Open Spina BifidaNCT04356703
Fetoscopic Open Spina Bifida Repair Using the SAFER Technique
An observational study of Open Spina Bifida and Myelomeningocele, run by Hospital Israelita Albert Einstein at 1 site in Brazil.
Trial summary
Children submitted to fetoscopic in utero myelomeningocele repair using the SAFER (Skin-over-biocellulose for Anternatal FEtoscopic Repair) technique will evaluate at 30 months or more, regarding ambulation, neurodevelopment, urinary and fecal status. Read more about this study
- Who is it for?
- Ages 12 months to 6 years
- Conditions the study is for
- Open Spina Bifida, Myelomeningocele, Meningomyelocele
- What is being tested?
- Neuropsicomotor developmental test
- How many people
- 30 participants
- Where
- 1 siteBrazil
- Study dates
- 1 Apr 2017to 1 Mar 2020
- Phase
- Observational
- Are there results?
- None posted in the registry
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 12 months to 6 years
- All sexes
- No healthy volunteers
- Child
Study population: Children submitted to prenatal repair of open spina bifida using the fetoscopic percutaneous repair technique - SAFER
Who may be able to join
- Children operated in utero using the SAFER technique
Who cannot join
- Death before 30 month
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Impact of Prenatal Correction of Spina Bifida Using Fetoscopy and the SAFER Technique on Long-term Neurodevelopment.
Children submitted to fetoscopic in utero myelomeningocele repair using the SAFER (Skin-over-biocellulose for Anternatal FEtoscopic Repair) technique will evaluate at 30 months or more, regarding ambulation, neurodevelopment, urinary and fecal status.
Read the detailed description
Children submitted to fetoscopic in utero myelomeningocele repair using the SAFER (Skin-over-biocellulose for Anternatal FEtoscopic Repair) technique will evaluate the neuropsicomotor development at 30 months of chronological age. The mental evaluation will use the Bayley III and thePEDI neurodevelopmental scale. The motor development will use the difference between anatomic and motor level of the lesion. Urinary and fecal continence will be evaluated using clinical and ultrasonographic parameters. For the anatomical level determination, immaging of the spine at the lesion level will be used. Motor level and ambulation will be evaluated through standard clinical examination.
- Conditions
- Open Spina Bifida, Myelomeningocele, Meningomyelocele, Chiari Malformation With Spina Bifida, Chiari Malformation Type 2
- Being tested
- Neuropsicomotor developmental test
- Keywords
- myelomeningocele, spina bifida, meningomyelocele, fetal surgery, fetoscopy, fetal disease, fetal treatment
- Medical subject headings
- Spina Bifida Cystica, Meningomyelocele, Arnold-Chiari Malformation, Spinal Dysraphism, Fetal Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Neuropsicomotor developmental testOther
Children will be evaluated trough Bayley III and PEDI scale. They also will be examined by pediatrician, neurosurgeon, orthopedics and phisiatry
| Study group | Role | What they receive |
|---|---|---|
| Children submitted to fetoscopic surgery | Children submitted to fetoscopic in utero myelomeningocele repair using the SAFER (Skin-over-biocellulose for Anternatal FEtoscopic Repair) technique will evaluate the neuropsicomotor development at 30 months of chronological age or older Receives: Other: Neuropsicomotor developmental test |
What is being measured
The primary outcome is the main question the study is designed to answer.
Hydrocephalus treatment - shunt or ETV
Need to treat hydrocephaly in the first year of life
Measured: 12 months corrected age or more
Ambulation
Ambulation Status
Measured: 30 months of cronological age or more
Neurodevelopmental test
PEDI scale will be performed
Measured: 30 months of cronogical age or more
Secondary outcomes (1)
Anatomical verus motor level
To compare anatomical level with motor level
Measured: 30 months of cronological age or more
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Apr 2020
- Study start1 Apr 2017
- Primary completion30 Sep 2019
- Study completion1 Mar 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (8)Papers the sponsor has linked to this study.
- Other trials for Open Spina BifidaStudies of the same condition, open and under way.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hospital Israelita Albert Einstein
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- DENISE A LAPA, MD, Principal investigator, Hospital Israelita Albert Einstein
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04356703
- Sponsor's protocol ID
- HIAE-SAFER
- Status last verified
- Apr 2020
- First submitted
- 20 Apr 2020
- Last updated
- 22 Apr 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Sanz Cortes M, Lapa DA, Acacio GL, Belfort M, Carreras E, Maiz N, Peiro JL, Lim FY, Miller J, Baschat A, Sepulveda G, Davila I, Gielchinsky Y, Benifla M, Stirnemann J, Ville Y, Yamamoto M, Figueroa H, Simpson L, Nicolaides KH. Proceedings of the First Annual Meeting of the International Fetoscopic Myelomeningocele Repair Consortium. Ultrasound Obstet Gynecol. 2019 Jun;53(6):855-863. doi: 10.1002/uog.20308. No abstract available.
