<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/NCT02973360</url>
  </required_header>
  <id_info>
    <org_study_id>OMEG-411-02</org_study_id>
    <nct_id>NCT02973360</nct_id>
  </id_info>
  <brief_title>Dose-Finding Study of SC411 in Children With Sickle Cell Disease</brief_title>
  <acronym>SCOT</acronym>
  <official_title>A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Dose-Finding Study and Open-Label Extension of SC411 in Children With Sickle Cell Disease</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Micelle BioPharma Inc</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Micelle BioPharma Inc</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      This is a Phase 2, randomized, double-blind, placebo-controlled, parallel-group, dose-finding&#xD;
      study of SC411 in children with sickle cell disease (SCD). The primary objective of the study&#xD;
      is to evaluate the safety and tolerability of three different doses of SC411 compared to a&#xD;
      placebo. All patients will undergo eight weeks of oral study treatment and a four-week safety&#xD;
      follow-up period. Patients will be randomized to one of three dose levels of SC411 or&#xD;
      placebo.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This Phase 2 study is to be conducted in two parts. Part A is a randomized, double-blind,&#xD;
      placebo-controlled, parallel-group, dose-finding study of SC411 in children with SCD. This&#xD;
      part of the study will consist of a screening period of up to 2 weeks, followed by an&#xD;
      eight-week treatment period. Part B is an optional 49-month open-label extension for patients&#xD;
      who have completed Part A.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Active, not recruiting</last_known_status>
  <start_date type="Actual">March 2, 2017</start_date>
  <completion_date type="Anticipated">January 2, 2022</completion_date>
  <primary_completion_date type="Actual">September 26, 2017</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>Triple (Participant, Care Provider, Investigator)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Evaluate safety &amp; tolerability of SC411 &amp; determine change from Baseline in the blood cells omega-3 fatty acids index in subjects, treated with active SC411 doses or placebo in Part A and safety and tolerability in Part B.</measure>
    <time_frame>Week 0 (baseline) through Month 52 (end of treatment)</time_frame>
    <description>Evaluate the safety and tolerability of three different doses of SC411 and determine the change from Baseline in the blood cells omega-3 fatty acids index in subjects, treated with either one of the three active doses of SC411 or matching placebo in Part A. Safety and tolerability will continue to be evaluated and reported during Part B.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Measurement of the pharmacokinetic (PK) parameter of AUC will be performed for three dose levels of SC411 will be measured.</measure>
    <time_frame>2 weeks before baseline (screening) through Week 52</time_frame>
    <description>Measurement of the pharmacokinetic (PK) parameter of AUC will be performed for three dose levels of SC411 will be measured.&#xD;
The steady-state pharmacokinetic (PK) profile of the three doses and area under the curve will be reported. The purpose is to assess the steady-state pharmacokinetic (PK) profile of the three dose levels of SC411, following two months of dosing, and at the end of treatment (EOT).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Measurement of the pharmacokinetic (PK) parameter of Tmax will be performed for three dose levels of SC411 will be measured.</measure>
    <time_frame>2 weeks before baseline (screening) through Week 52</time_frame>
    <description>Measurement of the pharmacokinetic (PK) parameter of Tmax will be performed for three dose levels of SC411 will be measured.&#xD;
The steady-state pharmacokinetic (PK) profile of the three doses and Tmax will be reported. The purpose is to assess the steady-state pharmacokinetic (PK) profile of the three dose levels of SC411, following two months of dosing, and at the end of treatment (EOT).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Measurement of the pharmacokinetic (PK) parameter of Cmax will be performed for three dose levels of SC411 will be measured.</measure>
    <time_frame>2 weeks before baseline (screening) through Week 52</time_frame>
