<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/NCT04438889</url>
  </required_header>
  <id_info>
    <org_study_id>AGMT_aMYELOIDr</org_study_id>
    <nct_id>NCT04438889</nct_id>
  </id_info>
  <brief_title>Austrian Myeloid Registry</brief_title>
  <acronym>aMYELOIDr</acronym>
  <official_title>Austrian Myeloid Registry</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Arbeitsgemeinschaft medikamentoese Tumortherapie</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Arbeitsgemeinschaft medikamentoese Tumortherapie</source>
  <oversight_info>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      The Austrian Myeloid Registry (aMYELOIDr) is a non-interventional study. It collects data&#xD;
      from patients with the myeloid diseases, primarily myelodysplastic syndromes (MDS), chronic&#xD;
      myelomonocytic leukemia (CMML) and acute myeloid leukemia (AML).The aMYELOIDr is multi-center&#xD;
      database collecting data at various sites in Austria and potentially also at other centers in&#xD;
      other countries in future. The registry has an electronic case report form (eCRF), where all&#xD;
      data is entered by clinical trial personnel and/or physicians. It is set up to collect&#xD;
      real-world experience in the management of patients with these diseases in Austria.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">July 13, 2020</start_date>
  <completion_date type="Anticipated">April 2030</completion_date>
  <primary_completion_date type="Anticipated">April 2029</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>10 Years</target_duration>
  <primary_outcome>
    <measure>To assess the treatment patterns (therapeutic landscape) of patients with myeloid diseases.</measure>
    <time_frame>Through study completion, median expected within 100 months</time_frame>
    <description>Due to the non-interventional nature of the aMYELOIDr, treatment indication, the decision to offer treatment, treatment choice, dose, schedule and dose reductions/escalations shall be exclusively based on the risk/benefit estimation of the treating physician. We recommend compliance current guidelines.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Impact of front-line treatment on overall survival (OS)</measure>
    <time_frame>Through study completion, median expected within 100 months</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Impact of number and choice of treatment lines on OS as of initial diagnosis and/or as of treatment start</measure>
    <time_frame>Through study completion, median expected within 100 months</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Overall response rate (ORR)</measure>
    <time_frame>Through study completion, median expected within 100 months</time_frame>
    <description>Response will be assessed according to current guidelines for the respective disease. Due to the non-interventional nature of the aMYELOIDr, timepoints and types of response assessment shall be exclusively based on the risk/benefit estimation of the treating physician. We recommend compliance current guidelines.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Event free survival (EFS)</measure>
    <time_frame>Through study completion, median expected within 100 months</time_frame>
    <description>Events include treatment failure, progressive disease, relapse after CR/CRi, death from any cause. Patients lost to-follow-up or still alive ans without event will be censored at last follow-up date</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>AML transformation</measure>
    <time_frame>Through study completion, median expected within 100 months</time_frame>
    <description>Time to transformation to AML for patients with a non-AML initial diagnosis</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Treatment safety</measure>
    <time_frame>Through study completion, median expected within 100 months</time_frame>
    <description>Investigators should report adverse reactions (for which a causal role of a medicine is suspected) to the concerned competent authorities following regulations in the current or future versions of Austrian legislation (Pharmakovigilanz-Verordnung 2013 (PhVO, Regulation on Pharmacovigilance), Österreichisches Arzneimittel Gesetz (AMG, Austrian Medicinal Products Act). Participation in this registry does not exempt the participating center from their legal reporting obligations. Documentation of causality, duration, frequency and severity of adverse events (AEs) according to Common Terminology Criteria for AE (CTCAE v.5).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concomitant treatments</measure>
    <time_frame>Through study completion, median expected within 100 months</time_frame>
    <description>Concomitant treatments (number of administration of e.g. prophylactic antibiotics, antivirals, antifungals)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Treatment characteristics</measure>
    <time_frame>Through study completion, median expected within 100 months</time_frame>