PubMed 31169957Lapa DA. Prenatal superior to postnatal myelomeningocele surgery. J Pediatr. 2018 Jul;198:322-325. doi: 10.1016/j.jpeds.2018.04.060. No abstract available.
PubMed 29936963Lapa DA. Endoscopic fetal surgery for neural tube defects. Best Pract Res Clin Obstet Gynaecol. 2019 Jul;58:133-141. doi: 10.1016/j.bpobgyn.2019.05.001. Epub 2019 Jun 29.
PubMed 31350160Carrabba G, Macchini F, Fabietti I, Schisano L, Meccariello G, Campanella R, Bertani G, Locatelli M, Boito S, Porro GA, Gabetta L, Picciolini O, Cinnante C, Triulzi F, Ciralli F, Mosca F, Lapa DA, Leva E, Rampini P, Persico N. Minimally invasive fetal surgery for myelomeningocele: preliminary report from a single center. Neurosurg Focus. 2019 Oct 1;47(4):E12. doi: 10.3171/2019.8.FOCUS19438.
PubMed 31574466Pedreira DA, Zanon N, Nishikuni K, Moreira de Sa RA, Acacio GL, Chmait RH, Kontopoulos EV, Quintero RA. Endoscopic surgery for the antenatal treatment of myelomeningocele: the CECAM trial. Am J Obstet Gynecol. 2016 Jan;214(1):111.e1-111.e11. doi: 10.1016/j.ajog.2015.09.065. Epub 2015 Sep 18.
PubMed 26386383Herrera SR, Leme RJ, Valente PR, Caldini EG, Saldiva PH, Pedreira DA. Comparison between two surgical techniques for prenatal correction of meningomyelocele in sheep. Einstein (Sao Paulo). 2012 Oct-Dec;10(4):455-61. doi: 10.1590/s1679-45082012000400011. English, Portuguese.
PubMed 23386086Pedreira DA, Quintero RA, Acacio GL, Caldini ET, Saldiva PH. Neoskin development in the fetus with the use of a three-layer graft: an animal model for in utero closure of large skin defects. J Matern Fetal Neonatal Med. 2011 Oct;24(10):1243-8. doi: 10.3109/14767058.2011.564486. Epub 2011 Aug 17.
PubMed 21848412Sanchez e Oliveira Rde C, Valente PR, Abou-Jamra RC, Araujo A, Saldiva PH, Pedreira DA. Biosynthetic cellulose induces the formation of a neoduramater following pre-natal correction of meningomyelocele in fetal sheep. Acta Cir Bras. 2007 May-Jun;22(3):174-81. doi: 10.1590/s0102-86502007000300004.
PubMed 17546289
Study at a glance
From the registry record, last updated by the sponsor on 22 Apr 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Impact of Prenatal Correction of Spina Bifida Using Fetoscopy and the SAFER Technique on Long-term Neurodevelopment.
- ClinicalTrials.gov ID
- NCT04356703
- Acronym / sponsor ID
- HIAE-SAFER
- Conditions
- Open Spina Bifida, Myelomeningocele, Meningomyelocele, Chiari Malformation With Spina Bifida, Chiari Malformation Type 2
- Keywords
- myelomeningocele, spina bifida, meningomyelocele, fetal surgery, fetoscopy, fetal disease, fetal treatment
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Hospital Israelita Albert EinsteinUniversity, hospital or other
- Study start
- 1 Apr 2017 (actual)
- Primary completion
- 30 Sep 2019 (actual)
- Study completion
- 1 Mar 2020 (actual)
- First posted
- 22 Apr 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.
Children submitted to fetoscopic in utero myelomeningocele repair using the SAFER (Skin-over-biocellulose for Anternatal FEtoscopic Repair) technique will evaluate at 30 months or more, regarding ambulation, neurodevelopment, urinary and fecal status.
Detailed description
Children submitted to fetoscopic in utero myelomeningocele repair using the SAFER (Skin-over-biocellulose for Anternatal FEtoscopic Repair) technique will evaluate the neuropsicomotor development at 30 months of chronological age. The mental evaluation will use the Bayley III and thePEDI neurodevelopmental scale. The motor development will use the difference between anatomic and motor level of the lesion. Urinary and fecal continence will be evaluated using clinical and ultrasonographic parameters. For the anatomical level determination, immaging of the spine at the lesion level will be used. Motor level and ambulation will be evaluated through standard clinical examination.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Children submitted to fetoscopic surgery | – | Other: Neuropsicomotor developmental testChildren submitted to fetoscopic in utero myelomeningocele repair using the SAFER (Skin-over-biocellulose for Anternatal FEtoscopic Repair) technique will evaluate the neuropsicomotor development at 30 months of chronological age or older |
Who may qualify
- Ages
- 12 months to 6 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Children submitted to prenatal repair of open spina bifida using the fetoscopic percutaneous repair technique - SAFER
Key inclusion criteria (1 listed)
- Children operated in utero using the SAFER technique
Key exclusion criteria (1 listed)
- Death before 30 month
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 |
|---|---|---|
| Neuropsicomotor developmental testChildren will be evaluated trough Bayley III and PEDI scale. They also will be examined by pediatrician, neurosurgeon, orthopedics and phisiatry | Other | Children submitted to fetoscopic surgery |
Primary outcome
Hydrocephalus treatment - shunt or ETV
Time frame: 12 months corrected age or more
Need to treat hydrocephaly in the first year of life
Ambulation
Time frame: 30 months of cronological age or more
Ambulation Status
Neurodevelopmental test
Time frame: 30 months of cronogical age or more
PEDI scale will be performed
1 secondary outcome
Anatomical verus motor level
Time frame: 30 months of cronological age or more
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Apr 2020.
