally, patients on the first line of treatment&#xD;
             without standard therapeutic options may also be included (eg g: inoperable plexiform&#xD;
             neurofibroma and MEK inhibitors, childhood fibrosarcoma and NTRK inhibitors).&#xD;
&#xD;
          -  Patient not eligible for an early phase clinical trial open to inclusion on French&#xD;
             territory or refusal of inclusion&#xD;
&#xD;
          -  Patient treated with a new drug discussed at an ICSID as part of an ATU (nominative or&#xD;
             cohort) issued by the ANSM or an off-label prescription of a drug of interest already&#xD;
             authorized in adults.&#xD;
&#xD;
          -  Patients treated in one of the SFCE centers authorized to prescribe chemotherapy&#xD;
&#xD;
          -  Patient who did not object to participate after being informed of the study. The&#xD;
             patient must be able and willing to cooperate in the study.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Patient included in an early phase clinical trial open to inclusions on French&#xD;
             territory.&#xD;
&#xD;
          -  Oral refusal to participate by the patient or their legal representatives, in reading&#xD;
             the information note specific to the study&#xD;
&#xD;
          -  Patient under guardianship or curatorship, deprived of liberty or in the impossibility&#xD;
             of expressing opposition to participating in the study&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>N/A</minimum_age>
    <maximum_age>25 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_contact>
    <last_name>Pablo BERLANGA, MD</last_name>
    <phone>+33 (0)1 42 11 42 11</phone>
    <email>pablo.berlanga@gustaveroussy.fr</email>
  </overall_contact>
  <overall_contact_backup>
    <last_name>Lee Aymar NDOUNGA DIAKOU, PhD</last_name>
    <phone>+33 (0)1 42 11 42 11</phone>
    <email>Leeaymar.NDOUNGA-DIAKOU@gustaveroussy.fr</email>
  </overall_contact_backup>
  <location>
    <facility>
      <name>Gustave Roussy</name>
      <address>
        <city>Villejuif</city>
        <state>Val De Marne</state>
        <zip>94805</zip>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Pablo BERLANGA, MD</last_name>
      <phone>+33 (0)1 42 11 42 11</phone>
      <email>pablo.berlanga@gustaveroussy.fr</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Amiens</name>
      <address>
        <city>Amiens</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Leslie ANDRY, MD</last_name>
      <email>Andry.Leslie@chu-amiens.fr</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Angers</name>
      <address>
        <city>Angers</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Emilie DE CARLI, MD</last_name>
      <email>EmDeCarli@chu-angers.fr</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Besançon</name>
      <address>
        <city>Besançon</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Sébastien KLEIN, MD</last_name>
      <email>s1klein@chu-besancon.fr</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Bordeaux</name>
      <address>
        <city>Bordeaux</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Stéphane DUCASSOU, MD</last_name>
      <email>stephane.ducassou@chu-bordeaux.fr</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU CAEN</name>
      <address>
        <city>Caen</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Damien BODET, MD</last_name>
      <email>bodet-d@chu-caen.fr</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Clermont-Ferrand</name>
      <address>
        <city>Clermont-Ferrand</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Justyna KANOLD</last_name>
      <email>jkanold@chu-clermontferrand.fr</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Dijon</name>
      <address>
        <city>Dijon</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Claire BRIANDET, MD</last_name>
      <email>claire.briandet@chu-dijon.fr</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Grenoble</name>
      <address>
        <city>Grenoble</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Dominique PLANTAZ, MD</last_name>
      <email>Dplantaz@chu-grenoble.fr</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>Centre Oscar Lambret</name>
      <address>
        <city>Lille</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Sandra RAIMBAULT, MD</last_name>
      <email>s-raimbault@o-lambret.fr</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Lille</name>
      <address>
        <city>Lille</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Brigitte NELKEN, MD</last_name>
      <email>brigitte.nelken@chru-lille.fr</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Limoges</name>
      <address>
        <city>Limoges</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Christophe PIGUET, MD</last_name>
      <email>Christophe.Piguet@chu-limoges.fr</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>Centre Léon Bérard</name>
      <address>
        <city>Lyon</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Nadège CORRADINI, MD</last_name>
      <email>Nadege.CORRADINI@ihope.fr</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Lyon</name>
      <address>
        <city>Lyon</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Carine HALFON-DOMENECH, MD</last_name>
      <email>carine.halfondomenech@ihope.fr</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>Hôpital de La Timone</name>
      <address>
        <city>Marseille</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Nicolas ANDRE, MD</last_name>
      <email>Nicolas.ANDRE@ap-hm.fr</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Montpellier</name>
      <address>
        <city>Montpellier</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Laure SAUMET, MD</last_name>
      <email>l-saumet@chu-montpellier.fr</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Nancy</name>
      <address>
        <city>Nancy</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Pascal CHASTAGNER, MD</last_name>
      <email>p.chastagner@chru-nancy.fr</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Nantes</name>
      <address>
        <city>Nantes</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Morgane CLEIREC, MD</last_name>
      <email>morgane.cleirec@chu-nantes.fr</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Nice</name>
      <address>
        <city>Nice</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Joy BENADIBA, MD</last_name>