    <description>Among others, the following treatment characteristics will be assessed for each treatment line: substance</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Treatment characteristics</measure>
    <time_frame>Through study completion, median expected within 100 months</time_frame>
    <description>Among others, the following treatment characteristics will be assessed for each treatment line: application date</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Treatment characteristics</measure>
    <time_frame>Through study completion, median expected within 100 months</time_frame>
    <description>Among others, the following treatment characteristics will be assessed for each treatment line: route</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Treatment characteristics</measure>
    <time_frame>Through study completion, median expected within 100 months</time_frame>
    <description>Among others, the following treatment characteristics will be assessed for each treatment line: dose</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Treatment characteristics</measure>
    <time_frame>Through study completion, median expected within 100 months</time_frame>
    <description>Among others, the following treatment characteristics will be assessed for each treatment line: inpatient or outpatient setting</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concomitant treatments best supportive care (BSC)</measure>
    <time_frame>Through study completion, median expected within 100 months</time_frame>
    <description>Concomitant best supportive care (BSC) measures (e.g. number of transfusions, growth factors, iron chelators)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Quality of life assessment EQ-5D-5L (optional)</measure>
    <time_frame>Through study completion, median expected within 100 months</time_frame>
    <description>EuroQol-5 Dimensions with 5 Levels. The descriptive system comprises five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 5 levels: no problems, slight problems, moderate problems, severe problems and extreme problems. The patient is asked to indicate his/her health state by ticking the box next to the most appropriate statement in each of the five dimensions.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>EORTC Quality of life assessment QLQ-C30 (optional)</measure>
    <time_frame>Through study completion, median expected within 100 months</time_frame>
    <description>European Organisation for Research and Treatment of Cancer Quality of life 30-item questionnaire of cancer patients incorporates five functional scales (physical, role, cognitive, emotional, and social), three symptom scales (fatigue, pain, and nausea and vomiting), a global health status / QoL scale, and a number of single items assessing additional symptoms commonly reported by cancer patients (dyspnoea, loss of appetite, insomnia, constipation anddiarrhoea) and perceived financial impact of the disease. It has four-point scale which are coded with &quot;Not at all&quot;, &quot;A little&quot;, &quot;Quite a bit&quot; and &quot;Very much&quot;</description>
  </secondary_outcome>
  <other_outcome>
    <measure>Minimal residual disease</measure>
    <time_frame>Through study completion, median expected within 100 months</time_frame>
    <description>Assess how many and which patients in Austria have minimal residual disease (MRD) assessments, assessed by which techniques and at which timepoints.</description>
  </other_outcome>
  <other_outcome>
    <measure>MRD-negativity on OS</measure>
    <time_frame>Through study completion, median expected within 100 months</time_frame>
    <description>Assess the impact of MRD-negativity on OS. MRD assessment via flow-cytometry and polymerase chain reactions according to the current ELN guidelines 2017 shall be exclusively based on the risk/benefit estimation of the treating physician.</description>
  </other_outcome>
  <other_outcome>
    <measure>Prognostic and predictive markers</measure>
    <time_frame>Through study completion, median expected within 100 months</time_frame>
    <description>Analyses of various factors known or thought to influence OS, ORR, EFS, PFS and time to next treatment in order to validate existing or establish novel prognostic and predictive markers</description>
  </other_outcome>
  <number_of_groups>4</number_of_groups>
  <enrollment type="Anticipated">3000</enrollment>
  <condition>Myeloid Diseases</condition>
  <arm_group>
    <arm_group_label>AML</arm_group_label>
    <description>Patients with WHO 2016 diagnosis of AML</description>
  </arm_group>
  <arm_group>
    <arm_group_label>MDS</arm_group_label>
    <description>Patients with WHO 2016 diagnosis of MDS</description>
  </arm_group>
  <arm_group>
    <arm_group_label>CMML</arm_group_label>
    <description>Patients with WHO 2016 diagnosis of CMML</description>
  </arm_group>
  <arm_group>
    <arm_group_label>PMF</arm_group_label>
    <description>Patients with WHO 2016 diagnosis of PMF</description>
  </arm_group>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Non-interventional</intervention_name>
