<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/NCT02257905</url>
  </required_header>
  <id_info>
    <org_study_id>42206633</org_study_id>
    <nct_id>NCT02257905</nct_id>
  </id_info>
  <brief_title>Allo SCT in Amyloidosis Non-interventional Study</brief_title>
  <official_title>A Non-interventional Prospective Study on Allogeneic Stem Cell Transplantation in Patients With AL Amyloidosis</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>European Society for Blood and Marrow Transplantation</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>European Society for Blood and Marrow Transplantation</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      Light-chain (AL-) amyloidosis is a very rare monoclonal plasma cell disorder with poor&#xD;
      prognosis. Rarity of disease has precluded performance of randomized controlled trials&#xD;
      comparing various possible treatment modalities. In general, treatment of AL amyloidosis has&#xD;
      been adapted from myeloma (MM) therapy. There is large experience with allo SCT in MM. Based&#xD;
      on small series of patients and case reports allogeneic transplant has emerged as potentially&#xD;
      effective. However, more formal proof of concept of using allogeneic hematopoietic&#xD;
      transplantation for treatment of AL Amyloidosis is lacking.&#xD;
&#xD;
      Therefore, given the limitations of conventionally collected registry data (dubious follow-up&#xD;
      information and extreme heterogeneity), we developed the: &quot;EBMT non-interventional&#xD;
      prospective study on allogeneic transplantation in AL Amyloidosis&quot; which means that&#xD;
      transplant centers that already do perform allogeneic transplants for AL Amyloidosis will be&#xD;
      encouraged to register their patients with AL Amyloidosis very timely with the EBMT, followed&#xD;
      by mandatory submission of EBMT MedB and follow-up forms. The diagnosis of AL Amyloidosis&#xD;
      would be based on uniform criteria. All EBMT centres performing allogeneic transplants for&#xD;
      Amyloidosis will be invited to participate in this study and centres will be asked to report&#xD;
      all AL Amyloidosis cases referred for transplantation using a simple registration form and&#xD;
      then to submit Amyloidosis MED B forms for each transplanted patient and follow-up forms as&#xD;
      necessary. In conclusion, it should be possible to largely improve the usual quality of&#xD;
      registry-based data and to generate scientifically sound knowledge on HSCT in an orphan&#xD;
      disease such as AL Amyloidosis.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Short description of the study:&#xD;
&#xD;
      We plan to select those centres within the EBMT that in the past have performed any&#xD;
      allogeneic transplant for amyloidosis and ask them to participate by completing a centre&#xD;
      registration form. When they have a patient they want to include in this study, the centre&#xD;
      will notify the CLWP data office of the planned allogeneic transplant the day before the&#xD;
      transplant takes place. For this, the centre can use a patient registration form. Once a&#xD;
      patient is included, MED B and regular follow-up are mandatory. Data entry of MED B forms and&#xD;
      follow up will be performed in the way that is usual for the centre (either by the centre&#xD;
      itself, by the national registry or by the Paris data office). The CLWP data office will keep&#xD;
      track of the data submitted.&#xD;
&#xD;
      Research design:&#xD;
&#xD;
      This study is designed as a non-interventional prospective study.&#xD;
&#xD;
      Study Population:&#xD;
&#xD;
      Male or female subjects, 18 years to 60 years, with AL amyloidosis who will receive&#xD;
      allogeneic transplantation.&#xD;
&#xD;
      Data to be collected/analysed:&#xD;
&#xD;
      The primary endpoint of this study is efficacy (best hematological remission and organ&#xD;
      response) of allogeneic SCT in patients with AL amyloidosis. The secondary endpoints are&#xD;
      acute and chronic GvHD, TRM and event-free and overall survival.&#xD;
&#xD;
      Purpose of the non-interventional prospective study request:&#xD;
&#xD;
      The main purpose of this non-interventional prospective study is the evaluation of the&#xD;
      effectiveness of allogeneic transplant for AL Amyloidosis. It is planned that results will be&#xD;
      presented on scientific symposia and that they will suffice for at least one publication&#xD;
      (original article).&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date>December 2009</start_date>
  <primary_completion_date type="Actual">July 2015</primary_completion_date>
