Clinical trial NCT05472428: Outcomes of Autologous Bone Marrow Mononuclear Cell Administration in the Treatment of Neurologic Sequelea in Children With Spina Bifida
Official title: Autologous Bone Marrow Mononuclear Cell Administration in the Treatment of Neurologic Sequela in Children With Spina Bifida
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Stem Cell Infusion
Keywords: Spina bifida
mononuclear cell
neurologic sequelea
Lead sponsor: Vinmec Research Institute of Stem Cell and Gene Technology
Sponsor class: OTHER
Collaborators: name: Vinmec Health Care System (Vingroup Joint Stock Company)
First posted: 2022-07-25
Enrollment: 11
Sex: ALL
Minimum age (years): 6
Maximum age (years): 15
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The aim of this study was to evaluate the safety and efficacy of autologous bone marrow mononuclear cell infusion in the management of neurological sequelae in children with spina bifida

Detailed description: The aim of this study was to evaluate the safety and effectiveness of autologous bone marrow mononuclear cells in 11 patients with spina bifida at Vinmec Research Institute of Stem Cell and Gene Technology in Hanoi, Vietnam from 2016 to 2020

Eligibility criteria: Inclusion Criteria:

* The patient who was diagnosed with lumbar spina bifida underwent spinal cord close-up surgery.
* Both genders.
* Aged between 6 months and 15 years old.
* Exhibited bowel disorders (constipation, fecal incontinence) and urinary dysfunction (urinary retention or leakage).

Exclusion Criteria:

* Vertebrae clefts in the chest, neck, and other spinal locations.
* Coagulopathy.
* Acute and chronic infection.
* Kidney function disorder, liver failure
* Patients with complex cardiovascular diseases (including valvular heart disease, cardiomyopathy, arrhythmia, congenital heart disease, hypertrophy syndrome).
* Distress

Primary outcomes:
measure: Adverse events and serious adverse events
timeFrame: up to the 12-month period following treatment
Secondary outcomes:
measure: Bristol stool scale
timeFrame: up to the 12-month period following treatment
measure: Rectoanal inhibitory reflex
timeFrame: up to the 12-month period following treatment
measure: Bladder sensation
timeFrame: up to the 12-month period following treatment
measure: Urinary retention
timeFrame: up to the 12-month period following treatment
measure: Urinary incontinence
timeFrame: up to the 12-month period following treatment
measure: Lower limb motor functions
timeFrame: up to the 12-month period following treatment
Other outcomes:
Locations:
city: Hanoi
country: Vietnam
facility: Vinmec Research Institute of Stem Cell and Gene Technology
Study officials:
affiliation: Vinmec Research Institute of Stem Cell and Gene Technology
name: Liem T Nguyen, MD., PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Liem NT, Chinh VD, Thinh NT, Minh ND, Duc HM. Improved Bowel Function in Patients with Spina Bifida After Bone Marrow-Derived Mononuclear Cell Transplantation: A Report of 2 Cases. Am J Case Rep. 2018 Aug 25;19:1010-1018. doi: 10.12659/AJCR.909801.
citation: Aghayan HR, Arjmand B, Yaghoubi M, Moradi-Lakeh M, Kashani H, Shokraneh F. Clinical outcome of autologous mononuclear cells transplantation for spinal cord injury: a systematic review and meta-analysis. Med J Islam Repub Iran. 2014 Oct 14;28:112. eCollection 2014.