NCT05472428

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

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
11

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Jul 2016

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

July 1, 2016

Completed
4.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 25, 2020

Completed
8 months until next milestone

Study Completion

Last participant's last visit for all outcomes

August 31, 2021

Completed
10 months until next milestone

First Submitted

Initial submission to the registry

July 9, 2022

Completed
16 days until next milestone

First Posted

Study publicly available on registry

July 25, 2022

Completed
Last Updated

July 25, 2022

Status Verified

July 1, 2022

Enrollment Period

4.5 years

First QC Date

July 9, 2022

Last Update Submit

July 20, 2022

Conditions

Stem Cell Infusion

Keywords

Spina bifidamononuclear cellneurologic sequelea

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (6)

  • Bristol stool scale

    up to the 12-month period following treatment

  • Rectoanal inhibitory reflex

    up to the 12-month period following treatment

  • Bladder sensation

    up to the 12-month period following treatment

  • Urinary retention

    up to the 12-month period following treatment

  • Urinary incontinence

    up to the 12-month period following treatment

  • +1 more secondary outcomes

Study Arms (1)

Autologous BMMNC transplantation

EXPERIMENTAL

\- 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

Interventions

Autologous bone marrow mononuclear cell transplantationCOMBINATION_PRODUCT

Transplantation of Autologous Bone Marrow Mononuclear cells

Autologous BMMNC transplantation

Eligibility Criteria

Age6 Months - 15 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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).

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Vinmec Research Institute of Stem Cell and Gene Technology

Hanoi, 100000, Vietnam

Location

Related Publications (4)

  • 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.

    PMID: 30143601RESULT

  • 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.

    PMID: 25678991RESULT

  • Than UTT, Nguyen LT, Nguyen PH, Nguyen XH, Trinh DP, Hoang DH, Nguyen PAT, Dang VD. Inflammatory mediators drive neuroinflammation in autism spectrum disorder and cerebral palsy. Sci Rep. 2023 Dec 18;13(1):22587. doi: 10.1038/s41598-023-49902-8.

    PMID: 38114596DERIVED

  • Nguyen LT, Le HT, Nguyen KT, Bui HT, Nguyen APT, Ngo DV, Hoang DM, Ngo MD. Outcomes of autologous bone marrow mononuclear cell administration in the treatment of neurologic sequelae in children with spina bifida. Stem Cell Res Ther. 2023 Apr 28;14(1):115. doi: 10.1186/s13287-023-03349-w.

    PMID: 37118832DERIVED

MeSH Terms

Conditions

Spinal Dysraphism

Condition Hierarchy (Ancestors)

Neural Tube DefectsNervous System MalformationsNervous System DiseasesCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Liem T Nguyen, MD., PhD

    Vinmec Research Institute of Stem Cell and Gene Technology

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Masking Details
Open Label
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 9, 2022

First Posted

July 25, 2022

Study Start

July 1, 2016

Primary Completion

December 25, 2020

Study Completion

August 31, 2021

Last Updated

July 25, 2022

Record last verified: 2022-07

Data Sharing

IPD Sharing
Will not share

Locations

VN(1)