  <study_type>Observational [Patient Registry]</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>
  <target_duration>5 Years</target_duration>
  <primary_outcome>
    <measure>efficacy of allo sct in patients with AL Amyloidosis</measure>
    <time_frame>5 years</time_frame>
    <description>• To evaluate the efficacy of allogeneic SCT in patients with AL Amyloidosis (best hematological remission and organ response).</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>EFS</measure>
    <time_frame>5 years</time_frame>
    <description>• To evaluate event-free survival.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>OS</measure>
    <time_frame>5 years</time_frame>
    <description>to evaluate overall survival</description>
  </secondary_outcome>
  <other_outcome>
    <measure>safety of allo sct in patients with AL Amyloidosis</measure>
    <time_frame>5 years</time_frame>
    <description>• To evaluate safety of allogeneic SCT in patients with AL Amyloidosis (TRM, incidence of acute and chronic GvHD, non-haematological toxicity and engraftment).</description>
  </other_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Actual">14</enrollment>
  <condition>AL Amyloidosis</condition>
  <arm_group>
    <arm_group_label>AL Amyloidosis patients who received allo HSCT</arm_group_label>
  </arm_group>
  <eligibility>
    <study_pop>
      <textblock>
        Male or female subjects, 18 years to 60 years, with AL amyloidosis who will receive&#xD;
        allogeneic transplantation&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  AL Amyloidosis, allogeneic HSCT after November 2009, between 18-60 years at time of&#xD;
             transplant.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  other diagnoses than AL Amyloidosis, auto transplant, transplant before November 2009,&#xD;
             younger than 18 or older than 60 at time of transplant.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>60 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Stefan Schoenland, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Medizinische Klinik, University of Heidelberg, Germany</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Nicolaus Kroeger, MD</last_name>
    <role>Study Chair</role>
    <affiliation>BMT Centre, University Hospital Eppendorf, Hamburg, Germany</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>CHRU Limoges</name>
      <address>
        <city>Limoges</city>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University Hospital Eppendorf</name>
      <address>
        <city>Hamburg</city>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Heidelberg</name>
      <address>
        <city>Heidelberg</city>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University Hospital</name>
      <address>
        <city>Basel</city>
        <country>Switzerland</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>France</country>
    <country>Germany</country>
    <country>Switzerland</country>
  </location_countries>
  <reference>
    <citation>Schönland et al. EBMT retrospective data, Blood 2005.</citation>
  </reference>
  <reference>
    <citation>Schönland et al. DLI data, Haematologica, 2008.</citation>
  </reference>
  <verification_date>July 2015</verification_date>
  <study_first_submitted>October 2, 2014</study_first_submitted>
  <study_first_submitted_qc>October 6, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">October 7, 2014</study_first_posted>
  <last_update_submitted>July 23, 2015</last_update_submitted>
  <last_update_submitted_qc>July 23, 2015</last_update_submitted_qc>
  <last_update_posted type="Estimate">July 24, 2015</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>AL Amyloidosis</keyword>
  <keyword>Amyloidosis</keyword>
  <keyword>allogeneic</keyword>
  <keyword>HSCT</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Immunoglobulin Light-chain Amyloidosis</mesh_term>
    <mesh_term>Amyloidosis</mesh_term>
  </condition_browse>
  <!-- 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/NCT02257905
org study id: 42206633
nct id: NCT02257905
lead sponsor: 
has dmc: No
Light-chain (AL-) amyloidosis is a very rare monoclonal plasma cell disorder with poor
      prognosis. Rarity of disease has precluded performance of randomized controlled trials
      comparing various possible treatment modalities. In general, treatment of AL amyloidosis has
      been adapted from myeloma (MM) therapy. There is large experience with allo SCT in MM. Based
      on small series of patients and case reports allogeneic transplant has emerged as potentially
      effective. However, more formal proof of concept of using allogeneic hematopoietic
      transplantation for treatment of AL Amyloidosis is lacking.

