<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/NCT04434729</url>
  </required_header>
  <id_info>
    <org_study_id>IRB-P00034727</org_study_id>
    <secondary_id>2020P000216</secondary_id>
    <nct_id>NCT04434729</nct_id>
  </id_info>
  <brief_title>Fetal Treatment of Galenic Malformations</brief_title>
  <official_title>Fetal Treatment of Galenic Malformations</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Darren Orbach</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Boston Children's Hospital</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>Yes</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      This is a prospective, single-arm non-randomized interventional study of fetuses to assess&#xD;
      the safety and efficacy of fetal embolization of Vein of Galen Malformation (VOGM). Subjects&#xD;
      will receive a one-time study intervention of fetal embolization. Follow-up assessments will&#xD;
      be collected every 4 weeks until delivery, as per standard of care. After delivery,&#xD;
      neurological assessments will be performed every 6 months for 2 years (adjusted for&#xD;
      gestational age). Data will be compared to historical cohorts.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      The study involves a single fetal intervention of maternal transuterine, fetal transcranial&#xD;
      torcular puncture and median prosencephalic vein embolization. Detachable platinum coils&#xD;
      (Target XL and XXL Detachable Coil, Stryker Neurovascular) will be used to pack the&#xD;
      prosencephalic varix. This procedure will take place in an obstetric OR at Brigham and&#xD;
      Women's Hospital. Data will be collected every 4 weeks post-procedure until delivery, as per&#xD;
      standard of care. Data regarding delivery and post-delivery status will also be collected&#xD;
      until the subject is discharged from the hospital. After delivery, neurological assessments&#xD;
      will be performed every 6 months for 2 years (adjusted for gestational age). Data will be&#xD;
      compared to historical cohorts.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">September 30, 2022</start_date>
  <completion_date type="Anticipated">December 2025</completion_date>
  <primary_completion_date type="Anticipated">December 2023</primary_completion_date>
  <phase>N/A</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>Prenatal safety of fetal embolization for patients with vein of Galen malformations</measure>
    <time_frame>From fetal embolization to delivery</time_frame>
    <description>The procedure is deemed safe if&#xD;
None of the following unacceptable events occur within 7 days of fetal embolization:&#xD;
Fetal death&#xD;
Fetal intracranial hemorrhage, either parenchymal or extra-axial, other than petechial hemorrhage.&#xD;
Maternal death&#xD;
None of the following unacceptable events occur between fetal embolization and delivery:&#xD;
Intra-procedural and post-procedural morbidity to the fetus and mother&#xD;
Failure of progression of the pregnancy to term (or expected delivery date, as determined by the obstetrician)&#xD;
Preterm delivery due to abruption, infection, rupture of membranes or contractions&#xD;
Maternal blood transfusion or unanticipated surgical intervention&#xD;
Presence of fetal imaging evidence of new brain injury other than hemorrhage</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Efficacy of fetal embolization for patients with vein of Galen malformations</measure>
    <time_frame>Within 30 days of birth</time_frame>
    <description>The procedure is deemed efficacious if none of the following serious postnatal events occur within 30 days of birth:&#xD;
Urgent neonatal embolization is needed. Without fetal intervention, we'd expect 80% of the cohort to require such intervention.&#xD;
Neonatal death. Without fetal intervention, we'd expect a mortality rate of 40% in this cohort.&#xD;
Brain MRI within the first three weeks after birth reveals parenchymal brain injury (acute infarct or gliosis) affecting &gt; 10% of the supratentorial brain volume. Without fetal intervention, we'd expect 30% of patients in this cohort to show this kind of injury.</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Anticipated">20</enrollment>
  <condition>Vein of Galen Malformations</condition>
  <arm_group>
    <arm_group_label>Fetal embolization of vein of Galen malformation</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>This is a single-arm study. Fetal subjects will undergo a one-time intervention of fetal embolization of vein of Galen malformation.</description>
  </arm_group>
  <intervention>
    <intervention_type>Device</intervention_type>
    <intervention_name>Fetal embolization of vein of Galen malformation using Target XL and XXL Detachable Coils</intervention_name>
    <description>The study involves a single fetal intervention of maternal transuterine, fetal transcranial torcular puncture and median prosencephalic vein embolization. Detachable platinum coils (Target XL and XXL Detachable Coil, Stryker Neurovascular) will be used to pack the prosencephalic varix.</description>
    <arm_group_label>Fetal embolization of vein of Galen malformation</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Pregnant woman carrying a fetus harboring a vein of Galen malformation in whom the&#xD;
