Autologous Bone Marrow Mononuclear Cell Transplantation Combined With Rehabilitation in Children With Sequelae of Hypoxic-Ischemic Encephalopathy or Intracranial Hemorrhage (BMMCT-CP)

A Randomized Controlled Trial Evaluating the Efficacy and Safety of Autologous Bone Marrow-Derived Mononuclear Cell Transplantation Combined With Rehabilitation in Children With Sequelae of Hypoxic-Ischemic Encephalopathy or Intracranial Hemorrhage

This study evaluates the safety and preliminary efficacy of autologous bone marrow mononuclear cell transplantation combined with rehabilitation in children with neurological sequelae caused by hypoxic-ischemic brain injury or intracranial hemorrhage. Participants in the intervention group will receive autologous bone marrow aspiration, mononuclear cell transplantation via intrathecal route, and rehabilitation, while the control group will receive rehabilitation alone. Outcomes will be compared between groups over follow-up time points.

Study Overview

• Status: Recruiting
• Conditions: Cerebral Palsy Hypoxic-Ischemic Encephalopathy Intracranial Hemorrhage
• Intervention / Treatment: Procedure: Autologous Bone Marrow Mononuclear Cell Transplantation

Detailed Description

Neurological sequelae following hypoxic-ischemic brain injury and intracranial hemorrhage in children remain a major clinical challenge with limited effective treatment options. Rehabilitation is the current standard of care but often results in incomplete recovery. Autologous bone marrow mononuclear cell transplantation has emerged as a promising therapeutic approach due to its potential neuroregenerative and neuroprotective effects. This phase II study aims to evaluate the safety and preliminary efficacy of combining autologous bone marrow mononuclear cell transplantation with rehabilitation compared to rehabilitation alone. The study will also contribute to the development of a standardized technical protocol for this intervention.

• Study Type: Interventional
• Enrollment (Estimated): 60
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: DAT HUU TRAN; Phone Number: +84987836686; Email: drtrandat1986@gmail.com

Study Locations

• Vietnam
  • Hanoi
    • Hanoi, Hanoi, Vietnam, 111111
      • Recruiting
      • Vietnam National Children's Hospital
      • Contact: DAT HUU DAT; Phone Number: +84-987836686; Email: drtrandat1986@gmail.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Children diagnosed with cerebral palsy
• Age from 12 months to 72 months
• Cerebral palsy caused by hypoxic-ischemic brain injury or intracranial hemorrhage
• Gross Motor Function Classification System (GMFCS) level III to V
• Completed required laboratory tests according to the study protocol
• Written informed consent obtained from the child's parent or legal guardian

Exclusion Criteria:

• Children with acute infection at the time of screening or enrollment
• Children with severe coagulopathy or contraindications to bone marrow aspiration
• Children with severe systemic diseases (e.g., severe cardiac, hepatic, or renal failure)
• Children with contraindications to anesthesia or intrathecal transplantation procedures
• Children with a history of malignancy
• Children who are unable to participate in or complete the rehabilitation program
• Parents or legal guardians who do not provide informed consent

