MyelomeningocelePhase 1/2CuReNCT04652908
Cellular Therapy for In Utero Repair of Myelomeningocele - The CuRe Trial
A Phase 1/2 study testing Placental Mesenchymal Stem Cells seeded on a commercially available dural graft extracellular matrix and Untreated contemporaneous cohort in people with Myelomeningocele, run by University of California, Davis at 1 site in United States.
Trial summary
Spina bifida, or myelomeningocele (MMC), is a birth defect that results in paralysis, excess fluid on the brain (hydrocephalus), and impaired ability to urinate and have bowel movements normally. Read more about this study
- Who is it for?
- Ages 19 to 25 weeks
- Conditions the study is for
- Myelomeningocele
- What is being tested?
- Placental Mesenchymal Stem Cells seeded on a commercially available dural graft extracellular matrix and Untreated contemporaneous cohort
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 55 participants
- Where
- 1 siteUnited States
- Study dates
- 21 Jun 2021to Mar 2027
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 19 to 25 weeks
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Eligibility for fetal surgery per the MOMS trial, which are:
- Myelomeningocele (including myeloschisis) at any level from T1 through S1 with hindbrain herniation. Lesion level will be confirmed by ultrasound and hindbrain herniation will be confirmed by MRI at the UC Davis Fetal Center
- Maternal age ≥18 years
- Gestational age at enrollment between 19 weeks 0 days and 25 weeks 6 days gestation as determined by clinical information and evaluation of first ultrasound
- Normal karyotype. Results by fluorescence in situ hybridization (FISH) will be acceptable if the patient is greater than 24 weeks gestation;
Who cannot join
- Not being eligible for fetal surgery per the MOMS trial, which includes:
- Multifetal pregnancy
- Insulin dependent pregestational diabetes
- Fetal anomaly not related to myelomeningocele.
- Kyphosis in the fetus of 30 degrees or more
- Current or planned cerclage or documented history of incompetent cervix, placenta previa or placental abruption
Show all 20Show fewer
- Short cervix < 20 mm measured by cervical ultrasound
- Obesity as defined by body mass index of 35 or greater
- Previous spontaneous singleton delivery prior to 37 weeks
- Maternal-fetal Rh isoimmunization, Kell sensitization or a history of neonatal alloimmune thrombocytopenia
- Maternal HIV or Hepatitis-B status positive due to the increased risk of transmission to the fetus during maternal-fetal surgery. If the patient's HIV or Hepatitis B status is unknown, the patient must be tested and found to have negative results before she can be enrolled
- Known Hepatitis-C positivity. If the patient's Hepatitis C status is unknown, she does not need to be screened
- Uterine anomaly such as large or multiple fibroids or Müllerian duct abnormality
- Other maternal medical condition which is a contraindication to surgery or general anesthesia. This includes any patient with a previous hysterotomy in the active segment of the uterus (whether from a previous classical cesarean, uterine anomaly such as an arcuate or bicornuate uterus, major myomectomy resection, or previous fetal surgery)
- Patient does not have a support person (e.g., husband, partner, mother)
- Inability to comply with the travel and follow-up requirements of fetal surgery
- Patient does not meet other psychosocial criteria (as determined by the psychosocial interviewer) to handle the implications of fetal surgery
- Participation in another intervention study that influences maternal and fetal morbidity and mortality or participation in this trial in a previous pregnancy;
- Maternal hypertension which would increase the risk of preeclampsia or preterm delivery (including, but not limited to: uncontrolled hypertension, chronic hypertension with end organ damage and new onset hypertension in current pregnancy)
- Active COVID-19 infection at time of fetal surgery as determined by positive test
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
UC Davis Health
Sacramento, California, 95817
Amy B Powne, MSN, RN, 916-794-2229, fctc@health.ucdavis.edu
Maria G Hernandez, 916-734-4156, mghernandez@health.ucdavis.edu
About this study
Official title: Phase 1/2a Trial of Placental Mesenchymal Stem Cells for Repair of Fetal Myelomeningocele
Spina bifida, or myelomeningocele (MMC), is a birth defect that results in paralysis, excess fluid on the brain (hydrocephalus), and impaired ability to urinate and have bowel movements normally. In a previous study (the MOMS trial), surgery before birth (in-utero/fetal surgery) was shown to reduce the need for shunting for hydrocephalus. There was also some improvement in ambulation, but 58 % of the children still could not walk unassisted.
