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

Primary Purpose

Stem Cell Infusion

Status
Completed
Phase
Phase 1
Locations
Vietnam
Study Type
Interventional
Intervention
Autologous bone marrow mononuclear cell transplantation
Sponsored by
Vinmec Research Institute of Stem Cell and Gene Technology
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Stem Cell Infusion focused on measuring Spina bifida, mononuclear cell, neurologic sequelea

Eligibility Criteria

6 Months - 15 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Vinmec Research Institute of Stem Cell and Gene Technology

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Autologous BMMNC transplantation

Arm Description

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

Outcomes

Primary Outcome Measures

Adverse events and serious adverse events
Incidence of the adverse events or serious adverse events after infusion

Secondary Outcome Measures

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.
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
Bladder sensation
The cystometry was used to assess bladder sensation
Urinary retention
urinary retention is assessed via cytometry
Urinary incontinence
Urinary incontinence is assessed via cytometry
Lower limb motor functions
Lower limb motor function was assessed via manual muscle testing (MMT)

Full Information

First Posted
July 9, 2022
Last Updated
July 20, 2022
Sponsor
Vinmec Research Institute of Stem Cell and Gene Technology
Collaborators
Vinmec Health Care System (Vingroup Joint Stock Company)
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1. Study Identification

Unique Protocol Identification Number
NCT05472428
Brief Title
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

2. Study Status

Record Verification Date
July 2022
Overall Recruitment Status
Completed
Study Start Date
July 1, 2016 (Actual)
Primary Completion Date
December 25, 2020 (Actual)
Study Completion Date
August 31, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vinmec Research Institute of Stem Cell and Gene Technology
Collaborators
Vinmec Health Care System (Vingroup Joint Stock Company)

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. 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
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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Stem Cell Infusion
Keywords
Spina bifida, mononuclear cell, neurologic sequelea

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Masking Description
Open Label
Allocation
N/A
Enrollment
11 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Autologous BMMNC transplantation
Arm Type
Experimental
Arm Description
- Autologous bone marrow mononuclear cell transplantation 2 intrathecal administrations of autologous bone marrow mononuclear cells at baseline and 6 months afterward
Intervention Type
Combination Product
Intervention Name(s)
Autologous bone marrow mononuclear cell transplantation
Intervention Description
Transplantation of Autologous Bone Marrow Mononuclear cells
Primary Outcome Measure Information:
Title
Adverse events and serious adverse events
Description
Incidence of the adverse events or serious adverse events after infusion
Time Frame
up to the 12-month period following treatment
Secondary Outcome Measure Information:
Title
Bristol stool scale
Description
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.
Time Frame
up to the 12-month period following treatment
Title
Rectoanal inhibitory reflex
Description
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
Time Frame
up to the 12-month period following treatment
Title
Bladder sensation
Description
The cystometry was used to assess bladder sensation
Time Frame
up to the 12-month period following treatment
Title
Urinary retention
Description
urinary retention is assessed via cytometry
Time Frame
up to the 12-month period following treatment
Title
Urinary incontinence
Description
Urinary incontinence is assessed via cytometry
Time Frame
up to the 12-month period following treatment
Title
Lower limb motor functions
Description
Lower limb motor function was assessed via manual muscle testing (MMT)
Time Frame
up to the 12-month period following treatment

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Months
Maximum Age & Unit of Time
15 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Liem T Nguyen, MD., PhD
Organizational Affiliation
Vinmec Research Institute of Stem Cell and Gene Technology
Official's Role
Principal Investigator
Facility Information:
Facility Name
Vinmec Research Institute of Stem Cell and Gene Technology
City
Hanoi
ZIP/Postal Code
100000
Country
Vietnam

12. IPD Sharing Statement

Plan to Share IPD
No
Citations:
PubMed Identifier
30143601
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.
Results Reference
result
PubMed Identifier
25678991
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.
Results Reference
result

Learn more about this trial

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

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