    <description>Only routine clinical data, which has already been recorded in the patient's medical chart, will be documented. Any other data assessments (e.g. quality of life analyses such as EQ-5D and QLQ-C30 have been approved by the Ehtikkommission für das Bundesland Salzburg and are optional.</description>
    <arm_group_label>AML</arm_group_label>
    <arm_group_label>CMML</arm_group_label>
    <arm_group_label>MDS</arm_group_label>
    <arm_group_label>PMF</arm_group_label>
  </intervention>
  <biospec_retention>Samples With DNA</biospec_retention>
  <biospec_descr>
    <textblock>
      The aMYELOIDr is accompanied by an optional biobanking program. Sample collection will be&#xD;
      limited to patients that have signed an additional biobanking IC.&#xD;
    </textblock>
  </biospec_descr>
  <eligibility>
    <study_pop>
      <textblock>
        Patients with AML, MDS, CMML or PMF according to WHO 2016 diagnostic criteria&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Age &gt;17 years&#xD;
&#xD;
          -  Diagnosis of myeloid disease according to WHO 2016&#xD;
&#xD;
          -  Signed patient informed consent (IC)&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Patient is unable or unwilling to sign IC&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Richard Greil, MD</last_name>
    <role>Study Chair</role>
    <affiliation>3rd Med Dept Paracelsus Medical University, Salzburg</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Lisa Pleyer, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>3rd Med Dept Paracelsus Medical University, Salzburg</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Matthias Florian</last_name>
    <phone>+436626404413</phone>
    <email>office@agmt.at</email>
  </overall_contact>
  <location>
    <facility>
      <name>LKH Feldkirch, Innere Medizin II, Interne E: Hämatologie und Onkologie</name>
      <address>
        <city>Feldkirch</city>
        <zip>6807</zip>
        <country>Austria</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Bernd Hartmann, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>KH der Barmherzigen Brüder, Innere Medizin I</name>
      <address>
        <city>Graz</city>
        <zip>8020</zip>
        <country>Austria</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Andrea Hammerl-Steiner, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Medizinische Universität Graz, Universitätsklinik für Innere Medizin, Klinische Abteilung für Hämatologie</name>
      <address>
        <city>Graz</city>
        <zip>8036</zip>
        <country>Austria</country>
      </address>
    </facility>
    <status>Not yet recruiting</status>
    <investigator>
      <last_name>Armin Zebisch, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Universitätsklinik Innsbruck, Univ.-Klinik für Innere Medizin V, Hämatologie und Onkologie</name>
      <address>
        <city>Innsbruck</city>
        <zip>6020</zip>
        <country>Austria</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Reinhard Stauder, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Universitätsklinikum Krems, Innere Medizin II Hämato-Onkologie</name>
      <address>
        <city>Krems</city>
        <zip>3500</zip>
        <country>Austria</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Martin Pecherstorfer, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>LKH Hochsteiermark, Abteilung für Hämato-Onkologie</name>
      <address>
        <city>Leoben</city>
        <zip>8700</zip>
        <country>Austria</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Christoph Tinchon, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Ordensklinikum Linz GmbH, Barmherzige Schwestern, Interne I: Internistische Onkologie, Hämatologie und Gastroenterologie</name>
      <address>
        <city>Linz</city>
        <zip>4010</zip>
        <country>Austria</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Gregor Aschauer, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Ordensklinikum Linz GmbH, Elisabethinen, I. Interne Abteilung Hämato-Onkologie</name>
      <address>
        <city>Linz</city>
        <zip>4020</zip>
        <country>Austria</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Sigrid Machherndl-Spandl, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Kepler Universitätsklinikum Linz, Med. Campus III., Univ.-Klinik für Hämatologie und Internistische Onkologie</name>
      <address>
        <city>Linz</city>
        <zip>4021</zip>
        <country>Austria</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Clemens Schmitt, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Universitätsklinik für Innere Med. III, PMU Salzburg</name>
      <address>
        <city>Salzburg</city>
        <zip>5020</zip>
        <country>Austria</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Richard Greil, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
    <investigator>
      <last_name>Lisa Pleyer, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Universitätsklinikum St. Pölten, Klinische Abteilung für Innere Medizin 1</name>
      <address>
        <city>St. Pölten</city>
        <zip>3100</zip>
