<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/NCT03303313</url>
  </required_header>
  <id_info>
    <org_study_id>ALN-CC5-004</org_study_id>
    <nct_id>NCT03303313</nct_id>
  </id_info>
  <brief_title>A Study of an Investigational Drug, Cemdisiran (ALN-CC5), in Patients With Atypical Hemolytic Uremic Syndrome</brief_title>
  <official_title>A Phase 2, Open Label, Multicenter Study of ALN-CC5 Administered Subcutaneously in Adult Patients With Atypical Hemolytic Uremic Syndrome</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Alnylam Pharmaceuticals</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Alnylam Pharmaceuticals</source>
  <oversight_info>
    <has_dmc>Yes</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>
      The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of&#xD;
      Cemdisiran in patients with aHUS.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Withdrawn</overall_status>
  <why_stopped>
    Study terminated early due to lack of enrollment.&#xD;
  </why_stopped>
  <start_date type="Actual">September 19, 2017</start_date>
  <completion_date type="Actual">September 12, 2018</completion_date>
  <primary_completion_date type="Actual">September 12, 2018</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>The effect of Cemdisiran on platelet count</measure>
    <time_frame>Week 32</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>The effect of Cemdisiran on hematological response as measured by platelet count</measure>
    <time_frame>after 32 weeks of treatment</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The effect of Cemdisiran on hematological response as measured by lactate dehydrogenase (LDH)</measure>
    <time_frame>after 32 weeks of treatment</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The effect of Cemdisiran on hematological response as measured by rescue plasma therapy</measure>
    <time_frame>after 32 weeks of treatment</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The effect of Cemdisiran on LDH response as measured by LDH</measure>
    <time_frame>after 32 weeks of treatment</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The effect of Cemdisiran on LDH response as measured by rescue plasma therapy</measure>
    <time_frame>after 32 weeks of treatment</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by platelet count</measure>
    <time_frame>after 32 weeks of treatment</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by LDH</measure>
    <time_frame>after 32 weeks of treatment</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by serum creatinine levels</measure>
    <time_frame>after 32 weeks of treatment</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by rescue plasma therapy</measure>
    <time_frame>after 32 weeks of treatment</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The effect of Cemdisiran on serum creatinine levels</measure>
    <time_frame>up to 84 weeks</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The effect of Cemdisiran on estimated glomerular filtration rate (eGFR)</measure>
    <time_frame>up to 84 weeks</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The effect of Cemdisiran on adverse events (AEs)</measure>
    <time_frame>up to 108 weeks</time_frame>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">0</enrollment>
  <condition>Atypical Hemolytic Uremic Syndrome</condition>
  <arm_group>
    <arm_group_label>Cemdisiran</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Cemdisiran</intervention_name>
    <description>Subcutaneous (sc) injection of Cemdisiran</description>
    <arm_group_label>Cemdisiran</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Willing to provide written informed consent and to comply with the study requirements&#xD;
&#xD;
          2. Age 18 years or older&#xD;
&#xD;
          3. Clinical diagnosis of primary aHUS&#xD;
&#xD;
          4. Clinical thrombotic microangiopathy (TMA) activity&#xD;
&#xD;
          5. Women of child-bearing potential must have a negative pregnancy test, cannot be breast&#xD;
             feeding, and must be willing to use a highly effective method of contraception&#xD;
&#xD;
          6. Previously vaccinated with meningococcal group ACWY conjugate vaccine and&#xD;
             meningococcal group B vaccine or willingness to receive these vaccinations&#xD;
&#xD;
          7. ADAMTS13 &gt;10% or other proven aHUS-associated mutation&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Clinically significant abnormal laboratory results&#xD;
&#xD;
          2. Positive Shiga toxin producing Escherichia coli test at Screening&#xD;
&#xD;
          3. Suspected secondary aHUS, in the opinion of the Investigator (unless there is a&#xD;
             documented aHUS-associated genetic mutation)&#xD;
&#xD;
          4. Positive direct Coombs test&#xD;
&#xD;
          5. Patients who have received hemodialysis for &gt;3 months&#xD;
&#xD;
          6. Bone marrow transplant recipients&#xD;
&#xD;
          7. Organ transplant recipients, except for kidney transplant recipients with primary aHUS&#xD;
             (confirmed by known genetic mutation and kidney biopsy)&#xD;
&#xD;
          8. Known history or evidence of systemic lupus erythematosus or antiphospholipid antibody&#xD;
             syndrome&#xD;
&#xD;
          9. History of multiple drug allergies or history of allergic reaction to an&#xD;
             oligonucleotide or GalNAc&#xD;
&#xD;
         10. Malignancy (except for non-melanoma skin cancers, cervical in-situ carcinoma, breast&#xD;
             ductal carcinoma in situ, or stage 1 prostate carcinoma) within the last 5 years&#xD;
&#xD;