    <description>The steady-state pharmacokinetic (PK) profile of the three doses and Cmax will be reported. The purpose is to assess the steady-state pharmacokinetic (PK) profile of the three dose levels of SC411, following two months of dosing, and at the end of treatment (EOT).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The safety &amp; long term tolerability of SC411 will be evaluated in Part A, &amp; a selected dose in Part B. The change from Baseline in blood cells omega-3 fatty acids index in subjects, treated with SC411 or placebo in Part A will be determined.</measure>
    <time_frame>Week 0 (baseline) through Month 52 (end of treatment)</time_frame>
    <description>Evaluate the safety and long term tolerability of three different doses of SC411 and determine the change from Baseline in the blood cells omega-3 fatty acids index in subjects, treated with either one of the three active doses of SC411 or matching placebo in Part A. Safety and long term tolerability will continue to be evaluated and reported during Part B.</description>
  </secondary_outcome>
  <other_outcome>
    <measure>The number of painful crises had by subjects receiving one of the three oral dose levels of SC411 compared to matching placebo will be evaluated from eDiary-records of patient reported outcomes.</measure>
    <time_frame>Week 0 (baseline) through Week 8 plus one day (end of treatment on Part A)</time_frame>
    <description>The exploratory objective of this study are to evaluate the effect of the three oral dose levels of SC411 compared to matching placebo from eDiary-records of patient reported outcomes of painful crises. The number of painful crises will be reported by the patient and evaluated based on SC411 compared to matching placebo.</description>
  </other_outcome>
  <other_outcome>
    <measure>The intensity of painful crises on a scale of 0 to 10, reported by subjects receiving one of the three oral dose levels of SC411 compared to matching placebo will be evaluated from eDiary-records of patient reported outcomes.</measure>
    <time_frame>Week 0 (baseline) through Week 8 plus one day (end of treatment on Part A)</time_frame>
    <description>The exploratory objective of this study are to evaluate the effect of the three oral dose levels of SC411 compared to matching placebo from eDiary-records of patient reported outcomes of intensity of painful crises on a scale of 0 to 10, reported by the patient. Painful crises will be reported by the patient and evaluated based on level of intensity.</description>
  </other_outcome>
  <other_outcome>
    <measure>The number of times the subject reports taking an analgesic at home by subjects receiving one of the three oral dose levels of SC411 compared to matching placebo will be evaluated from eDiary-records of patient reported outcomes.</measure>
    <time_frame>Week 0 (baseline) through Week 8 plus one day (end of treatment on Part A)</time_frame>
    <description>The exploratory objective of this study are to evaluate the effect of the three oral dose levels of SC411 compared to matching placebo from eDiary-records of patient reported outcomes of the number of times the patient reports taking an analgesic at home. The number of times the patient reports taking an analgesic at home will be reported by the patient and evaluated.</description>
  </other_outcome>
  <other_outcome>
    <measure>The number of school days missed because of pain by subjects receiving one of the three oral dose levels of SC411 compared to matching placebo will be evaluated from eDiary-records of patient reported outcomes.</measure>
    <time_frame>Week 0 (baseline) through Week 8 plus one day (end of treatment on Part A)</time_frame>
    <description>The exploratory objective of this study are to evaluate the effect of the three oral dose levels of SC411 compared to matching placebo from eDiary-records of patient reported outcomes of the number of times the patient reports taking an analgesic at home. The number of school days missed because of pain will be reported by the patient and evaluated.</description>
  </other_outcome>
  <other_outcome>
    <measure>The number of blood transfusions on subjects receiving one of the three oral dose levels of SC411 compared to matching placebo will be evaluated.</measure>
    <time_frame>Week 0 (baseline) through Week 8 plus one day (end of treatment on Part A)</time_frame>
    <description>The exploratory objectives of this study are to evaluate the effect of the three oral dose levels of SC411 compared to matching placebo on the number of blood transfusions. (acute simple blood transfusions and exchange blood transfusions).</description>