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: BMMNC Transplantation Plus Rehabilitation; Participants receive autologous bone marrow mononuclear cell transplantation via intrathecal administration combined with rehabilitation.	Procedure: Autologous Bone Marrow Mononuclear Cell Transplantation; Autologous bone marrow mononuclear cells (BMMNCs) are collected from the patient's iliac crest under sterile conditions. Bone marrow aspiration is performed under appropriate anesthesia, followed by processing to isolate mononuclear cells using density gradient centrifugation. The prepared BMMNCs are administered via intrathecal injection under aseptic conditions. The dosage of cells is determined based on body weight and viability criteria. Patients are monitored for immediate and delayed adverse events following transplantation. In addition to cell transplantation, participants receive standardized rehabilitation therapy, including physical therapy, occupational therapy, and neurodevelopmental interventions, according to institutional protocols. This combined intervention aims to enhance neurological recovery through both regenerative cellular therapy and functional rehabilitation.; Other Names: Rehabilitation
No Intervention: Rehabilitation Alone; Participants receive rehabilitation without cell transplantation.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Gross Motor Function Measure (GMFM-88) total score from baseline to 12 months	Gross motor function will be assessed using the Gross Motor Function Measure (GMFM-88), a validated and standardized clinical scale for evaluating motor function in children with neurological impairment. The primary outcome is the change in GMFM-88 total score from baseline to 12 months. The change in score will be calculated and compared between the intervention group (autologous bone marrow mononuclear cell transplantation plus rehabilitation) and the control group (rehabilitation alone).	Baseline and 12 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in GMFM score at 3, 6, 9 months	Gross motor function will be assessed using the Gross Motor Function Measure (GMFM-88), a standardized clinical tool for children with cerebral palsy and related neurological impairment. The change in GMFM-88 total score from baseline to 3, 6,9 months will be calculated and compared between the intervention and control groups.	Baseline and 3, 6, 9 months
Change in Gross Motor Function Classification System (GMFCS) level from baseline to 12 months	Gross motor function classification will be assessed using the Gross Motor Function Classification System (GMFCS), which categorizes motor function based on self-initiated movement abilities, particularly sitting and mobility. Changes in GMFCS level from baseline to 12 months will be evaluated and compared between the intervention and control groups.	Baseline and 12 months
Change in Manual Ability Classification System (MACS or Mini-MACS) level from baseline to 12 months	Manual ability will be assessed using the Manual Ability Classification System (MACS) or Mini-MACS depending on the child's age. Changes in manual ability level from baseline to 12 months will be evaluated and compared between the intervention and control groups.	Baseline and 12 months
Incidence of transplantation-related adverse events	Adverse events related to bone marrow aspiration, anesthesia, intrathecal transplantation, and follow-up will be recorded. These include local pain at the aspiration site, low back pain after transplantation, fever, irritability, vomiting, rash, and other procedure-related complications. The incidence and severity of adverse events will be monitored and summarized throughout the study.	From intervention through 12 months follow-up

Collaborators and Investigators

• Sponsor: National Children's Hospital, Vietnam

Publications and helpful links

Helpful Links

• Related Info

Study record dates

Study Major Dates

• Study Start (Actual): December 15, 2025
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): April 1, 2028

Study Registration Dates

• First Submitted: April 8, 2026
• First Submitted That Met QC Criteria: April 8, 2026
• First Posted (Actual): April 14, 2026

Study Record Updates

• Last Update Posted (Actual): April 14, 2026
• Last Update Submitted That Met QC Criteria: April 8, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: Bone Marrow Mononuclear Cells Autologous Transplantation Intrathecal Injection Rehabilitation Pediatric Brain Injury
• Additional Relevant MeSH Terms: Therapeutics; Patient Care; Health Services; Health Care Facilities Workforce and Services; Aftercare; Continuity of Patient Care; Rehabilitation
• Other Study ID Numbers: 16/HĐ-K2ĐT (Other Grant/Funding Number: ministry of health)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Individual participant data (IPD) that underlie the results reported in this study will be shared after de-identification. Data will be available beginning 6 months following publication and ending 3 years after publication. Data will be shared with researchers who provide a methodologically sound proposal, for the purpose of achieving aims in the approved proposal. Proposals should be directed to the corresponding investigator. Data access will be provided after approval by the study team and institutional review board.
• IPD Sharing Time Frame: Data will be available beginning 6 months following publication of the primary results and ending 3 years after publication.
• IPD Sharing Access Criteria: Access to de-identified individual participant data and supporting documents will be provided to qualified researchers who submit a methodologically sound proposal. Proposals should be directed to the corresponding investigator. Data access will be granted following review and approval by the study team and the institutional review board. A data use agreement may be required.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF; ANALYTIC_CODE

Drug and device information, study documents

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