<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/NCT03774784</url>
  </required_header>
  <id_info>
    <org_study_id>ALN-LECT2-NT-001</org_study_id>
    <nct_id>NCT03774784</nct_id>
  </id_info>
  <brief_title>A Study of the Natural History of Leukocyte Chemotactic Factor 2 Amyloidosis (ALECT2) Disease</brief_title>
  <official_title>A Study of the Natural History of Leukocyte Chemotactic Factor 2 Amyloidosis (ALECT2) Disease</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Alnylam Pharmaceuticals</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Alnylam Pharmaceuticals</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 purpose of this study is to characterize the natural history of leukocyte chemotactic&#xD;
      factor 2 amyloidotic (ALECT2) disease. In this observational study participants with ALECT2&#xD;
      disease will be enrolled. Participants, who have already reached end-stage renal disease&#xD;
      (ESRD), will provide retrospective chart review data and biological specimens at baseline&#xD;
      only. Other participants, in addition to retrospective chart review, will be followed&#xD;
      prospectively.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Refer to www.studyALECT2.com&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Terminated</overall_status>
  <why_stopped>
    The study was terminated due to the impacts of COVID-19 on patient safety and enrollment.&#xD;
  </why_stopped>
  <start_date type="Actual">January 29, 2019</start_date>
  <completion_date type="Actual">May 18, 2020</completion_date>
  <primary_completion_date type="Actual">May 18, 2020</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>Change in Estimated Glomerular Filtration Rate (eGFR) from Baseline Up to End of Study (Month 48)</measure>
    <time_frame>Baseline (Day 1), Months 6, 12, 18, 24, 30, 36, 42 and 48</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Time to End-Stage Renal Disease (ESRD)</measure>
    <time_frame>From baseline to end of study (Month 48)</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>Level of Leukocyte Chemotactic Factor 2 (LECT2) Messenger Ribonucleic Acid (mRNA) in Blood</measure>
    <time_frame>Baseline (Day 1), Months 6, 12, 18, 24, 30, 36, 42 and 48</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Level of LECT2 mRNA in Urine</measure>
    <time_frame>Baseline (Day 1), Months 12, 24, 36 and 48</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Level of LECT2 Protein in Blood</measure>
    <time_frame>Baseline (Day 1), Months 6, 12, 18, 24, 30, 36, 42 and 48</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Level of LECT2 Protein in Urine</measure>
    <time_frame>Baseline (Day 1), Months 12, 24, 36 and 48</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Percentage of Participants With Proteinuria</measure>
    <time_frame>Baseline (Day 1), Months 12, 24, 36 and 48</time_frame>
  </secondary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Actual">26</enrollment>
  <condition>Amyloidosis</condition>
  <condition>Leukocyte Chemotactic Factor 2 Amyloidosis</condition>
  <arm_group>
    <arm_group_label>ALECT2 Disease</arm_group_label>
  </arm_group>
  <eligibility>
    <study_pop>
      <textblock>
        Participants with biopsy-proven ALECT2.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Males and females, age 18 years or older;&#xD;
&#xD;
          -  Renal biopsy-proven diagnosis of ALECT2;&#xD;
&#xD;
          -  For patients with kidney disease that could be due to ALECT2, a renal biopsy may be&#xD;
             obtained to confirm ALECT2 diagnosis.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
        - There are no exclusion criteria for this observational study.&#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>Joshua Friedman, MD</last_name>
    <role>Study Director</role>
    <affiliation>Alnylam Pharmaceuticals</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Scottsdale</city>
        <state>Arizona</state>
        <zip>85259</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Tucson</city>
        <state>Arizona</state>
        <zip>85724</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Denver</city>
        <state>Colorado</state>
        <zip>80210</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Chicago</city>
        <state>Illinois</state>
        <zip>60611</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Indianapolis</city>
        <state>Indiana</state>
        <zip>46202</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Baltimore</city>
        <state>Maryland</state>
        <zip>21205</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02118</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Detroit</city>
        <state>Michigan</state>
        <zip>48201</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Rochester</city>
        <state>Minnesota</state>
        <zip>55902</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Albuquerque</city>
        <state>New Mexico</state>