  </other_outcome>
  <number_of_arms>4</number_of_arms>
  <enrollment type="Actual">68</enrollment>
  <condition>Sickle Cell Disease</condition>
  <arm_group>
    <arm_group_label>Dose Level 1</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Target dose 20 mg/kg Docosahexaenoic acid</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Dose Level 2</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Target dose 36 mg/kg Docosahexaenoic acid</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Dose Level 3</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Target dose 60 mg/kg Docosahexaenoic acid</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Placebo</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>Soybean oil</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Docosahexaenoic Acid</intervention_name>
    <description>Oral Capsule</description>
    <arm_group_label>Dose Level 1</arm_group_label>
    <arm_group_label>Dose Level 2</arm_group_label>
    <arm_group_label>Dose Level 3</arm_group_label>
    <other_name>SC411</other_name>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Placebo</intervention_name>
    <description>Oral Capsule</description>
    <arm_group_label>Placebo</arm_group_label>
    <other_name>Soybean oil</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Aged greater than or equal to 5 years and less than or equal to 17 years at screening;&#xD;
&#xD;
          2. Has been diagnosed with SCD that includes the phenotypes HbSS, hemoglobin SC, and&#xD;
             HbS/beta-thalassemia. Hemoglobin phenotyping must be previously documented by either&#xD;
             hemoglobin high-performance liquid chromatography [HPLC] or electrophoresis at time of&#xD;
             Screening. If a patient does not have documented hemoglobin phenotyping at the time of&#xD;
             Screening, or has received a blood transfusion within the two months prior to the&#xD;
             Screening Visit, hemoglobin phenotyping should be documented by hemoglobin HPLC;&#xD;
&#xD;
          3. Has had at least two and no more than ten documented episodes of clinical sickle cell&#xD;
             crises within 12 months prior to the Screening Visit. A sickle cell crisis is defined&#xD;
             as an episode of vaso-occlusive event:&#xD;
&#xD;
               -  Painful crisis defined as new onset of pain that lasts two or more hours for&#xD;
                  which there is no explanation other than vaso-occlusion, and which requires&#xD;
                  therapy with oral or parenteral opioids, non-steroidal anti-inflammatory drugs,&#xD;
                  or other analgesics prescribed by a healthcare provider in a medical setting such&#xD;
                  as a hospital, clinic, or emergency room visit, or documented telephone&#xD;
                  management (Ballas, 2010; Heeney, 2016; Jacob, 2005); and&#xD;
&#xD;
               -  Acute chest syndrome defined as acute illness characterized by a new segmental&#xD;
                  pulmonary infiltrate on a chest x-ray, and fever (greater than or equal to&#xD;
                  38.5°C) or respiratory symptoms such as hypoxia, chest pain, tachypnea, wheezing,&#xD;
                  or cough (Ballas, 2010);&#xD;
&#xD;
          4. Is either not on hydroxyurea at the Screening Visit and does not plan on receiving it&#xD;
             during the course of the first 12 weeks of the study (Part A), or has received&#xD;
             hydroxyurea for a minimum of 6 months and, except for safety reasons, will remain on&#xD;
             the same weight-based dose of hydroxyurea from screening throughout the duration of&#xD;
             Part A of the study;&#xD;
&#xD;
          5. Parent or guardian is able to give written informed consent, and the potential&#xD;
             pediatric patient is able to provide assent in a manner approved by the Institutional&#xD;
             Review Board (IRB) and comply with the requirements of the study other than for safety&#xD;
             reasons; and&#xD;
&#xD;
          6. If sexually active, agrees to use a reliable method of birth control (eg, barrier,&#xD;
             birth control pills, abstinence) during the study and for one month following the last&#xD;
             dose of study drug.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Has a significant medical condition that required hospitalization (other than sickle&#xD;
             cell crisis) within two months of the Screening Visit;&#xD;