        <country>Austria</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Petra Pichler, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Klinikum Steyr, Innere Medizin II</name>
      <address>
        <city>Steyr</city>
        <zip>4400</zip>
        <country>Austria</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Johannes Andel, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Klinikum Wels-Grieskirchen, Abteilung für Innere Medizin IV</name>
      <address>
        <city>Wels</city>
        <zip>4600</zip>
        <country>Austria</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Sonja Heibl, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Hanusch KH, 3. Med. Abteilung</name>
      <address>
        <city>Wien</city>
        <zip>1140</zip>
        <country>Austria</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Felix Keil, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Klinik Ottakring, 1. Med. Abteilung</name>
      <address>
        <city>Wien</city>
        <zip>1160</zip>
        <country>Austria</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Martin Schreder, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location_countries>
    <country>Austria</country>
  </location_countries>
  <link>
    <url>https://www.agmt.at</url>
    <description>Sponsor website</description>
  </link>
  <results_reference>
    <citation>Falantes J, Pleyer L, Thepot S, Almeida AM, Maurillo L, Martinez-Robles V, Stauder R, Itzykson R, Pinto R, Venditti A, Bargay J, Burgstaller S, Martinez MP, Seegers V, Cortesao E, Foncillas MA, Gardin C, Montesinos P, Musto P, Fenaux P, Greil R, Sanz MA, Ramos F; European ALMA + Investigators. Real life experience with frontline azacitidine in a large series of older adults with acute myeloid leukemia stratified by MRC/LRF score: results from the expanded international E-ALMA series (E-ALMA+). Leuk Lymphoma. 2018 May;59(5):1113-1120. doi: 10.1080/10428194.2017.1365854. Epub 2017 Aug 24.</citation>
    <PMID>28838276</PMID>
  </results_reference>
  <results_reference>
    <citation>Leisch M, Weiss L, Lindlbauer N, Jungbauer C, Egle A, Rohde E, Greil R, Grabmer C, Pleyer L. Red blood cell alloimmunization in 184 patients with myeloid neoplasms treated with azacitidine - A retrospective single center experience. Leuk Res. 2017 Aug;59:12-19. doi: 10.1016/j.leukres.2017.05.006. Epub 2017 May 9.</citation>
    <PMID>28535394</PMID>
  </results_reference>
  <results_reference>
    <citation>Huemer F, Weiss L, Faber V, Neureiter D, Egle A, Geissler K, Voskova D, Zebisch A, Burgstaller S, Pichler A, Stauder R, Sperr W, Lang A, Pfeilstocker M, Machherndl-Spandl S, Stampfl M, Greil R, Pleyer L. Establishment and validation of a novel risk model for estimating time to first treatment in 120 patients with chronic myelomonocytic leukaemia. Wien Klin Wochenschr. 2018 Feb;130(3-4):115-125. doi: 10.1007/s00508-018-1315-2. Epub 2018 Jan 30.</citation>
    <PMID>29383443</PMID>
  </results_reference>
  <results_reference>
    <citation>Almeida AM, Prebet T, Itzykson R, Ramos F, Al-Ali H, Shammo J, Pinto R, Maurillo L, Wetzel J, Musto P, Van De Loosdrecht AA, Costa MJ, Esteves S, Burgstaller S, Stauder R, Autzinger EM, Lang A, Krippl P, Geissler D, Falantes JF, Pedro C, Bargay J, Deben G, Garrido A, Bonanad S, Diez-Campelo M, Thepot S, Ades L, Sperr WR, Valent P, Fenaux P, Sekeres MA, Greil R, Pleyer L. Clinical Outcomes of 217 Patients with Acute Erythroleukemia According to Treatment Type and Line: A Retrospective Multinational Study. Int J Mol Sci. 2017 Apr 14;18(4):837. doi: 10.3390/ijms18040837.</citation>
    <PMID>28420120</PMID>
  </results_reference>
  <results_reference>
    <citation>Pleyer L, Dohner H, Dombret H, Seymour JF, Schuh AC, Beach CL, Swern AS, Burgstaller S, Stauder R, Girschikofsky M, Sill H, Schlick K, Thaler J, Halter B, Machherndl Spandl S, Zebisch A, Pichler A, Pfeilstocker M, Autzinger EM, Lang A, Geissler K, Voskova D, Sperr WR, Hojas S, Rogulj IM, Andel J, Greil R. Azacitidine for Front-Line Therapy of Patients with AML: Reproducible Efficacy Established by Direct Comparison of International Phase 3 Trial Data with Registry Data from the Austrian Azacitidine Registry of the AGMT Study Group. Int J Mol Sci. 2017 Feb 15;18(2):415. doi: 10.3390/ijms18020415.</citation>
    <PMID>28212292</PMID>
  </results_reference>
  <results_reference>
    <citation>Pleyer L, Burgstaller S, Stauder R, Girschikofsky M, Sill H, Schlick K, Thaler J, Halter B, Machherndl-Spandl S, Zebisch A, Pichler A, Pfeilstocker M, Autzinger EM, Lang A, Geissler K, Voskova D, Geissler D, Sperr WR, Hojas S, Rogulj IM, Andel J, Greil R. Azacitidine front-line in 339 patients with myelodysplastic syndromes and acute myeloid leukaemia: comparison of French-American-British and World Health Organization classifications. J Hematol Oncol. 2016 Apr 16;9:39. doi: 10.1186/s13045-016-0263-4.</citation>
    <PMID>27084507</PMID>
  </results_reference>
  <results_reference>