        <zip>87106</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Columbus</city>
        <state>Ohio</state>
        <zip>43210</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Portland</city>
        <state>Oregon</state>
        <zip>97239</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Houston</city>
        <state>Texas</state>
        <zip>77004</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Houston</city>
        <state>Texas</state>
        <zip>77030</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>San Antonio</city>
        <state>Texas</state>
        <zip>78212</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Saint George</city>
        <state>Utah</state>
        <zip>84790</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Alexandria</city>
        <country>Egypt</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Assiut</city>
        <country>Egypt</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Cairo</city>
        <country>Egypt</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Mansoura City</city>
        <country>Egypt</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Chandigarh</city>
        <country>India</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Ludhiāna</city>
        <country>India</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Manipal</city>
        <country>India</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Nadiād</city>
        <country>India</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Tamil Nadu</city>
        <country>India</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Kuala Lumpur</city>
        <country>Malaysia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Mexicali</city>
        <state>Baja</state>
        <zip>21200</zip>
        <country>Mexico</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Mexico</city>
        <state>Mexico DF</state>
        <zip>14080</zip>
        <country>Mexico</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Culiacán</city>
        <state>Sinaloa</state>
        <zip>80230</zip>
        <country>Mexico</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Monterrey</city>
        <country>Mexico</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>London</city>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Egypt</country>
    <country>India</country>
    <country>Malaysia</country>
    <country>Mexico</country>
    <country>United Kingdom</country>
    <country>United States</country>
  </location_countries>
  <verification_date>June 2020</verification_date>
  <study_first_submitted>December 11, 2018</study_first_submitted>
  <study_first_submitted_qc>December 11, 2018</study_first_submitted_qc>
  <study_first_posted type="Actual">December 13, 2018</study_first_posted>
  <last_update_submitted>June 12, 2020</last_update_submitted>
  <last_update_submitted_qc>June 12, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">June 16, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Amyloidosis</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/NCT03774784
org study id: ALN-LECT2-NT-001
nct id: NCT03774784
lead sponsor: 
is fda regulated drug: No
is fda regulated device: No
The purpose of this study is to characterize the natural history of leukocyte chemotactic
      factor 2 amyloidotic (ALECT2) disease. In this observational study participants with ALECT2
      disease will be enrolled. Participants, who have already reached end-stage renal disease
      (ESRD), will provide retrospective chart review data and biological specimens at baseline
      only. Other participants, in addition to retrospective chart review, will be followed
      prospectively.
Refer to www.studyALECT2.com
observational model: Cohort
time perspective: Prospective
measure: Change in Estimated Glomerular Filtration Rate (eGFR) from Baseline Up to End of Study (Month 48)
time frame: Baseline (Day 1), Months 6, 12, 18, 24, 30, 36, 42 and 48
measure: Time to End-Stage Renal Disease (ESRD)
time frame: From baseline to end of study (Month 48)
measure: Level of Leukocyte Chemotactic Factor 2 (LECT2) Messenger Ribonucleic Acid (mRNA) in Blood
time frame: Baseline (Day 1), Months 6, 12, 18, 24, 30, 36, 42 and 48
measure: Level of LECT2 mRNA in Urine
time frame: Baseline (Day 1), Months 12, 24, 36 and 48
measure: Level of LECT2 Protein in Blood
time frame: Baseline (Day 1), Months 6, 12, 18, 24, 30, 36, 42 and 48
measure: Level of LECT2 Protein in Urine
time frame: Baseline (Day 1), Months 12, 24, 36 and 48
measure: Percentage of Participants With Proteinuria
time frame: Baseline (Day 1), Months 12, 24, 36 and 48
arm group label: ALECT2 Disease
study pop: 
sampling method: Probability Sample
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Joshua Friedman, MD
role: Study Director
affiliation: Alnylam Pharmaceuticals
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country: Egypt
country: India
country: Malaysia
country: Mexico
country: United Kingdom
country: United States
responsible party type: Sponsor
mesh term: Amyloidosis
sharing ipd: No