      <email>benadiba.j@chu-nice.fr</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>Hôpital Armand Trousseau</name>
      <address>
        <city>Paris</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Arnaud PETIT, MD</last_name>
      <email>arnaud.petit@aphp.fr</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>Hôpital Robert-Debré</name>
      <address>
        <city>Paris</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Marion STRULLU, MD</last_name>
      <email>marion.strullu@aphp.fr</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>Institut Curie</name>
      <address>
        <city>Paris</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Isabelle AERTS, MD</last_name>
      <email>isabelle.aerts@curie.fr</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Poitiers</name>
      <address>
        <city>Poitiers</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Frederic MILLOT, MD</last_name>
      <email>f.millot@chu-poitiers.fr</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Reims</name>
      <address>
        <city>Reims</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Claire PUCHART, MD</last_name>
      <email>cpluchart@chu-reims.fr</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Rennes</name>
      <address>
        <city>Rennes</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Chloé PUISIEUX, MD</last_name>
      <email>Chloe.PUISEUX@chu-rennes.fr</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Rouen</name>
      <address>
        <city>Rouen</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Pascal SCHNEIDER</last_name>
      <email>Pascale.Schneider@chu-rouen.fr</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU La Réunion</name>
      <address>
        <city>Saint-Denis</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Yves REGUERRE, MD</last_name>
      <email>yves.reguerre@chu-reunion.fr</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Saint-Etienne</name>
      <address>
        <city>Saint-Étienne</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Sandrine THOUVENIN, MD</last_name>
      <email>sandrine.thouvenin@chu-st-etienne.fr</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Strasbourg</name>
      <address>
        <city>Strasbourg</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Natacha ENTZ-WERLE</last_name>
      <email>Natacha.Entz-Werle@chru-strasbourg.fr</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Toulouse</name>
      <address>
        <city>Toulouse</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Marion Gambart, MD</last_name>
      <email>gambart.m@chu-toulouse.fr</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>CHU Tours</name>
      <address>
        <city>Tours</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Jill SERRE, MD</last_name>
      <email>J.SERRE@chu-tours.fr</email>
    </contact>
  </location>
  <location_countries>
    <country>France</country>
  </location_countries>
  <verification_date>June 2023</verification_date>
  <study_first_submitted>July 15, 2020</study_first_submitted>
  <study_first_submitted_qc>July 15, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">July 20, 2020</study_first_posted>
  <last_update_submitted>June 2, 2023</last_update_submitted>
  <last_update_submitted_qc>June 2, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">June 6, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <patient_data>
    <sharing_ipd>Undecided</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/NCT04477681
org study id: 2019-A01317-50
secondary id: 2019/2848
nct id: NCT04477681
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
It involves collecting safety and efficacy data, under the actual conditions of use of
      medicines in children and adolescents, using a validated tool (Ennov EDC) and relying on the
      network of Interregional pediatric oncology appeal organizations (OIR) identified by INCa
      since 2010 and responsible for the organization of Pluridisciplinary Pediatric Interregional
      (RCPPI) and National Consultative Meetings which discuss each case of relapse in order to
      define the best therapeutic options.
The objective is to aim for completeness throughout the national territory. This is why this
      project will be carried out in close collaboration with the RIOs and RCPPI and that of the 31
      centers of the SFCE which bring together more than 400 doctors pediatric oncologists and
      hematologists on the French territory
observational model: Cohort
time perspective: Prospective
measure: Collect access data
time frame: up to 1 year
description: Collect access data of children, adolescents and young adults in therapeutic failures and not eligible for a clinical trial with innovative molecules, whether they are targeted therapies, immunotherapies or chemotherapies
arm group label: Patients in therapeutic failure or relapse
description: Any child, adolescent or young adult, treated for a pediatric tumor or leukemia, in therapeutic failure or relapse without standard treatment option, not eligible / refusal of inclusion in a clinical study open on the territory and treated with an innovative drug within the framework of an ATU or outside AMM, in one of the centers of the SFCE (Société Française Cancer Enfant)
intervention type: Other
intervention name: Data collection
description: In particular, the patient's demographic data, medical history, previous and concomitant treatments, data on the treatment of interest (legal framework, ATU / off-label), dosage, start date, etc.), clinical data will be collected. biological (including molecular profiling of the tumor if available) and radiological follow-up, information on adverse effects. The data to be filled in is that of the CERFA pharmacovigilance form of the ANSM, but additional fields can be created if necessary.
arm group label: Patients in therapeutic failure or relapse
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: N/A
maximum age: 25 Years
healthy volunteers: No
last name: Pablo BERLANGA, MD
phone: +33 (0)1 42 11 42 11
email: pablo.berlanga@gustaveroussy.fr
last name: Lee Aymar NDOUNGA DIAKOU, PhD
phone: +33 (0)1 42 11 42 11
email: Leeaymar.NDOUNGA-DIAKOU@gustaveroussy.fr
facility: 
status: Recruiting
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contact: 
facility: 
status: Recruiting
contact: 
facility: 
status: Recruiting
contact: 
country: France
responsible party type: Sponsor
sharing ipd: Undecided