xml: <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/NCT05993416</url>
    </required_header>
    <id_info>
      <org_study_id>DCR-PHXC-401</org_study_id>
      <nct_id>NCT05993416</nct_id>
    </id_info>
    <brief_title>Treatment of Primary Hyperoxaluria Type 1 With Nedosiran</brief_title>
    <official_title>Treatment of Primary Hyperoxaluria Type 1 With Nedosiran</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>Dicerna Pharmaceuticals, Inc., a Novo Nordisk company</agency>
        <agency_class>Industry</agency_class>
      </lead_sponsor>
    </sponsors>
    <source>Dicerna Pharmaceuticals, Inc., a Novo Nordisk company</source>
    <brief_summary>
      <textblock>
        The purpose of this program is to provide participants with access to an investigational&#xD;
        drug, nedosiran, for treatment of primary hyperoxaluria type 1 (PH1). Eligible participants&#xD;
        may receive nedosiran in this program until the drug is commercially available or until Novo&#xD;
        Nordisk terminates the program, whichever comes first. Novo Nordisk may terminate the program&#xD;
        at any time for any reason, including if the drug receives regulatory approval and becomes&#xD;
        commercially available, or if the drug does not receive regulatory approval. Nedosiran will&#xD;
        be given once a month with a thin needle in the thigh or abdomen. The study doctor will ask&#xD;
        the participant to come to the clinic monthly. The study doctor may allow participant to take&#xD;
        nedosiran at home for self-administration. The participant should let the doctor know if they&#xD;
        are unable to make a visit so it can be rescheduled. Participants to inform the study doctor&#xD;
        of any medications they are taking, including over the counter medicines, vitamins, and&#xD;
        herbal medicines. If any medications change in dose, or new medications are added,&#xD;
        participants should inform the study doctor. Study doctor should be informed of any new or&#xD;
        continued health problems or any changes in the participant's health.&#xD;
      </textblock>
    </brief_summary>
    <overall_status>Available</overall_status>
    <study_type>Expanded Access</study_type>
    <has_expanded_access>No</has_expanded_access>
    <expanded_access_info>
      <expanded_access_type_treatment>Yes</expanded_access_type_treatment>
    </expanded_access_info>
    <condition>Primary Hyperoxaluria Type 1 (PH1)</condition>
    <intervention>
      <intervention_type>Drug</intervention_type>
      <intervention_name>Nedosiran</intervention_name>
      <description>In adults and in adolescents (aged 12-17 years) weighing greater than or equal to (&gt;=) 50 kilograms (kg), Nedosiran will be administered via subcutaneous (SC) injection once monthly at a dose of 170 milligrams (mg). In adults and adolescents weighing &lt; 50 kg, nedosiran will be administered once monthly at a dose of 136 mg. The dose for participants aged 6 to 11 years will be 3.5 mg/kg monthly, not to exceed 170 mg.</description>
    </intervention>
    <eligibility>
      <criteria>
        <textblock>
          Early Access Eligibility Criteria:&#xD;
  &#xD;
            -  Written, signed, and dated informed patient/parent consent; and for patients who were&#xD;
               minors, age-appropriate assent (performed according to local regulations)&#xD;
  &#xD;
            -  Male or female patients at least 6 years of age&#xD;
  &#xD;
            -  Genetically-confirmed diagnosis of PH1&#xD;
  &#xD;
            -  Accessible for treatment and follow-up and be able to comply with treatment monitoring&#xD;
               requirements&#xD;
  &#xD;
            -  Not eligible for an ongoing RNAi therapy trial and not participating simultaneously in&#xD;
               any interventional clinical research study&#xD;
  &#xD;
            -  Patients with PH1 that are not satisfactorily treated with current standard of care;&#xD;
               i.e., 24-hour urinary oxalate (Uox) excretion ≥ 0.7 (millimoles) mmol for participants&#xD;
               18 years and older, or greater or lesser than (≥) 0.7 mmol per 1.73 metre square (m^2)&#xD;