    <citation>Ramos F, Thepot S, Pleyer L, Maurillo L, Itzykson R, Bargay J, Stauder R, Venditti A, Seegers V, Martinez-Robles V, Burgstaller S, Recher C, Deben G, Gaidano G, Gardin C, Musto P, Greil R, Sanchez-Guijo F, Fenaux P; European ALMA Investigators. Azacitidine frontline therapy for unfit acute myeloid leukemia patients: clinical use and outcome prediction. Leuk Res. 2015 Mar;39(3):296-306. doi: 10.1016/j.leukres.2014.12.013. Epub 2014 Dec 31.</citation>
    <PMID>25601157</PMID>
  </results_reference>
  <results_reference>
    <citation>Pleyer L, Burgstaller S, Girschikofsky M, Linkesch W, Stauder R, Pfeilstocker M, Schreder M, Tinchon C, Sliwa T, Lang A, Sperr WR, Krippl P, Geissler D, Voskova D, Schlick K, Thaler J, Machherndl-Spandl S, Theiler G, Eckmullner O, Greil R. Azacitidine in 302 patients with WHO-defined acute myeloid leukemia: results from the Austrian Azacitidine Registry of the AGMT-Study Group. Ann Hematol. 2014 Nov;93(11):1825-38. doi: 10.1007/s00277-014-2126-9. Epub 2014 Jun 21.</citation>
    <PMID>24951123</PMID>
  </results_reference>
  <results_reference>
    <citation>Pleyer L, Germing U, Sperr WR, Linkesch W, Burgstaller S, Stauder R, Girschikofsky M, Schreder M, Pfeilstocker M, Lang A, Sliwa T, Geissler D, Schlick K, Placher-Sorko G, Theiler G, Thaler J, Mitrovic M, Neureiter D, Valent P, Greil R. Azacitidine in CMML: matched-pair analyses of daily-life patients reveal modest effects on clinical course and survival. Leuk Res. 2014 Apr;38(4):475-83. doi: 10.1016/j.leukres.2014.01.006. Epub 2014 Jan 18.</citation>
    <PMID>24522248</PMID>
  </results_reference>
  <results_reference>
    <citation>Melchardt T, Weiss L, Pleyer L, Steinkirchner S, Auberger J, Hopfinger G, Greil R, Egle A. Complications of 5-azacytidine: Three cases of severe ischemic colitis in elderly patients with myelodysplastic syndrome. Oncol Lett. 2013 Dec;6(6):1756-1758. doi: 10.3892/ol.2013.1629. Epub 2013 Oct 15.</citation>
    <PMID>24260071</PMID>
  </results_reference>
  <results_reference>
    <citation>Pleyer L, Stauder R, Burgstaller S, Schreder M, Tinchon C, Pfeilstocker M, Steinkirchner S, Melchardt T, Mitrovic M, Girschikofsky M, Lang A, Krippl P, Sliwa T, Egle A, Linkesch W, Voskova D, Angermann H, Greil R. Azacitidine in patients with WHO-defined AML - results of 155 patients from the Austrian Azacitidine Registry of the AGMT-Study Group. J Hematol Oncol. 2013 Apr 29;6:32. doi: 10.1186/1756-8722-6-32.</citation>
    <PMID>23627920</PMID>
  </results_reference>
  <results_reference>
    <citation>Valentiny C, Mitrovic M, Pleyer L, Steurer M, Willenbacher W, Stauder R. Complete remission after a single cycle of azacitidine in a case of relapsed acute myeloid leukemia. Wien Klin Wochenschr. 2013 Jan;125(1-2):50-3. doi: 10.1007/s00508-012-0319-6. Epub 2013 Jan 5.</citation>
    <PMID>23292646</PMID>
  </results_reference>
  <results_reference>
    <citation>Weiss L, Melchardt T, Neureiter D, Kemmerling R, Moshir S, Pleyer L, Greil R, Egle A. Complete remission of Waldenstrom macroglobulinemia with azacitidine and rituximab. J Clin Oncol. 2011 Aug 20;29(24):e696-8. doi: 10.1200/JCO.2011.35.8283. Epub 2011 Jul 5. No abstract available.</citation>
    <PMID>21730268</PMID>
  </results_reference>
  <verification_date>December 2020</verification_date>
  <study_first_submitted>February 24, 2020</study_first_submitted>
  <study_first_submitted_qc>June 17, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">June 19, 2020</study_first_posted>
  <last_update_submitted>December 3, 2020</last_update_submitted>
  <last_update_submitted_qc>December 3, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">December 4, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>MDS</keyword>
  <keyword>CMML</keyword>
  <keyword>AML</keyword>
  <keyword>PMF</keyword>
  <keyword>Austrian Myeloid Registry</keyword>
  <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/NCT04438889
org study id: AGMT_aMYELOIDr
nct id: NCT04438889
lead sponsor: 
is fda regulated drug: No
is fda regulated device: No
The Austrian Myeloid Registry (aMYELOIDr) is a non-interventional study. It collects data
      from patients with the myeloid diseases, primarily myelodysplastic syndromes (MDS), chronic
      myelomonocytic leukemia (CMML) and acute myeloid leukemia (AML).The aMYELOIDr is multi-center
      database collecting data at various sites in Austria and potentially also at other centers in
      other countries in future. The registry has an electronic case report form (eCRF), where all
      data is entered by clinical trial personnel and/or physicians. It is set up to collect
      real-world experience in the management of patients with these diseases in Austria.
observational model: Cohort
time perspective: Prospective
measure: To assess the treatment patterns (therapeutic landscape) of patients with myeloid diseases.
time frame: Through study completion, median expected within 100 months
description: Due to the non-interventional nature of the aMYELOIDr, treatment indication, the decision to offer treatment, treatment choice, dose, schedule and dose reductions/escalations shall be exclusively based on the risk/benefit estimation of the treating physician. We recommend compliance current guidelines.