      Therefore, given the limitations of conventionally collected registry data (dubious follow-up
      information and extreme heterogeneity), we developed the: "EBMT non-interventional
      prospective study on allogeneic transplantation in AL Amyloidosis" which means that
      transplant centers that already do perform allogeneic transplants for AL Amyloidosis will be
      encouraged to register their patients with AL Amyloidosis very timely with the EBMT, followed
      by mandatory submission of EBMT MedB and follow-up forms. The diagnosis of AL Amyloidosis
      would be based on uniform criteria. All EBMT centres performing allogeneic transplants for
      Amyloidosis will be invited to participate in this study and centres will be asked to report
      all AL Amyloidosis cases referred for transplantation using a simple registration form and
      then to submit Amyloidosis MED B forms for each transplanted patient and follow-up forms as
      necessary. In conclusion, it should be possible to largely improve the usual quality of
      registry-based data and to generate scientifically sound knowledge on HSCT in an orphan
      disease such as AL Amyloidosis.
Short description of the study:

      We plan to select those centres within the EBMT that in the past have performed any
      allogeneic transplant for amyloidosis and ask them to participate by completing a centre
      registration form. When they have a patient they want to include in this study, the centre
      will notify the CLWP data office of the planned allogeneic transplant the day before the
      transplant takes place. For this, the centre can use a patient registration form. Once a
      patient is included, MED B and regular follow-up are mandatory. Data entry of MED B forms and
      follow up will be performed in the way that is usual for the centre (either by the centre
      itself, by the national registry or by the Paris data office). The CLWP data office will keep
      track of the data submitted.

      Research design:

      This study is designed as a non-interventional prospective study.

      Study Population:

      Male or female subjects, 18 years to 60 years, with AL amyloidosis who will receive
      allogeneic transplantation.

      Data to be collected/analysed:

      The primary endpoint of this study is efficacy (best hematological remission and organ
      response) of allogeneic SCT in patients with AL amyloidosis. The secondary endpoints are
      acute and chronic GvHD, TRM and event-free and overall survival.

      Purpose of the non-interventional prospective study request:

      The main purpose of this non-interventional prospective study is the evaluation of the
      effectiveness of allogeneic transplant for AL Amyloidosis. It is planned that results will be
      presented on scientific symposia and that they will suffice for at least one publication
      (original article).
observational model: Cohort
time perspective: Prospective
measure: efficacy of allo sct in patients with AL Amyloidosis
time frame: 5 years
description: • To evaluate the efficacy of allogeneic SCT in patients with AL Amyloidosis (best hematological remission and organ response).
measure: EFS
time frame: 5 years
description: • To evaluate event-free survival.
measure: OS
time frame: 5 years
description: to evaluate overall survival
measure: safety of allo sct in patients with AL Amyloidosis
time frame: 5 years
description: • To evaluate safety of allogeneic SCT in patients with AL Amyloidosis (TRM, incidence of acute and chronic GvHD, non-haematological toxicity and engraftment).
arm group label: AL Amyloidosis patients who received allo HSCT
study pop: 
sampling method: Probability Sample
criteria: 
gender: All
minimum age: 18 Years
maximum age: 60 Years
healthy volunteers: No
last name: Stefan Schoenland, MD
role: Principal Investigator
affiliation: Medizinische Klinik, University of Heidelberg, Germany
last name: Nicolaus Kroeger, MD
role: Study Chair
affiliation: BMT Centre, University Hospital Eppendorf, Hamburg, Germany
facility: 
facility: 
facility: 
facility: 
country: France
country: Germany
country: Switzerland
citation: Schönland et al. EBMT retrospective data, Blood 2005.
citation: Schönland et al. DLI data, Haematologica, 2008.
responsible party type: Sponsor
mesh term: Immunoglobulin Light-chain Amyloidosis
mesh term: Amyloidosis