Outcomes of Autologous Bone Marrow Mononuclear Cell Administration in the Treatment of Neurologic Sequelea in Children With Spina Bifida

Phase 1

Completed

• Conditions: Stem Cell Infusion
• Interventions: Combination Product: Autologous bone marrow mononuclear cell transplantation

• Registration Number: NCT05472428
• Lead Sponsor: Vinmec Research Institute of Stem Cell and Gene Technology

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

Study Timeline

• Study Start: 2016-07-01
• Primary Completion: 2020-12-25
• Study Completion: 2021-08-31
• Status Verified: 2022-07
• Study First Submitted: 2022-07-09
• Study First Submitted QC: 2022-07-20
• Last Update Submitted: 2022-07-20
• Study First Posted: 2022-07-25
• Last Update Posted: 2022-07-25

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 11

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Autologous BMMNC transplantation	Autologous bone marrow mononuclear cell transplantation	- Autologous bone marrow mononuclear cell transplantation 2 intrathecal administrations of autologous bone marrow mononuclear cells at baseline and 6 months afterward

Primary Outcome Measures

Name	Time	Method
Adverse events and serious adverse events	up to the 12-month period following treatment	Incidence of the adverse events or serious adverse events after infusion

Secondary Outcome Measures

Name	Time	Method
Lower limb motor functions	up to the 12-month period following treatment	Lower limb motor function was assessed via manual muscle testing (MMT)
Rectoanal inhibitory reflex	up to the 12-month period following treatment	The rectoanal inhibitory reflex (RAIR) is a reflex characterized by transient involuntary relaxation of the internal anal sphincter in response to distention of the rectum with the normal value \<= 14.7 ml
Bladder sensation	up to the 12-month period following treatment	The cystometry was used to assess bladder sensation
Urinary retention	up to the 12-month period following treatment	urinary retention is assessed via cytometry
Bristol stool scale	up to the 12-month period following treatment	The Bristol stool scale comes in 7 types: Type 1-2 indicate constipation; type 3-4 are ideal stools as they are easier to pass; type 5-7 may show diarrhea and urgency.
Urinary incontinence	up to the 12-month period following treatment	Urinary incontinence is assessed via cytometry

Trial Locations

Locations (1)

Vinmec Research Institute of Stem Cell and Gene Technology; 🇻🇳 Hanoi, Vietnam