               body surface area (BSA) for participants less than 18 years of age&#xD;
  &#xD;
            -  Estimated GFR at screening ≥ 30 millilitres per minute (mL/min) normalized to 1.73 m^2&#xD;
               BSA&#xD;
  &#xD;
            -  No renal or hepatic transplantation; prior or planned within the treatment period&#xD;
  &#xD;
            -  No documented evidence of clinical manifestations of systemic oxalosis (including pre&#xD;
               existing retinal, heart, or skin calcifications, or history of severe bone pain,&#xD;
               pathological fractures, or bone deformations)&#xD;
  &#xD;
            -  Patient not currently on dialysis&#xD;
  &#xD;
            -  Plasma oxalate ≤ 30 micromoles per litre (μmol/L)&#xD;
  &#xD;
            -  Female patients not breastfeeding or pregnant&#xD;
  &#xD;
            -  The potential benefit for the individual patient justifies the potential risks of&#xD;
               treatment as per prescribing physician judgment&#xD;
        </textblock>
      </criteria>
      <gender>All</gender>
      <minimum_age>6 Years</minimum_age>
      <maximum_age>N/A</maximum_age>
    </eligibility>
    <overall_contact>
      <last_name>Medical Information</last_name>
      <phone>+44 20 39 408 111</phone>
      <email>Nedosiran@tannerpharma.com</email>
    </overall_contact>
    <verification_date>August 2023</verification_date>
    <study_first_submitted>August 6, 2023</study_first_submitted>
    <study_first_submitted_qc>August 7, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">August 15, 2023</study_first_posted>
    <last_update_submitted>August 7, 2023</last_update_submitted>
    <last_update_submitted_qc>August 7, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">August 15, 2023</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>Hyperoxaluria, Primary</mesh_term>
    </condition_browse>
    <!-- Results have not yet been posted for this study                                          -->
  </clinical_study>
  
  
text: 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/NCT05993416
  org study id: DCR-PHXC-401
  nct id: NCT05993416
  lead sponsor: 
  The purpose of this program is to provide participants with access to an investigational
        drug, nedosiran, for treatment of primary hyperoxaluria type 1 (PH1). Eligible participants
        may receive nedosiran in this program until the drug is commercially available or until Novo
        Nordisk terminates the program, whichever comes first. Novo Nordisk may terminate the program
        at any time for any reason, including if the drug receives regulatory approval and becomes
        commercially available, or if the drug does not receive regulatory approval. Nedosiran will
        be given once a month with a thin needle in the thigh or abdomen. The study doctor will ask
        the participant to come to the clinic monthly. The study doctor may allow participant to take
        nedosiran at home for self-administration. The participant should let the doctor know if they
        are unable to make a visit so it can be rescheduled. Participants to inform the study doctor
        of any medications they are taking, including over the counter medicines, vitamins, and
        herbal medicines. If any medications change in dose, or new medications are added,
        participants should inform the study doctor. Study doctor should be informed of any new or
        continued health problems or any changes in the participant's health.
  expanded access type treatment: Yes
  intervention type: Drug
  intervention name: Nedosiran
  description: In adults and in adolescents (aged 12-17 years) weighing greater than or equal to (>=) 50 kilograms (kg), Nedosiran will be administered via subcutaneous (SC) injection once monthly at a dose of 170 milligrams (mg). In adults and adolescents weighing < 50 kg, nedosiran will be administered once monthly at a dose of 136 mg. The dose for participants aged 6 to 11 years will be 3.5 mg/kg monthly, not to exceed 170 mg.
  criteria: 
  gender: All
  minimum age: 6 Years
  maximum age: N/A
  last name: Medical Information
  phone: +44 20 39 408 111
  email: Nedosiran@tannerpharma.com
  responsible party type: Sponsor
  mesh term: Hyperoxaluria, Primary