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

• Sponsor: Vinmec Research Institute of Stem Cell and Gene Technology (Other)
• Overall Status: Completed
• CT.gov ID: NCT05472428
• Collaborator: Vinmec Health Care System (Vingroup Joint Stock Company) (Other)
• Enrollment: 11
• Location: 1
• Arm: 1
• Actual Duration (Months): 62
• Patients Per Site Per Month: 0.2

Study Details

Study Description

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

Condition or Disease	Intervention/Treatment	Phase
Stem Cell Infusion	Combination Product: Autologous bone marrow mononuclear cell transplantation	Phase 1

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 Design

• Study Type: Interventional
• Actual Enrollment: 11 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Masking Description: Open Label
• Primary Purpose: Treatment
• Official Title: Autologous Bone Marrow Mononuclear Cell Administration in the Treatment of Neurologic Sequela in Children With Spina Bifida
• Actual Study Start Date: Jul 1, 2016
• Actual Primary Completion Date: Dec 25, 2020
• Actual Study Completion Date: Aug 31, 2021

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures

1. 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

1. 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.

2. 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

3. Bladder sensation [up to the 12-month period following treatment]

   The cystometry was used to assess bladder sensation

4. Urinary retention [up to the 12-month period following treatment]

   urinary retention is assessed via cytometry

5. Urinary incontinence [up to the 12-month period following treatment]

   Urinary incontinence is assessed via cytometry

6. Lower limb motor functions [up to the 12-month period following treatment]

   Lower limb motor function was assessed via manual muscle testing (MMT)

Eligibility Criteria

Criteria

• Ages Eligible for Study: 6 Months to 15 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Vinmec Research Institute of Stem Cell and Gene Technology	Hanoi		Vietnam	100000

Sponsors and Collaborators

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

Investigators

• Principal Investigator: Liem T Nguyen, MD., PhD, Vinmec Research Institute of Stem Cell and Gene Technology

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Vinmec Research Institute of Stem Cell and Gene Technology
• ClinicalTrials.gov Identifier: NCT05472428
• Other Study ID Numbers: CS. ĐT.19H1
• First Posted: Jul 25, 2022
• Last Update Posted: Jul 25, 2022
• Last Verified: Jul 1, 2022

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Vinmec Research Institute of Stem Cell and Gene Technology: Spina bifida; mononuclear cell; neurologic sequelea
• Additional relevant MeSH terms: Spinal Dysraphism

Study Results

No Results Posted as of Jul 25, 2022