Vein of Galen Malformations Clinical Trial
Official title:
Fetal Treatment of Galenic Malformations
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.
| Status | Recruiting |
| Enrollment | 20 |
| Est. completion date | December 2027 |
| Est. primary completion date | December 2025 |
| Accepts healthy volunteers | No |
| Gender | Female |
| Age group | 18 Years and older |
| Eligibility | Inclusion Criteria: - Pregnant woman carrying a fetus harboring a vein of Galen malformation in whom the straight sinus or falcine sinus draining the prosencephalic varix measures 7 mm or more on fetal MRI (medio-lateral diameter measured at the narrowest point of the sinus along the rostral-caudal axis, assessed on a T2-weighted coronal slice). - Fetal gestational age between 23 weeks and term as determined by clinical information and evaluation of first ultrasound. - Anatomic diagnosis of fetal vein of Galen malformation. - Well preserved brain parenchyma. - Maternal age of 18 years and older. - Eligible for continuous lumbar epidural anesthesia. - Able to travel to study site for study evaluation, procedures and visits. Exclusion Criteria: - Extensive fetal brain parenchymal injury/gliosis, >10% of supratentorial brain volume (i.e., SFP presentation). This is a degree of fetal brain injury beyond which the risk of significant neurological morbidity is high, based on studies of prenatal ischemic stroke. - Irreversible fetal non-brain organ injury (e.g. hydrops fetalis as a manifestation of heart failure, a finding which portends fatal outcome in fetuses with vein of Galen malformation), i.e., SFP presentation. - Fetus with VOGM in whom the straight sinus or falcine sinus draining the prosencephalic varix measures less than 8 mm on fetal MRI (T2-weighted coronal slice, medio-lateral diameter measured at the narrowest point of the sinus along the anterior-posterior axis), fitting fetal MRI criteria for likely evolution into the IT cohort. - Severe maternal obesity pre-pregnancy as defined by body mass index (BMI) of 40 or greater. - Fetuses with major congenital anomalies. - Evidence of preterm labor, rupture of membranes or abruption. - Maternal coagulopathy: INR > 1.2; PT/PTT above normal ranges for the lab; platelets <100. - Medical disease requiring current anticoagulation including maternal deep vein thrombosis. - Prior maternal medical history that would preclude epidural anesthesia. - Multi-fetal pregnancy. - Placenta previa or accreta. - Participation in another fetal study that influences maternal and fetal morbidity and mortality. - Known maternal hypersensitivity to 316LM stainless steel. - Supine hypotensive syndrome. |
| Country | Name | City | State |
|---|---|---|---|
| United States | Boston Children's Hospital | Boston | Massachusetts |
| Lead Sponsor | Collaborator |
|---|---|
| Darren Orbach |
United States,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Prenatal safety of fetal embolization for patients with vein of Galen malformations | 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, probably related to the fetal intervention, e.g. placental abruption with subsequent sequelae Preterm delivery < 28 weeks, probably related to the fetal intervention Maternal blood transfusion or unanticipated surgical intervention, probably related to the fetal intervention Presence of fetal imaging evidence of new brain injury in a location or pattern unexpected for the natural history of vein of Galen malformation, probably related to the fetal intervention |
From fetal embolization to delivery | |
| Secondary | Efficacy of fetal embolization for patients with vein of Galen malformations | 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. |
Within 30 days of birth |