This study is testing living stem cells from placenta added to the fetal repair in an effort to improve the ability to walk. Previous animal studies have shown dramatic improvement in walking and bowel and bladder function when placental stem cells are added to MMC repair. Use of these "living" cells may protect the developing spinal cord, prevent further injury, and may even reverse existing damage to the nerves that control movement. This study is assessing the safety and efficacy of adding stem cells to open fetal surgery for MMC in humans.
Read the detailed description
Historically, treatment of MMC was limited to post-natal surgery to close the dura and skin over the spinal cord to prevent meningitis, which had no effect on motor function. The potential benefit of earlier intervention was realized when prenatal ultrasound of patients with MMC early in gestation revealed near-normal leg movements despite displaying paralysis at birth. This finding gave credence to the two-hit hypothesis that paralysis was progressive during prenatal life and suggested that fetal intervention could prevent the secondary damage to the spinal cord. Fetal repair of MMC did confer improvement in motor function of children treated in the Management of Myelomeningocele (MOMS) randomized controlled trial. The promising results of the MOMS trial demonstrated the potential for improvement of paralysis for these patients, but distal motor function still remained severely impaired in the majority of patients with MMC with standard in utero repair alone. While this demonstrated that the ideal time to intervene to prevent paralysis is in utero with the goal of preventing the accrual of ongoing damage to the spinal cord, there is still room for improvement. The remarkable regenerative capacity of the fetal environment combined with regenerative capacity of placental mesenchymal stem cells offers potential for augmentation of the fetal repair of MMC with a novel therapy to further reduce and repair the sustained spinal cord damage.
- Conditions
- Myelomeningocele
- Being tested
- Placental Mesenchymal Stem Cells seeded on a commercially available dural graft extracellular matrix, Untreated contemporaneous cohort
- Medical subject headings
- Meningomyelocele
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Placental Mesenchymal Stem Cells seeded on a commercially available dural graft extracellular matrixBiological
Also called PMSC-ECM
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.
Untreated contemporaneous cohortOther
Also called non-PMSC-ECM
The addition of a non-PMSC treated cohort, the untreated contemporaneous cohort, has been added at the request of the FDA to provide contemporaneous patients for validation of the continued relevance of use of the outcomes of the MOMS trial as the comparison arm for the Phase 2a portion of the study.
| Study group | Role | What they receive |
|---|---|---|
| Treatment with PMSC-ECM | Experimental | One-time administration of PMSC-ECM during the course of in utero fetal myelomeningocele surgery will be administered Receives: Biological: Placental Mesenchymal Stem Cells seeded on a commercially available dural graft extracellular matrix |
| non-PMSC untreated contemporaneous cohort | Other | Contemporaneous cohort of patients undergoing routine fetal or postnatal MMC repair without PMSC-ECM (non-PMSC untreated contemporaneous cohort). Receives: Other: Untreated contemporaneous cohort |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of the placenta-derived mesenchymal stem cell (PMSC-ECM) Product
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.
Measured: Assessed at birth
Secondary outcomes (1)
Efficacy of the PMSC-ECM Product
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.
Measured: 30 months.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Dec 2020
- Study start21 Jun 2021
- Primary completionMar 2027 (estimated)
- Study completionMar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for MyelomeningoceleStudies of the same condition, open and under way.
- More trials run by University of California, DavisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- Treatment arm subjects receiving PMSC-ECM (Placental Mesenchymal Stem Cells seeded on a commercially available dural graft extracellular matrix). Additionally, we will follow a contemporaneous cohort of patients undergoing routine fetal or postnatal MMC repair without PMSC-ECM (non-PMSC untreated contemporaneous cohort). 35 participants will be enrolled under the treatment arm and 20 participants will be enrolled under the untreated contemporaneous cohort. The addition of a non-PMSC treated cohort, the untreated contemporaneous cohort, has been added at the request of the FDA to provide contemporaneous patients for validation of the continued relevance of use of the outcomes of the MOMS trial as the comparison arm for the Phase 2a portion of the study.