&#xD;
          2. Has a known allergy or hypersensitivity to fish or shellfish;&#xD;
&#xD;
          3. Has a known allergy or hypersensitivity to soy;&#xD;
&#xD;
          4. Is planning to initiate, terminate, or alter the dosing of hydroxyurea during the&#xD;
             first 12 weeks of the study, other than for safety reasons;&#xD;
&#xD;
          5. Has chronic daily use (more than 30 consecutive days during the last six months prior&#xD;
             to enrollment) of opioid analgesia for any reason;&#xD;
&#xD;
          6. Has a diagnosis of chronic pain or chronic pain syndrome (eg, chronic pain from the&#xD;
             repeated vaso-occlusive events, chronic pain from avascular necrosis);&#xD;
&#xD;
          7. Has a history of Human Immunodeficiency Virus (HIV), Hepatitis B, or Hepatitis C&#xD;
             infection;&#xD;
&#xD;
          8. Has a history of documented episode(s) of priapism within 12 months of the Screening&#xD;
             Visit;&#xD;
&#xD;
          9. Has a history of atrial or ventricular arrhythmia;&#xD;
&#xD;
         10. Has an international normalized ratio (INR) &gt;2.0, or is on regular anticoagulation&#xD;
             therapy, or has a history of a known bleeding diathesis;&#xD;
&#xD;
         11. Has thrombocytopenia (platelets less than 80,000) or is on chronic acetylsalicylic&#xD;
             acid therapy;&#xD;
&#xD;
         12. Has increased risk of stroke: documented abnormal or &quot;high conditional&quot; transcranial&#xD;
             Doppler (TCD) mean velocity (TCD V) by STOP criteria (Adams, 1998) within the&#xD;
             preceding year or has a history of known cerebrovascular disease:&#xD;
&#xD;
               -  &quot;High conditional&quot; = TCD V greater than or equal to 185 to 199 cm/sec, or TCDi V&#xD;
                  greater than or equal to 170 to 184 cm/sec, or TCD maximum V greater than or&#xD;
                  equal to 250 cm/sec; or&#xD;
&#xD;
               -  Abnormal = TCD V greater than or equal to 200 cm/sec, or abnormal high TCDi V&#xD;
                  greater than or equal to 185 cm/sec, or TCD maximum V greater than or equal to&#xD;
                  250 cm/sec;&#xD;
&#xD;
         13. Has received a blood transfusion or exchange transfusion in the three months prior to&#xD;
             the Screening Visit;&#xD;
&#xD;
         14. Has renal insufficiency (creatinine greater than 1.5 times upper limit of normal&#xD;
             [ULN], or requiring peritoneal dialysis or hemodialysis);&#xD;
&#xD;
         15. Has liver dysfunction (ALT greater than 2.0 times ULN);&#xD;
&#xD;
         16. Has other concomitant chronic medical or psychiatric condition that, in the opinion of&#xD;
             the Investigator, would compromise participation in the study or confound the&#xD;
             evaluation of the study outcome;&#xD;
&#xD;
         17. Is pregnant or lactating or has the intention of becoming pregnant during the study&#xD;
             (if a female of child-bearing potential or partner of a patient participating in the&#xD;
             study);&#xD;
&#xD;
         18. Is currently taking, or has been treated with, any form of omega-3 fatty acid or fish&#xD;
             oil supplement within 30 days of the Screening Visit or during the course of the&#xD;
             study;&#xD;
&#xD;
         19. Has been treated with an experimental anti-sickling medication/treatment within 30&#xD;
             days of the Screening Visit or during the course of the study;&#xD;
&#xD;
         20. Is currently taking or has been treated with any investigational drug for any disease&#xD;
             within 30 days of the Screening Visit or during the course of the study;&#xD;
&#xD;
         21. Is currently enrolled in an investigational drug or device study and/or has&#xD;
             participated in such a study within 30 days of the Screening Visit or during the&#xD;
             course of the study; or&#xD;
&#xD;
         22. There are factors that would, in the judgment of the Investigator, make it difficult&#xD;
             for the patient to comply with the requirements of the study (eg, inability to swallow&#xD;
             capsules due to past history of stroke, or poor compliance).&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>5 Years</minimum_age>
    <maximum_age>17 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Matthew Heeney, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Boston Children's Hospital</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Children's of Alabama - University of Alabama</name>
