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

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

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

Study Overview

• Status: Completed
• Conditions: Stem Cell Infusion
• Intervention / Treatment: Combination product: Autologous bone marrow mononuclear cell transplantation

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 Type: Interventional
• Enrollment (Actual): 11
• Phase: Phase 1

Contacts and Locations

Study Locations

• Vietnam
  • Hanoi, Vietnam, 100000
    • Vinmec Research Institute of Stem Cell and Gene Technology

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 months to 15 years (CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: Autologous BMMNC transplantation; - Autologous bone marrow mononuclear cell transplantation 2 intrathecal administrations of autologous bone marrow mononuclear cells at baseline and 6 months afterward	Combination product: Autologous bone marrow mononuclear cell transplantation; Transplantation of Autologous Bone Marrow Mononuclear cells

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Adverse events and serious adverse events	Incidence of the adverse events or serious adverse events after infusion	up to the 12-month period following treatment

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Bristol stool scale	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.	up to the 12-month period following treatment
Rectoanal inhibitory reflex	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	up to the 12-month period following treatment
Bladder sensation	The cystometry was used to assess bladder sensation	up to the 12-month period following treatment
Urinary retention	urinary retention is assessed via cytometry	up to the 12-month period following treatment
Urinary incontinence	Urinary incontinence is assessed via cytometry	up to the 12-month period following treatment
Lower limb motor functions	Lower limb motor function was assessed via manual muscle testing (MMT)	up to the 12-month period following treatment

Collaborators and Investigators

• Sponsor: Vinmec Research Institute of Stem Cell and Gene Technology
• Collaborators: Vinmec Health Care System (Vingroup Joint Stock Company)

Publications and helpful links

General Publications

• 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.
• 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.

Study record dates

Study Major Dates

• Study Start (ACTUAL): July 1, 2016
• Primary Completion (ACTUAL): December 25, 2020
• Study Completion (ACTUAL): August 31, 2021

Study Registration Dates

• First Submitted: July 9, 2022
• First Submitted That Met QC Criteria: July 20, 2022
• First Posted (ACTUAL): July 25, 2022

Study Record Updates

• Last Update Posted (ACTUAL): July 25, 2022
• Last Update Submitted That Met QC Criteria: July 20, 2022
• Last Verified: July 1, 2022

More Information

Terms related to this study

• Keywords: Spina bifida; mononuclear cell; neurologic sequelea
• Additional Relevant MeSH Terms: Nervous System Diseases; Congenital Abnormalities; Nervous System Malformations; Neural Tube Defects; Spinal Dysraphism
• Other Study ID Numbers: CS. ĐT.19H1

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No