Cellular Therapy for In Utero Repair of Myelomeningocele - The CuRe Trial (CuRe)
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|ClinicalTrials.gov Identifier: NCT04652908|
Recruitment Status : Not yet recruiting
First Posted : December 3, 2020
Last Update Posted : December 3, 2020
Spina bifida, or myelomeningocele (MMC), is a disorder where the lower part of the spinal cord of the fetus is exposed, meaning there is no bone or skin covering it. This is dangerous because the spinal cord contains cells which control one's ability to move their legs and walk, and also to be able to urinate and have bowel movements normally. One of the current treatments for fetal MMC is to perform a surgery on the fetus before it is born which has many names including in utero surgery, prenatal surgery, or fetal surgery. This is a surgery that occurs inside the uterus (the womb) where the surgeon closes the opening in your fetus' back to cover the exposed spinal cord.
Researchers have found that adding stem cells to the repair is effective in improving the ability of animals with MMC to walk, and that the stem cells are safe in animal studies. These stem cells are thought to protect the cells in the spinal cord that control movement and developmental outcomes. This study is being performed to look at the safety and effectiveness of stem cells on the fetus's exposed spinal cord during prenatal surgery.
|Condition or disease||Intervention/treatment||Phase|
|Myelomeningocele||Biological: Placental Mesenchymal Stem Cells seeded on a commercially available dural graft extracellular matrix||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||35 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase 1/2a Trial of Placental Mesenchymal Stem Cells for Repair of Fetal Myelomeningocele|
|Estimated Study Start Date :||February 2021|
|Estimated Primary Completion Date :||February 2024|
|Estimated Study Completion Date :||February 2024|
Experimental: Treatment with PMSC-ECM
One-time administration of PMSC-ECM during the course of in utero fetal myelomeningocele surgery will be administered
Biological: Placental Mesenchymal Stem Cells seeded on a commercially available dural graft extracellular matrix
As in the current standard fetal surgery, under sonographic guidance, initial uterine entry will be accomplished by uterine stapling device or similar. The fetus will be given an intramuscular injection of pain medications and paralytic. The myelomeningocele will be closed in a standardized manner under magnification. As in the standard fetal operation, the spinal cord will be dissected from surrounding tissue and allowed to drop into the spinal canal. The PMSC-ECM product will then be tailored to the size of the spinal cord and applied topically, cell side down. The PMSC-ECM product will be sutured in place to the dura. Finally, the fetal skin will be closed in the standard fashion. The amniotic fluid volume will be replaced and antibiotics will be added. The uterus will be closed. The abdominal fascial layer and skin will be closed in routine fashion.
Other Name: PMSC-ECM
- Safety of the placenta-derived mesenchymal stem cell (PMSC-ECM) Product [ Time Frame: Assessed at birth ]Will be assessed by evaluating the presence or absence of cerebrospinal fluid leak, infection at the MMC repair site, failure of the MMC repair site to heal, and any unexpected growths or tumor formation. These will be assessed at birth by physical exam, brain and spinal ultrasound , and brain and spinal MRI.
- Efficacy of the PMSC-ECM Product [ Time Frame: 30 months. ]This is primarily evaluated by improvement in motor function 2 or more levels greater than expected by anatomic level of the defect and by patients' ability to walk independently. Bowel function will be assessed by caregiver questionnaires on bowel habits, and by anorectal manometry. Urologic function will be assessed by caregiver questionnaires regarding urologic function, by renal and bladder ultrasounds to evaluate for hydronephrosis and bladder abnormalities, and by video urodynamics.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04652908
|Contact: Amy B Powne, MSN, RNemail@example.com|
|Principal Investigator:||Diana L Farmer, MD||UC Davis School of Medicine|