      <address>
        <city>Birmingham</city>
        <state>Alabama</state>
        <zip>35233</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>UCSF Benioff Children's Hospital Oakland</name>
      <address>
        <city>Oakland</city>
        <state>California</state>
        <zip>94609</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Florida Health at Shands</name>
      <address>
        <city>Gainesville</city>
        <state>Florida</state>
        <zip>32610</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Batchelor Children's Research Institute - University of Miami</name>
      <address>
        <city>Miami</city>
        <state>Florida</state>
        <zip>33136</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Healthcare of Atlanta - Emory University</name>
      <address>
        <city>Atlanta</city>
        <state>Georgia</state>
        <zip>30322</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Boston Children's Hospital</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02115</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospital of Michigan</name>
      <address>
        <city>Detroit</city>
        <state>Michigan</state>
        <zip>48201</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Mississippi Medical Center</name>
      <address>
        <city>Jackson</city>
        <state>Mississippi</state>
        <zip>39216</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>East Carolina University</name>
      <address>
        <city>Greenville</city>
        <state>North Carolina</state>
        <zip>27834</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Medical University of South Carolina</name>
      <address>
        <city>Charleston</city>
        <state>South Carolina</state>
        <zip>29425</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Texas Children's Hospital - Baylor College of Medicine</name>
      <address>
        <city>Houston</city>
        <state>Texas</state>
        <zip>77030</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <link>
    <url>https://aspho.planion.com/Web.User/AbstractDet?ACCOUNT=ASPHO&amp;ABSID=14095&amp;CONF=AM18&amp;ssoOverride=OFF&amp;CKEY</url>
    <description>EFFECTS OF SC411 ON BLOOD CELL MEMBRANE OMEGA-3 INDEX AND SELECT SICKLE CELL DISEASE BIOMARKERS IN THE SCOT TRIAL: A PHASE 2 RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP, MULTI-CENTER STUDY</description>
  </link>
  <link>
    <url>https://aspho.planion.com/Web.User/AbstractDet?ACCOUNT=ASPHO&amp;ABSID=14068&amp;CONF=AM18&amp;ssoOverride=OFF&amp;CKEY</url>
    <description>CLINICAL EFFECT OF SC411 ON CHILDREN WITH SICKLE CELL DISEASE IN THE SCOT TRIAL: A PHASE 2 RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP, DOSE-FINDING MULTI-CENTER STUDY</description>
  </link>
  <results_reference>
    <citation>Daak AA, Dampier CD, Fuh B, Kanter J, Alvarez OA, Black LV, McNaull MA, Callaghan MU, George A, Neumayr L, Hilliard LM, Sancilio F, Rabinowicz AL, Heeney MM. Double-blind, randomized, multicenter phase 2 study of SC411 in children with sickle cell disease (SCOT trial). Blood Adv. 2018 Aug 14;2(15):1969-1979. doi: 10.1182/bloodadvances.2018021444.</citation>
    <PMID>30097463</PMID>
  </results_reference>
  <verification_date>January 2019</verification_date>
  <study_first_submitted>November 22, 2016</study_first_submitted>
  <study_first_submitted_qc>November 23, 2016</study_first_submitted_qc>
  <study_first_posted type="Estimate">November 25, 2016</study_first_posted>
  <last_update_submitted>January 29, 2019</last_update_submitted>
  <last_update_submitted_qc>January 29, 2019</last_update_submitted_qc>
  <last_update_posted type="Actual">January 31, 2019</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>Anemia, Sickle Cell</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/NCT02973360
org study id: OMEG-411-02
nct id: NCT02973360
lead sponsor: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
This is a Phase 2, randomized, double-blind, placebo-controlled, parallel-group, dose-finding
      study of SC411 in children with sickle cell disease (SCD). The primary objective of the study
      is to evaluate the safety and tolerability of three different doses of SC411 compared to a
      placebo. All patients will undergo eight weeks of oral study treatment and a four-week safety
      follow-up period. Patients will be randomized to one of three dose levels of SC411 or
      placebo.