measure: Impact of front-line treatment on overall survival (OS)
time frame: Through study completion, median expected within 100 months
measure: Impact of number and choice of treatment lines on OS as of initial diagnosis and/or as of treatment start
time frame: Through study completion, median expected within 100 months
measure: Overall response rate (ORR)
time frame: Through study completion, median expected within 100 months
description: Response will be assessed according to current guidelines for the respective disease. Due to the non-interventional nature of the aMYELOIDr, timepoints and types of response assessment shall be exclusively based on the risk/benefit estimation of the treating physician. We recommend compliance current guidelines.
measure: Event free survival (EFS)
time frame: Through study completion, median expected within 100 months
description: Events include treatment failure, progressive disease, relapse after CR/CRi, death from any cause. Patients lost to-follow-up or still alive ans without event will be censored at last follow-up date
measure: AML transformation
time frame: Through study completion, median expected within 100 months
description: Time to transformation to AML for patients with a non-AML initial diagnosis
measure: Treatment safety
time frame: Through study completion, median expected within 100 months
description: Investigators should report adverse reactions (for which a causal role of a medicine is suspected) to the concerned competent authorities following regulations in the current or future versions of Austrian legislation (Pharmakovigilanz-Verordnung 2013 (PhVO, Regulation on Pharmacovigilance), Österreichisches Arzneimittel Gesetz (AMG, Austrian Medicinal Products Act). Participation in this registry does not exempt the participating center from their legal reporting obligations. Documentation of causality, duration, frequency and severity of adverse events (AEs) according to Common Terminology Criteria for AE (CTCAE v.5).
measure: Concomitant treatments
time frame: Through study completion, median expected within 100 months
description: Concomitant treatments (number of administration of e.g. prophylactic antibiotics, antivirals, antifungals)
measure: Treatment characteristics
time frame: Through study completion, median expected within 100 months
description: Among others, the following treatment characteristics will be assessed for each treatment line: substance
measure: Treatment characteristics
time frame: Through study completion, median expected within 100 months
description: Among others, the following treatment characteristics will be assessed for each treatment line: application date
measure: Treatment characteristics
time frame: Through study completion, median expected within 100 months
description: Among others, the following treatment characteristics will be assessed for each treatment line: route
measure: Treatment characteristics
time frame: Through study completion, median expected within 100 months
description: Among others, the following treatment characteristics will be assessed for each treatment line: dose
measure: Treatment characteristics
time frame: Through study completion, median expected within 100 months
description: Among others, the following treatment characteristics will be assessed for each treatment line: inpatient or outpatient setting
measure: Concomitant treatments best supportive care (BSC)
time frame: Through study completion, median expected within 100 months
description: Concomitant best supportive care (BSC) measures (e.g. number of transfusions, growth factors, iron chelators)
measure: Quality of life assessment EQ-5D-5L (optional)
time frame: Through study completion, median expected within 100 months
description: EuroQol-5 Dimensions with 5 Levels. The descriptive system comprises five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 5 levels: no problems, slight problems, moderate problems, severe problems and extreme problems. The patient is asked to indicate his/her health state by ticking the box next to the most appropriate statement in each of the five dimensions.
measure: EORTC Quality of life assessment QLQ-C30 (optional)
time frame: Through study completion, median expected within 100 months
description: European Organisation for Research and Treatment of Cancer Quality of life 30-item questionnaire of cancer patients incorporates five functional scales (physical, role, cognitive, emotional, and social), three symptom scales (fatigue, pain, and nausea and vomiting), a global health status / QoL scale, and a number of single items assessing additional symptoms commonly reported by cancer patients (dyspnoea, loss of appetite, insomnia, constipation anddiarrhoea) and perceived financial impact of the disease. It has four-point scale which are coded with "Not at all", "A little", "Quite a bit" and "Very much"
measure: Minimal residual disease
time frame: Through study completion, median expected within 100 months
description: Assess how many and which patients in Austria have minimal residual disease (MRD) assessments, assessed by which techniques and at which timepoints.
measure: MRD-negativity on OS
time frame: Through study completion, median expected within 100 months
description: Assess the impact of MRD-negativity on OS. MRD assessment via flow-cytometry and polymerase chain reactions according to the current ELN guidelines 2017 shall be exclusively based on the risk/benefit estimation of the treating physician.
measure: Prognostic and predictive markers
time frame: Through study completion, median expected within 100 months
description: Analyses of various factors known or thought to influence OS, ORR, EFS, PFS and time to next treatment in order to validate existing or establish novel prognostic and predictive markers
arm group label: AML
description: Patients with WHO 2016 diagnosis of AML
arm group label: MDS
description: Patients with WHO 2016 diagnosis of MDS
arm group label: CMML
description: Patients with WHO 2016 diagnosis of CMML
arm group label: PMF
description: Patients with WHO 2016 diagnosis of PMF
intervention type: Other
intervention name: Non-interventional
description: Only routine clinical data, which has already been recorded in the patient's medical chart, will be documented. Any other data assessments (e.g. quality of life analyses such as EQ-5D and QLQ-C30 have been approved by the Ehtikkommission für das Bundesland Salzburg and are optional.