             straight sinus or falcine sinus draining the prosencephalic varix measures 8 mm or&#xD;
             more on fetal MRI (medio-lateral diameter measured at the narrowest point of the sinus&#xD;
             along the rostral-caudal axis, assessed on a T2-weighted coronal slice).&#xD;
&#xD;
          -  Fetal gestational age between 23 weeks and term as determined by clinical information&#xD;
             and evaluation of first ultrasound.&#xD;
&#xD;
          -  Anatomic diagnosis of fetal vein of Galen malformation.&#xD;
&#xD;
          -  Well preserved brain parenchyma.&#xD;
&#xD;
          -  Maternal age of 18 years and older.&#xD;
&#xD;
          -  Eligible for continuous lumbar epidural anesthesia.&#xD;
&#xD;
          -  Able to travel to study site for study evaluation, procedures and visits.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Extensive fetal brain parenchymal injury/gliosis, &gt;10% of supratentorial brain volume&#xD;
             (i.e., SFP presentation). This is a degree of fetal brain injury beyond which the risk&#xD;
             of significant neurological morbidity is high, based on studies of prenatal ischemic&#xD;
             stroke.&#xD;
&#xD;
          -  Irreversible fetal non-brain organ injury (e.g. hydrops fetalis as a manifestation of&#xD;
             heart failure, a finding which portends fatal outcome in fetuses with vein of Galen&#xD;
             malformation), i.e., SFP presentation.&#xD;
&#xD;
          -  Fetus with VOGM in whom the straight sinus or falcine sinus draining the&#xD;
             prosencephalic varix measures less than 8 mm on fetal MRI (T2-weighted coronal slice,&#xD;
             medio-lateral diameter measured at the narrowest point of the sinus along the&#xD;
             anterior-posterior axis), fitting fetal MRI criteria for likely evolution into the IT&#xD;
             cohort.&#xD;
&#xD;
          -  Severe maternal obesity pre-pregnancy as defined by body mass index (BMI) of 40 or&#xD;
             greater.&#xD;
&#xD;
          -  Fetuses with major congenital anomalies.&#xD;
&#xD;
          -  Evidence of preterm labor, rupture of membranes or abruption.&#xD;
&#xD;
          -  Maternal coagulopathy: INR &gt; 1.2; PT/PTT above normal ranges for the lab; platelets&#xD;
             &lt;100.&#xD;
&#xD;
          -  Medical disease requiring current anticoagulation including maternal deep vein&#xD;
             thrombosis.&#xD;
&#xD;
          -  Prior maternal medical history that would preclude epidural anesthesia.&#xD;
&#xD;
          -  Multi-fetal pregnancy.&#xD;
&#xD;
          -  Placenta previa or accreta.&#xD;
&#xD;
          -  Participation in another fetal study that influences maternal and fetal morbidity and&#xD;
             mortality.&#xD;
&#xD;
          -  Known maternal hypersensitivity to 316LM stainless steel.&#xD;
      </textblock>
    </criteria>
    <gender>Female</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Darren Orbach, MD PhD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Boston Children's Hospital</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Darren Orbach, MD PhD</last_name>
    <phone>617-355-5012</phone>
    <email>Darren.Orbach@childrens.harvard.edu</email>
  </overall_contact>
  <location>
    <facility>
      <name>Boston Children's Hospital</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02115</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Darren Orbach, MD PhD</last_name>
      <phone>617-355-5012</phone>
      <email>Darren.Orbach@childrens.harvard.edu</email>
    </contact>
    <investigator>
      <last_name>Darren Orbach, MD PhD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>January 2023</verification_date>
  <study_first_submitted>June 13, 2020</study_first_submitted>
  <study_first_submitted_qc>June 13, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">June 17, 2020</study_first_posted>
  <last_update_submitted>January 9, 2023</last_update_submitted>
  <last_update_submitted_qc>January 9, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">January 10, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor-Investigator</responsible_party_type>
    <investigator_affiliation>Boston Children's Hospital</investigator_affiliation>
    <investigator_full_name>Darren Orbach</investigator_full_name>
    <investigator_title>Chief, Neurointerventional Radiology</investigator_title>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Vein of Galen Malformations</mesh_term>
    <mesh_term>Congenital Abnormalities</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/NCT04434729
org study id: IRB-P00034727
secondary id: 2020P000216
nct id: NCT04434729
lead sponsor: 
has dmc: Yes
is fda regulated drug: No
is fda regulated device: Yes
This is a prospective, single-arm non-randomized interventional study of fetuses to assess
      the safety and efficacy of fetal embolization of Vein of Galen Malformation (VOGM). Subjects
      will receive a one-time study intervention of fetal embolization. Follow-up assessments will
      be collected every 4 weeks until delivery, as per standard of care. After delivery,
      neurological assessments will be performed every 6 months for 2 years (adjusted for
      gestational age). Data will be compared to historical cohorts.