This Phase 2 study is to be conducted in two parts. Part A is a randomized, double-blind,
      placebo-controlled, parallel-group, dose-finding study of SC411 in children with SCD. This
      part of the study will consist of a screening period of up to 2 weeks, followed by an
      eight-week treatment period. Part B is an optional 49-month open-label extension for patients
      who have completed Part A.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Triple (Participant, Care Provider, Investigator)
measure: Evaluate safety & tolerability of SC411 & determine change from Baseline in the blood cells omega-3 fatty acids index in subjects, treated with active SC411 doses or placebo in Part A and safety and tolerability in Part B.
time frame: Week 0 (baseline) through Month 52 (end of treatment)
description: Evaluate the safety and tolerability of three different doses of SC411 and determine the change from Baseline in the blood cells omega-3 fatty acids index in subjects, treated with either one of the three active doses of SC411 or matching placebo in Part A. Safety and tolerability will continue to be evaluated and reported during Part B.
measure: Measurement of the pharmacokinetic (PK) parameter of AUC will be performed for three dose levels of SC411 will be measured.
time frame: 2 weeks before baseline (screening) through Week 52
description: Measurement of the pharmacokinetic (PK) parameter of AUC will be performed for three dose levels of SC411 will be measured.
The steady-state pharmacokinetic (PK) profile of the three doses and area under the curve will be reported. The purpose is to assess the steady-state pharmacokinetic (PK) profile of the three dose levels of SC411, following two months of dosing, and at the end of treatment (EOT).
measure: Measurement of the pharmacokinetic (PK) parameter of Tmax will be performed for three dose levels of SC411 will be measured.
time frame: 2 weeks before baseline (screening) through Week 52
description: Measurement of the pharmacokinetic (PK) parameter of Tmax will be performed for three dose levels of SC411 will be measured.
The steady-state pharmacokinetic (PK) profile of the three doses and Tmax will be reported. The purpose is to assess the steady-state pharmacokinetic (PK) profile of the three dose levels of SC411, following two months of dosing, and at the end of treatment (EOT).
measure: Measurement of the pharmacokinetic (PK) parameter of Cmax will be performed for three dose levels of SC411 will be measured.
time frame: 2 weeks before baseline (screening) through Week 52
description: The steady-state pharmacokinetic (PK) profile of the three doses and Cmax will be reported. The purpose is to assess the steady-state pharmacokinetic (PK) profile of the three dose levels of SC411, following two months of dosing, and at the end of treatment (EOT).
measure: The safety & long term tolerability of SC411 will be evaluated in Part A, & a selected dose in Part B. The change from Baseline in blood cells omega-3 fatty acids index in subjects, treated with SC411 or placebo in Part A will be determined.
time frame: Week 0 (baseline) through Month 52 (end of treatment)
description: Evaluate the safety and long term tolerability of three different doses of SC411 and determine the change from Baseline in the blood cells omega-3 fatty acids index in subjects, treated with either one of the three active doses of SC411 or matching placebo in Part A. Safety and long term tolerability will continue to be evaluated and reported during Part B.
measure: The number of painful crises had by subjects receiving one of the three oral dose levels of SC411 compared to matching placebo will be evaluated from eDiary-records of patient reported outcomes.
time frame: Week 0 (baseline) through Week 8 plus one day (end of treatment on Part A)
description: The exploratory objective of this study are to evaluate the effect of the three oral dose levels of SC411 compared to matching placebo from eDiary-records of patient reported outcomes of painful crises. The number of painful crises will be reported by the patient and evaluated based on SC411 compared to matching placebo.
measure: The intensity of painful crises on a scale of 0 to 10, reported by subjects receiving one of the three oral dose levels of SC411 compared to matching placebo will be evaluated from eDiary-records of patient reported outcomes.
time frame: Week 0 (baseline) through Week 8 plus one day (end of treatment on Part A)
description: The exploratory objective of this study are to evaluate the effect of the three oral dose levels of SC411 compared to matching placebo from eDiary-records of patient reported outcomes of intensity of painful crises on a scale of 0 to 10, reported by the patient. Painful crises will be reported by the patient and evaluated based on level of intensity.