- Background: PubMed 31169957Sanz Cortes M, Lapa DA, Acacio GL, Belfort M, Carreras E, Maiz N, Peiro JL, Lim FY, Miller J, Baschat A, Sepulveda G, Davila I, Gielchinsky Y, Benifla M, Stirnemann J, Ville Y, Yamamoto M, Figueroa H, Simpson L, Nicolaides KH. Proceedings of the First Annual Meeting of the International Fetoscopic Myelomeningocele Repair Consortium. Ultrasound Obstet Gynecol. 2019 Jun;53(6):855-863. doi: 10.1002/uog.20308. No abstract available.
- Background: PubMed 29936963Lapa DA. Prenatal superior to postnatal myelomeningocele surgery. J Pediatr. 2018 Jul;198:322-325. doi: 10.1016/j.jpeds.2018.04.060. No abstract available.
- Background: PubMed 31350160Lapa DA. Endoscopic fetal surgery for neural tube defects. Best Pract Res Clin Obstet Gynaecol. 2019 Jul;58:133-141. doi: 10.1016/j.bpobgyn.2019.05.001. Epub 2019 Jun 29.
- Background: PubMed 31574466Carrabba G, Macchini F, Fabietti I, Schisano L, Meccariello G, Campanella R, Bertani G, Locatelli M, Boito S, Porro GA, Gabetta L, Picciolini O, Cinnante C, Triulzi F, Ciralli F, Mosca F, Lapa DA, Leva E, Rampini P, Persico N. Minimally invasive fetal surgery for myelomeningocele: preliminary report from a single center. Neurosurg Focus. 2019 Oct 1;47(4):E12. doi: 10.3171/2019.8.FOCUS19438.
- Background: PubMed 26386383Pedreira DA, Zanon N, Nishikuni K, Moreira de Sa RA, Acacio GL, Chmait RH, Kontopoulos EV, Quintero RA. Endoscopic surgery for the antenatal treatment of myelomeningocele: the CECAM trial. Am J Obstet Gynecol. 2016 Jan;214(1):111.e1-111.e11. doi: 10.1016/j.ajog.2015.09.065. Epub 2015 Sep 18.
- Background: PubMed 23386086Herrera SR, Leme RJ, Valente PR, Caldini EG, Saldiva PH, Pedreira DA. Comparison between two surgical techniques for prenatal correction of meningomyelocele in sheep. Einstein (Sao Paulo). 2012 Oct-Dec;10(4):455-61. doi: 10.1590/s1679-45082012000400011. English, Portuguese.
- Background: PubMed 21848412Pedreira DA, Quintero RA, Acacio GL, Caldini ET, Saldiva PH. Neoskin development in the fetus with the use of a three-layer graft: an animal model for in utero closure of large skin defects. J Matern Fetal Neonatal Med. 2011 Oct;24(10):1243-8. doi: 10.3109/14767058.2011.564486. Epub 2011 Aug 17.
- Background: PubMed 17546289Sanchez e Oliveira Rde C, Valente PR, Abou-Jamra RC, Araujo A, Saldiva PH, Pedreira DA. Biosynthetic cellulose induces the formation of a neoduramater following pre-natal correction of meningomyelocele in fetal sheep. Acta Cir Bras. 2007 May-Jun;22(3):174-81. doi: 10.1590/s0102-86502007000300004.
- Individual participant dataSharing planned: Undecided
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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. This study is not recruiting at the moment.
DENISE A LAPA, MD principal investigator
Hospital Israelita Albert Einstein
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Open Spina Bifida and Myelomeningocele, run by Hospital Israelita Albert Einstein at 1 site in Brazil.
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 1 inclusion and 1 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: finished, as of the sponsor's last update on 22 Apr 2020. 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 Brazil. 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Hydrocephalus treatment - shunt or ETV (time frame: 12 months corrected age or more). The study lists 3 primary outcomes in all. 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 NCT04356703, which also lists 8 linked publications.
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 22 Apr 2020.