arm group label: AML
arm group label: CMML
arm group label: MDS
arm group label: PMF
The aMYELOIDr is accompanied by an optional biobanking program. Sample collection will be
      limited to patients that have signed an additional biobanking IC.
study pop: 
sampling method: Probability Sample
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Richard Greil, MD
role: Study Chair
affiliation: 3rd Med Dept Paracelsus Medical University, Salzburg
last name: Lisa Pleyer, MD
role: Principal Investigator
affiliation: 3rd Med Dept Paracelsus Medical University, Salzburg
last name: Matthias Florian
phone: +436626404413
email: office@agmt.at
facility: 
status: Recruiting
investigator: 
facility: 
status: Recruiting
investigator: 
facility: 
status: Not yet recruiting
investigator: 
facility: 
status: Recruiting
investigator: 
facility: 
status: Recruiting
investigator: 
facility: 
status: Recruiting
investigator: 
facility: 
status: Recruiting
investigator: 
facility: 
status: Recruiting
investigator: 
facility: 
status: Recruiting
investigator: 
facility: 
status: Recruiting
investigator: 
investigator: 
facility: 
status: Recruiting
investigator: 
facility: 
status: Recruiting
investigator: 
facility: 
status: Recruiting
investigator: 
facility: 
status: Recruiting
investigator: 
facility: 
status: Recruiting
investigator: 
country: Austria
url: https://www.agmt.at
description: Sponsor website
citation: Falantes J, Pleyer L, Thepot S, Almeida AM, Maurillo L, Martinez-Robles V, Stauder R, Itzykson R, Pinto R, Venditti A, Bargay J, Burgstaller S, Martinez MP, Seegers V, Cortesao E, Foncillas MA, Gardin C, Montesinos P, Musto P, Fenaux P, Greil R, Sanz MA, Ramos F; European ALMA + Investigators. Real life experience with frontline azacitidine in a large series of older adults with acute myeloid leukemia stratified by MRC/LRF score: results from the expanded international E-ALMA series (E-ALMA+). Leuk Lymphoma. 2018 May;59(5):1113-1120. doi: 10.1080/10428194.2017.1365854. Epub 2017 Aug 24.
PMID: 28838276
citation: Leisch M, Weiss L, Lindlbauer N, Jungbauer C, Egle A, Rohde E, Greil R, Grabmer C, Pleyer L. Red blood cell alloimmunization in 184 patients with myeloid neoplasms treated with azacitidine - A retrospective single center experience. Leuk Res. 2017 Aug;59:12-19. doi: 10.1016/j.leukres.2017.05.006. Epub 2017 May 9.
PMID: 28535394
citation: Huemer F, Weiss L, Faber V, Neureiter D, Egle A, Geissler K, Voskova D, Zebisch A, Burgstaller S, Pichler A, Stauder R, Sperr W, Lang A, Pfeilstocker M, Machherndl-Spandl S, Stampfl M, Greil R, Pleyer L. Establishment and validation of a novel risk model for estimating time to first treatment in 120 patients with chronic myelomonocytic leukaemia. Wien Klin Wochenschr. 2018 Feb;130(3-4):115-125. doi: 10.1007/s00508-018-1315-2. Epub 2018 Jan 30.
PMID: 29383443
citation: Almeida AM, Prebet T, Itzykson R, Ramos F, Al-Ali H, Shammo J, Pinto R, Maurillo L, Wetzel J, Musto P, Van De Loosdrecht AA, Costa MJ, Esteves S, Burgstaller S, Stauder R, Autzinger EM, Lang A, Krippl P, Geissler D, Falantes JF, Pedro C, Bargay J, Deben G, Garrido A, Bonanad S, Diez-Campelo M, Thepot S, Ades L, Sperr WR, Valent P, Fenaux P, Sekeres MA, Greil R, Pleyer L. Clinical Outcomes of 217 Patients with Acute Erythroleukemia According to Treatment Type and Line: A Retrospective Multinational Study. Int J Mol Sci. 2017 Apr 14;18(4):837. doi: 10.3390/ijms18040837.
PMID: 28420120
citation: Pleyer L, Dohner H, Dombret H, Seymour JF, Schuh AC, Beach CL, Swern AS, Burgstaller S, Stauder R, Girschikofsky M, Sill H, Schlick K, Thaler J, Halter B, Machherndl Spandl S, Zebisch A, Pichler A, Pfeilstocker M, Autzinger EM, Lang A, Geissler K, Voskova D, Sperr WR, Hojas S, Rogulj IM, Andel J, Greil R. Azacitidine for Front-Line Therapy of Patients with AML: Reproducible Efficacy Established by Direct Comparison of International Phase 3 Trial Data with Registry Data from the Austrian Azacitidine Registry of the AGMT Study Group. Int J Mol Sci. 2017 Feb 15;18(2):415. doi: 10.3390/ijms18020415.