         11. Patients with a poor prognosis that is expected to limit their life expectancy to less&#xD;
             than 3 months, in the opinion of the Investigator&#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>Nader Najafian, MD</last_name>
    <role>Study Director</role>
    <affiliation>Alnylam Pharmaceuticals</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Sarajevo</city>
        <country>Bosnia and Herzegovina</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Calgary</city>
        <zip>T2N 2T9</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Tallinn</city>
        <country>Estonia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Tartu</city>
        <country>Estonia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Tbilisi</city>
        <country>Georgia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Riga</city>
        <country>Latvia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Kaunas</city>
        <country>Lithuania</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Vilnius</city>
        <country>Lithuania</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Skopje</city>
        <country>Macedonia, The Former Yugoslav Republic of</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Chisinau</city>
        <country>Moldova, Republic of</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Belgrade</city>
        <country>Serbia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Trial Site</name>
      <address>
        <city>Örebro</city>
        <country>Sweden</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Bosnia and Herzegovina</country>
    <country>Canada</country>
    <country>Estonia</country>
    <country>Georgia</country>
    <country>Latvia</country>
    <country>Lithuania</country>
    <country>Macedonia, The Former Yugoslav Republic of</country>
    <country>Moldova, Republic of</country>
    <country>Serbia</country>
    <country>Sweden</country>
  </location_countries>
  <verification_date>September 2018</verification_date>
  <study_first_submitted>September 25, 2017</study_first_submitted>
  <study_first_submitted_qc>October 2, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">October 6, 2017</study_first_posted>
  <last_update_submitted>September 27, 2018</last_update_submitted>
  <last_update_submitted_qc>September 27, 2018</last_update_submitted_qc>
  <last_update_posted type="Actual">October 1, 2018</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>RNAi therapeutic</keyword>
  <keyword>Atypical Hemolytic Uremic Syndrome</keyword>
  <keyword>Hemolysis</keyword>
  <keyword>Thrombocytopenia</keyword>
  <keyword>Renal insufficiency</keyword>
  <keyword>Thrombotic Microangiopathy</keyword>
  <keyword>aHUS</keyword>
  <keyword>TMA</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Azotemia</mesh_term>
    <mesh_term>Hemolytic-Uremic Syndrome</mesh_term>
    <mesh_term>Atypical Hemolytic Uremic Syndrome</mesh_term>
    <mesh_term>Syndrome</mesh_term>
    <mesh_term>Hemolysis</mesh_term>
  </condition_browse>
  <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/NCT03303313
org study id: ALN-CC5-004
nct id: NCT03303313
lead sponsor: 
has dmc: Yes
is fda regulated drug: No
is fda regulated device: No
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of
      Cemdisiran in patients with aHUS.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: The effect of Cemdisiran on platelet count
time frame: Week 32
measure: The effect of Cemdisiran on hematological response as measured by platelet count
time frame: after 32 weeks of treatment
measure: The effect of Cemdisiran on hematological response as measured by lactate dehydrogenase (LDH)
time frame: after 32 weeks of treatment
measure: The effect of Cemdisiran on hematological response as measured by rescue plasma therapy
time frame: after 32 weeks of treatment
measure: The effect of Cemdisiran on LDH response as measured by LDH
time frame: after 32 weeks of treatment
measure: The effect of Cemdisiran on LDH response as measured by rescue plasma therapy
time frame: after 32 weeks of treatment
measure: The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by platelet count
time frame: after 32 weeks of treatment
measure: The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by LDH
time frame: after 32 weeks of treatment
measure: The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by serum creatinine levels
time frame: after 32 weeks of treatment
measure: The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by rescue plasma therapy
time frame: after 32 weeks of treatment
measure: The effect of Cemdisiran on serum creatinine levels
time frame: up to 84 weeks
measure: The effect of Cemdisiran on estimated glomerular filtration rate (eGFR)
time frame: up to 84 weeks
measure: The effect of Cemdisiran on adverse events (AEs)
time frame: up to 108 weeks
arm group label: Cemdisiran
arm group type: Experimental
intervention type: Drug
intervention name: Cemdisiran
description: Subcutaneous (sc) injection of Cemdisiran
arm group label: Cemdisiran
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Nader Najafian, MD
role: Study Director
affiliation: Alnylam Pharmaceuticals
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: Bosnia and Herzegovina
country: Canada
country: Estonia
country: Georgia
country: Latvia
country: Lithuania
country: Macedonia, The Former Yugoslav Republic of
country: Moldova, Republic of
country: Serbia
country: Sweden
responsible party type: Sponsor
mesh term: Azotemia
mesh term: Hemolytic-Uremic Syndrome
mesh term: Atypical Hemolytic Uremic Syndrome
mesh term: Syndrome
mesh term: Hemolysis
sharing ipd: Undecided