- Masking
- None (open label)
- Enrolment
- 55 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of California, Davis
- Sponsor type
- University, hospital or other
- Collaborators
- California Institute for Regenerative Medicine (CIRM)
- Responsible party
- Sponsor
- Investigators
- Diana L Farmer, MD, Principal investigator, UC Davis School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04652908
- Sponsor's protocol ID
- 1617774
- Acronym
- CuRe
- Status last verified
- Jan 2026
- First submitted
- 25 Nov 2020
- Last updated
- 20 Jan 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Farmer DL, Kumar P, Reynolds E, Lee SY, Powne AB, Pivetti CD, Zwienenberg M, McLennan AS, Nolta JA, Brown EG, Saadai P, Hirose S, Wang A. Feasibility and safety of cellular therapy for in-utero repair of myelomeningocele (CuRe Trial): a first-in-human, phase 1, single-arm study. Lancet. 2026 Feb 28;407(10531):867-875. doi: 10.1016/S0140-6736(25)02466-3.
PubMed 41763744
Study at a glance
From the registry record, last updated by the sponsor on 20 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1/2a Trial of Placental Mesenchymal Stem Cells for Repair of Fetal Myelomeningocele
- ClinicalTrials.gov ID
- NCT04652908
- Acronym / sponsor ID
- CuRe / 1617774
- Conditions
- Myelomeningocele
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 55 participants (estimated)
- Lead sponsor
- University of California, DavisUniversity, hospital or other
- Collaborators
- California Institute for Regenerative Medicine (CIRM)
- Study start
- 21 Jun 2021 (actual)
- Primary completion
- Mar 2027 (estimated)
- Study completion
- Mar 2027 (estimated)
- First posted
- 3 Dec 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Spina bifida, or myelomeningocele (MMC), is a birth defect that results in paralysis, excess fluid on the brain (hydrocephalus), and impaired ability to urinate and have bowel movements normally. In a previous study (the MOMS trial), surgery before birth (in-utero/fetal surgery) was shown to reduce the need for shunting for hydrocephalus. There was also some improvement in ambulation, but 58 % of the children still could not walk unassisted.
This study is testing living stem cells from placenta added to the fetal repair in an effort to improve the ability to walk. Previous animal studies have shown dramatic improvement in walking and bowel and bladder function when placental stem cells are added to MMC repair. Use of these "living" cells may protect the developing spinal cord, prevent further injury, and may even reverse existing damage to the nerves that control movement. This study is assessing the safety and efficacy of adding stem cells to open fetal surgery for MMC in humans.
Detailed description
Historically, treatment of MMC was limited to post-natal surgery to close the dura and skin over the spinal cord to prevent meningitis, which had no effect on motor function. The potential benefit of earlier intervention was realized when prenatal ultrasound of patients with MMC early in gestation revealed near-normal leg movements despite displaying paralysis at birth. This finding gave credence to the two-hit hypothesis that paralysis was progressive during prenatal life and suggested that fetal intervention could prevent the secondary damage to the spinal cord. Fetal repair of MMC did confer improvement in motor function of children treated in the Management of Myelomeningocele (MOMS) randomized controlled trial. The promising results of the MOMS trial demonstrated the potential for improvement of paralysis for these patients, but distal motor function still remained severely impaired in the majority of patients with MMC with standard in utero repair alone. While this demonstrated that the ideal time to intervene to prevent paralysis is in utero with the goal of preventing the accrual of ongoing damage to the spinal cord, there is still room for improvement. The remarkable regenerative capacity of the fetal environment combined with regenerative capacity of placental mesenchymal stem cells offers potential for augmentation of the fetal repair of MMC with a novel therapy to further reduce and repair the sustained spinal cord damage.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Treatment arm subjects receiving PMSC-ECM (Placental Mesenchymal Stem Cells seeded on a commercially available dural graft extracellular matrix). Additionally, we will follow a contemporaneous cohort of patients undergoing routine fetal or postnatal MMC repair without PMSC-ECM (non-PMSC untreated contemporaneous cohort). 35 participants will be enrolled under the treatment arm and 20 participants will be enrolled under the untreated contemporaneous cohort. The addition of a non-PMSC treated cohort, the untreated contemporaneous cohort, has been added at the request of the FDA to provide contemporaneous patients for validation of the continued relevance of use of the outcomes of the MOMS trial as the comparison arm for the Phase 2a portion of the study.