PMID: 28212292
citation: Pleyer L, Burgstaller S, Stauder R, Girschikofsky M, Sill H, Schlick K, Thaler J, Halter B, Machherndl-Spandl S, Zebisch A, Pichler A, Pfeilstocker M, Autzinger EM, Lang A, Geissler K, Voskova D, Geissler D, Sperr WR, Hojas S, Rogulj IM, Andel J, Greil R. Azacitidine front-line in 339 patients with myelodysplastic syndromes and acute myeloid leukaemia: comparison of French-American-British and World Health Organization classifications. J Hematol Oncol. 2016 Apr 16;9:39. doi: 10.1186/s13045-016-0263-4.
PMID: 27084507
citation: Ramos F, Thepot S, Pleyer L, Maurillo L, Itzykson R, Bargay J, Stauder R, Venditti A, Seegers V, Martinez-Robles V, Burgstaller S, Recher C, Deben G, Gaidano G, Gardin C, Musto P, Greil R, Sanchez-Guijo F, Fenaux P; European ALMA Investigators. Azacitidine frontline therapy for unfit acute myeloid leukemia patients: clinical use and outcome prediction. Leuk Res. 2015 Mar;39(3):296-306. doi: 10.1016/j.leukres.2014.12.013. Epub 2014 Dec 31.
PMID: 25601157
citation: Pleyer L, Burgstaller S, Girschikofsky M, Linkesch W, Stauder R, Pfeilstocker M, Schreder M, Tinchon C, Sliwa T, Lang A, Sperr WR, Krippl P, Geissler D, Voskova D, Schlick K, Thaler J, Machherndl-Spandl S, Theiler G, Eckmullner O, Greil R. Azacitidine in 302 patients with WHO-defined acute myeloid leukemia: results from the Austrian Azacitidine Registry of the AGMT-Study Group. Ann Hematol. 2014 Nov;93(11):1825-38. doi: 10.1007/s00277-014-2126-9. Epub 2014 Jun 21.
PMID: 24951123
citation: Pleyer L, Germing U, Sperr WR, Linkesch W, Burgstaller S, Stauder R, Girschikofsky M, Schreder M, Pfeilstocker M, Lang A, Sliwa T, Geissler D, Schlick K, Placher-Sorko G, Theiler G, Thaler J, Mitrovic M, Neureiter D, Valent P, Greil R. Azacitidine in CMML: matched-pair analyses of daily-life patients reveal modest effects on clinical course and survival. Leuk Res. 2014 Apr;38(4):475-83. doi: 10.1016/j.leukres.2014.01.006. Epub 2014 Jan 18.
PMID: 24522248
citation: Melchardt T, Weiss L, Pleyer L, Steinkirchner S, Auberger J, Hopfinger G, Greil R, Egle A. Complications of 5-azacytidine: Three cases of severe ischemic colitis in elderly patients with myelodysplastic syndrome. Oncol Lett. 2013 Dec;6(6):1756-1758. doi: 10.3892/ol.2013.1629. Epub 2013 Oct 15.
PMID: 24260071
citation: Pleyer L, Stauder R, Burgstaller S, Schreder M, Tinchon C, Pfeilstocker M, Steinkirchner S, Melchardt T, Mitrovic M, Girschikofsky M, Lang A, Krippl P, Sliwa T, Egle A, Linkesch W, Voskova D, Angermann H, Greil R. Azacitidine in patients with WHO-defined AML - results of 155 patients from the Austrian Azacitidine Registry of the AGMT-Study Group. J Hematol Oncol. 2013 Apr 29;6:32. doi: 10.1186/1756-8722-6-32.
PMID: 23627920
citation: Valentiny C, Mitrovic M, Pleyer L, Steurer M, Willenbacher W, Stauder R. Complete remission after a single cycle of azacitidine in a case of relapsed acute myeloid leukemia. Wien Klin Wochenschr. 2013 Jan;125(1-2):50-3. doi: 10.1007/s00508-012-0319-6. Epub 2013 Jan 5.
PMID: 23292646
citation: Weiss L, Melchardt T, Neureiter D, Kemmerling R, Moshir S, Pleyer L, Greil R, Egle A. Complete remission of Waldenstrom macroglobulinemia with azacitidine and rituximab. J Clin Oncol. 2011 Aug 20;29(24):e696-8. doi: 10.1200/JCO.2011.35.8283. Epub 2011 Jul 5. No abstract available.
PMID: 21730268
responsible party type: Sponsor
sharing ipd: Undecided