measure: The number of times the subject reports taking an analgesic at home by subjects receiving one of the three oral dose levels of SC411 compared to matching placebo will be evaluated from eDiary-records of patient reported outcomes.
time frame: Week 0 (baseline) through Week 8 plus one day (end of treatment on Part A)
description: The exploratory objective of this study are to evaluate the effect of the three oral dose levels of SC411 compared to matching placebo from eDiary-records of patient reported outcomes of the number of times the patient reports taking an analgesic at home. The number of times the patient reports taking an analgesic at home will be reported by the patient and evaluated.
measure: The number of school days missed because of pain by subjects receiving one of the three oral dose levels of SC411 compared to matching placebo will be evaluated from eDiary-records of patient reported outcomes.
time frame: Week 0 (baseline) through Week 8 plus one day (end of treatment on Part A)
description: The exploratory objective of this study are to evaluate the effect of the three oral dose levels of SC411 compared to matching placebo from eDiary-records of patient reported outcomes of the number of times the patient reports taking an analgesic at home. The number of school days missed because of pain will be reported by the patient and evaluated.
measure: The number of blood transfusions on subjects receiving one of the three oral dose levels of SC411 compared to matching placebo will be evaluated.
time frame: Week 0 (baseline) through Week 8 plus one day (end of treatment on Part A)
description: The exploratory objectives of this study are to evaluate the effect of the three oral dose levels of SC411 compared to matching placebo on the number of blood transfusions. (acute simple blood transfusions and exchange blood transfusions).
arm group label: Dose Level 1
arm group type: Experimental
description: Target dose 20 mg/kg Docosahexaenoic acid
arm group label: Dose Level 2
arm group type: Experimental
description: Target dose 36 mg/kg Docosahexaenoic acid
arm group label: Dose Level 3
arm group type: Experimental
description: Target dose 60 mg/kg Docosahexaenoic acid
arm group label: Placebo
arm group type: Placebo Comparator
description: Soybean oil
intervention type: Drug
intervention name: Docosahexaenoic Acid
description: Oral Capsule
arm group label: Dose Level 1
arm group label: Dose Level 2
arm group label: Dose Level 3
other name: SC411
intervention type: Drug
intervention name: Placebo
description: Oral Capsule
arm group label: Placebo
other name: Soybean oil
criteria: 
gender: All
minimum age: 5 Years
maximum age: 17 Years
healthy volunteers: No
last name: Matthew Heeney, MD
role: Principal Investigator
affiliation: Boston Children's Hospital
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country: United States
url: https://aspho.planion.com/Web.User/AbstractDet?ACCOUNT=ASPHO&ABSID=14095&CONF=AM18&ssoOverride=OFF&CKEY
description: EFFECTS OF SC411 ON BLOOD CELL MEMBRANE OMEGA-3 INDEX AND SELECT SICKLE CELL DISEASE BIOMARKERS IN THE SCOT TRIAL: A PHASE 2 RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP, MULTI-CENTER STUDY
url: https://aspho.planion.com/Web.User/AbstractDet?ACCOUNT=ASPHO&ABSID=14068&CONF=AM18&ssoOverride=OFF&CKEY
description: CLINICAL EFFECT OF SC411 ON CHILDREN WITH SICKLE CELL DISEASE IN THE SCOT TRIAL: A PHASE 2 RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP, DOSE-FINDING MULTI-CENTER STUDY
citation: Daak AA, Dampier CD, Fuh B, Kanter J, Alvarez OA, Black LV, McNaull MA, Callaghan MU, George A, Neumayr L, Hilliard LM, Sancilio F, Rabinowicz AL, Heeney MM. Double-blind, randomized, multicenter phase 2 study of SC411 in children with sickle cell disease (SCOT trial). Blood Adv. 2018 Aug 14;2(15):1969-1979. doi: 10.1182/bloodadvances.2018021444.
PMID: 30097463
responsible party type: Sponsor
mesh term: Anemia, Sickle Cell
sharing ipd: No