| Arm | Type | Receives |
|---|---|---|
| Treatment with PMSC-ECM | Experimental | Biological: Placental Mesenchymal Stem Cells seeded on a commercially available dural graft extracellular matrixOne-time administration of PMSC-ECM during the course of in utero fetal myelomeningocele surgery will be administered |
| non-PMSC untreated contemporaneous cohort | Other | Other: Untreated contemporaneous cohortContemporaneous cohort of patients undergoing routine fetal or postnatal MMC repair without PMSC-ECM (non-PMSC untreated contemporaneous cohort). |
Who may qualify
- Ages
- 19 to 25 weeks
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Myelomeningocele (including myeloschisis) at any level from T1 through S1 with hindbrain herniation. Lesion level will be confirmed by ultrasound and hindbrain herniation will be confirmed by MRI at the UC Davis Fetal Center
- Maternal age ≥18 years
- Gestational age at enrollment between 19 weeks 0 days and 25 weeks 6 days gestation as determined by clinical information and evaluation of first ultrasound
- Normal karyotype. Results by fluorescence in situ hybridization (FISH) will be acceptable if the patient is greater than 24 weeks gestation;
Key exclusion criteria (20 listed)
- Multifetal pregnancy
- Insulin dependent pregestational diabetes
- Fetal anomaly not related to myelomeningocele.
- Kyphosis in the fetus of 30 degrees or more
- Current or planned cerclage or documented history of incompetent cervix, placenta previa or placental abruption
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Placental Mesenchymal Stem Cells seeded on a commercially available dural graft extracellular matrixAlso: PMSC-ECMAs 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. | Biological | Treatment with PMSC-ECM |
| Untreated contemporaneous cohortAlso: non-PMSC-ECMThe addition of a non-PMSC treated cohort, the untreated contemporaneous cohort, has been added at the request of the FDA to provide contemporaneous patients for validation of the continued relevance of use of the outcomes of the MOMS trial as the comparison arm for the Phase 2a portion of the study. | Other | non-PMSC untreated contemporaneous cohort |
Primary outcome
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.
1 secondary outcome
Efficacy of the PMSC-ECM Product
Time frame: 30 months.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Jan 2026.
- Derived from this study: PubMed 41763744Farmer DL, Kumar P, Reynolds E, Lee SY, Powne AB, Pivetti CD, Zwienenberg M, McLennan AS, Nolta JA, Brown EG, Saadai P, Hirose S, Wang A. Feasibility and safety of cellular therapy for in-utero repair of myelomeningocele (CuRe Trial): a first-in-human, phase 1, single-arm study. Lancet. 2026 Feb 28;407(10531):867-875. doi: 10.1016/S0140-6736(25)02466-3.
- Individual participant dataSharing planned: No
Related trials for Myelomeningocele
Recruiting
Transcutaneous Spinal Stimulation for Children and Youth With Spina Bifida
Myelomeningocele, Spina Bifida
Active, not recruitingPhase 1
Patch vs. No Patch Fetoscopic Meningomyelocele Repair Study
Neural Tube Defects
Recruiting
Autologous Human Umbilical Cord Tissue Patch for Postnatal Closure of Open Neural Tube Defects
Spina Bifida
Recruiting
tSCS in Children With Spina Bifida
Spina Bifida, Myelomeningocele
Active, not recruiting
Fetoscopic NEOX Cord 1K® Spina Bifida Repair
Spina Bifida, Myelomeningocele
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Diana L Farmer, MD principal investigator
UC Davis School of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Placental Mesenchymal Stem Cells seeded on a commercially available dural graft extracellular matrix and Untreated contemporaneous cohort in people with Myelomeningocele, run by University of California, Davis at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 20 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Amy B Powne, MSN, RN (916-794-2229, fctc@health.ucdavis.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety of the placenta-derived mesenchymal stem cell (PMSC-ECM) Product (time frame: Assessed at birth). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04652908, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jan 2026.