Stem Cell InfusionPhase 1NCT05472428
Outcomes of Autologous Bone Marrow Mononuclear Cell Administration in the Treatment of Neurologic Sequelea in Children With Spina Bifida
A Phase 1 study testing Autologous bone marrow mononuclear cell transplantation in people with Stem Cell Infusion, run by Vinmec Research Institute of Stem Cell and Gene Technology at 1 site in Vietnam.
Trial 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 Read more about this study
- Who is it for?
- Ages 6 months to 15 years
- Conditions the study is for
- Stem Cell Infusion
- What is being tested?
- Autologous bone marrow mononuclear cell transplantation
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 1 siteVietnam
- Study dates
- 1 Jul 2016to 31 Aug 2021
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 15 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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).
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
Vietnam1 site
Vinmec Research Institute of Stem Cell and Gene Technology
Hanoi, 100000
About this study
Official title: 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
Read the 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
- Conditions
- Stem Cell Infusion
- Being tested
- Autologous bone marrow mononuclear cell transplantation
- Keywords
- Spina bifida, mononuclear cell, neurologic sequelea
- Medical subject headings
- Spinal Dysraphism
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous bone marrow mononuclear cell transplantationCombination product
Transplantation of Autologous Bone Marrow Mononuclear cells
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Combination Product: Autologous bone marrow mononuclear cell transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse events and serious adverse events
Incidence of the adverse events or serious adverse events after infusion
Measured: up to the 12-month period following treatment
Secondary outcomes (6)
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.
Measured: 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
Measured: up to the 12-month period following treatment
Bladder sensation
The cystometry was used to assess bladder sensation
Measured: up to the 12-month period following treatment
Urinary retention
urinary retention is assessed via cytometry
Measured: up to the 12-month period following treatment
Urinary incontinence
Urinary incontinence is assessed via cytometry
Measured: up to the 12-month period following treatment
Lower limb motor functions
Lower limb motor function was assessed via manual muscle testing (MMT)
Measured: up to the 12-month period following treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jul 2022
- Study start1 Jul 2016
- Primary completion25 Dec 2020
- Study completion31 Aug 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Stem Cell InfusionStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vinmec Research Institute of Stem Cell and Gene Technology
- Sponsor type
- University, hospital or other
- Collaborators
- Vinmec Health Care System (Vingroup Joint Stock Company)
- Responsible party
- Sponsor
- Investigators
- Liem T Nguyen, MD., PhD, Principal investigator, Vinmec Research Institute of Stem Cell and Gene Technology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05472428
- Sponsor's protocol ID
- CS. ĐT.19H1
- Status last verified
- Jul 2022
- First submitted
- 9 Jul 2022
- Last updated
- 25 Jul 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
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.
PubMed 30143601Aghayan 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.
PubMed 25678991Than 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.
PubMed 38114596Nguyen 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.
PubMed 37118832
Study at a glance
From the registry record, last updated by the sponsor on 25 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Autologous Bone Marrow Mononuclear Cell Administration in the Treatment of Neurologic Sequela in Children With Spina Bifida
- ClinicalTrials.gov ID
- NCT05472428
- Acronym / sponsor ID
- CS. ĐT.19H1
- Conditions
- Stem Cell Infusion
- Keywords
- Spina bifida, mononuclear cell, neurologic sequelea
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- Vinmec Research Institute of Stem Cell and Gene TechnologyUniversity, hospital or other
- Collaborators
- Vinmec Health Care System (Vingroup Joint Stock Company)
- Study start
- 1 Jul 2016 (actual)
- Primary completion
- 25 Dec 2020 (actual)
- Study completion
- 31 Aug 2021 (actual)
- First posted
- 25 Jul 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Autologous BMMNC transplantation | Experimental | Combination Product: 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 |
Who may qualify
- Ages
- 6 months to 15 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- 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).
Key exclusion criteria (6 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Autologous bone marrow mononuclear cell transplantationTransplantation of Autologous Bone Marrow Mononuclear cells | Combination product | Autologous BMMNC transplantation |
Primary outcome
Adverse events and serious adverse events
Time frame: up to the 12-month period following treatment
Incidence of the adverse events or serious adverse events after infusion
6 secondary outcomes
Bristol stool scale
Time frame: up to the 12-month period following treatment
Rectoanal inhibitory reflex
Time frame: up to the 12-month period following treatment
Bladder sensation
Time frame: up to the 12-month period following treatment
Urinary retention
Time frame: up to the 12-month period following treatment
Urinary incontinence
Time frame: up to the 12-month period following treatment
Lower limb motor functions
Time frame: up to the 12-month period following treatment
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jul 2022.
- Results publication: PubMed 30143601Liem 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 publication: PubMed 25678991Aghayan 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.
- Derived from this study: PubMed 38114596Than 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.
- Derived from this study: PubMed 37118832Nguyen 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.
- Individual participant dataSharing planned: No
Related trials for Stem Cell Infusion
RecruitingPhase 2
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
Not yet recruitingPhase 1
Expanded Donor Stem Cells for Poor Graft Function After Stem Cell Transplantation in Children
Poor Graft Function, Hematopoietic Stem Cell Transplantation Complications
RecruitingPhase 1
Prophylactic DLI-X for Leukemia Relapse After HCT
Acute Lymphoid Leukemia, Acute Myeloid Leukemia
RecruitingPhase 1
Safety of Cultured Allogeneic Adult Umbilical Cord or Placental Derived Mesenchymal Stem Cell Intravenous Infusion and Intra-Articular Injection for RA
Rheumatoid Arthritis
RecruitingPhase 1
Safety of Cultured Allogeneic Adult Umbilical Cord or Placental Derived Mesenchymal Stem Cell Intravenous Infusion for CP
Cerebral Palsy
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Liem T Nguyen, MD., PhD principal investigator
Vinmec Research Institute of Stem Cell and Gene Technology
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Autologous bone marrow mononuclear cell transplantation in people with Stem Cell Infusion, run by Vinmec Research Institute of Stem Cell and Gene Technology at 1 site in Vietnam.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 25 Jul 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Vietnam. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse events and serious adverse events (time frame: up to the 12-month period following treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05472428, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Jul 2022.