The study involves a single fetal intervention of maternal transuterine, fetal transcranial
      torcular puncture and median prosencephalic vein embolization. Detachable platinum coils
      (Target XL and XXL Detachable Coil, Stryker Neurovascular) will be used to pack the
      prosencephalic varix. This procedure will take place in an obstetric OR at Brigham and
      Women's Hospital. Data will be collected every 4 weeks post-procedure until delivery, as per
      standard of care. Data regarding delivery and post-delivery status will also be collected
      until the subject is discharged from the hospital. After delivery, neurological assessments
      will be performed every 6 months for 2 years (adjusted for gestational age). Data will be
      compared to historical cohorts.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Prenatal safety of fetal embolization for patients with vein of Galen malformations
time frame: From fetal embolization to delivery
description: The procedure is deemed safe if
None of the following unacceptable events occur within 7 days of fetal embolization:
Fetal death
Fetal intracranial hemorrhage, either parenchymal or extra-axial, other than petechial hemorrhage.
Maternal death
None of the following unacceptable events occur between fetal embolization and delivery:
Intra-procedural and post-procedural morbidity to the fetus and mother
Failure of progression of the pregnancy to term (or expected delivery date, as determined by the obstetrician)
Preterm delivery due to abruption, infection, rupture of membranes or contractions
Maternal blood transfusion or unanticipated surgical intervention
Presence of fetal imaging evidence of new brain injury other than hemorrhage
measure: Efficacy of fetal embolization for patients with vein of Galen malformations
time frame: Within 30 days of birth
description: The procedure is deemed efficacious if none of the following serious postnatal events occur within 30 days of birth:
Urgent neonatal embolization is needed. Without fetal intervention, we'd expect 80% of the cohort to require such intervention.
Neonatal death. Without fetal intervention, we'd expect a mortality rate of 40% in this cohort.
Brain MRI within the first three weeks after birth reveals parenchymal brain injury (acute infarct or gliosis) affecting > 10% of the supratentorial brain volume. Without fetal intervention, we'd expect 30% of patients in this cohort to show this kind of injury.
arm group label: Fetal embolization of vein of Galen malformation
arm group type: Experimental
description: This is a single-arm study. Fetal subjects will undergo a one-time intervention of fetal embolization of vein of Galen malformation.
intervention type: Device
intervention name: Fetal embolization of vein of Galen malformation using Target XL and XXL Detachable Coils
description: The study involves a single fetal intervention of maternal transuterine, fetal transcranial torcular puncture and median prosencephalic vein embolization. Detachable platinum coils (Target XL and XXL Detachable Coil, Stryker Neurovascular) will be used to pack the prosencephalic varix.
arm group label: Fetal embolization of vein of Galen malformation
criteria: 
gender: Female
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Darren Orbach, MD PhD
role: Principal Investigator
affiliation: Boston Children's Hospital
last name: Darren Orbach, MD PhD
phone: 617-355-5012
email: Darren.Orbach@childrens.harvard.edu
facility: 
status: Recruiting
contact: 
investigator: 
country: United States
responsible party type: Sponsor-Investigator
investigator affiliation: Boston Children's Hospital
investigator full name: Darren Orbach
investigator title: Chief, Neurointerventional Radiology
mesh term: Vein of Galen Malformations
mesh term: Congenital Abnormalities
sharing ipd: No