Evaluating Bone Marrow Cell Transplant for Treating Cerebral Palsy From Brain Hypoxia

NCT06629324 | Recruiting Phase 2 DMC

• 1 other identifier: ISC-22.72
• Study Type: interventional
• Target: 58
• Locations: 1 country
• Sites: 2

Brief Summary

This clinical trial aims to evaluate the effectiveness of autologous bone marrow mononuclear cell transfusion in treating cerebral palsy caused by cerebral hypoxia. The key questions the study seeks to answer are:

• What is the safety profile in terms of adverse events (AE) and serious adverse events (SAE) observed over the 9 months following the first transplantation?
• How does autologous bone marrow mononuclear cell (BM MNC) transplantation impact the gross motor function (GMFM-88) scores and Gross Motor Function Classification System (GMFCS) scores in children with cerebral palsy?
• How does autologous BM MNC transplantation influence muscle tone (Modified Ashworth Scale score) and hand motor function (MACS/Mini-MACS scale) in children with cerebral palsy, 9 months post the initial transplantation? Fifty-eight selected patients, aged 1 to 10 years and diagnosed with spastic cerebral palsy due to brain hypoxia, will be randomly divided into two groups:
• Group A: will receive two BM MNC infusions with the first at baseline and the second at 6 months ± 21 days (T6) via the spinal route.
• Group B: will serve as the control group for the first 9 months. During this period, patients will not receive cell transplantation but will undergo a similar rehabilitation and medication regimen as Group A. After 9 months, Group B will receive two BM MNC infusions: the first at 9 months ± 21 days (T9) and the second at 15 months ± 21 days (T15) via the spinal route, with a follow-up at 18 months ± 21 days (T18) compared to baseline.
• Both groups: will undergo rehabilitation for 10 days per month, three times, either at rehabilitation centers or performed by a rehabilitation technician at home. After this period, continued training will be conducted by family members. The combined medication regimen will include muscle relaxants (if muscle spasticity is present), vitamins, and neuroprotective drugs (Piracetam).

Conditions

Autism Spectrum Disorder

Outcome Measures

Primary Outcomes (5)

• Adverse events and Serious adverse events

  The incidence of adverse events or serious adverse events following transplantation includes events leading to death, life-threatening events, events requiring hospitalization or prolonging hospital stay, events resulting in significant or permanent disability, and events causing birth defects in newborns.

  Up to 9 months for Group A and 18 months for Group B during the study.

• Gross Motor Function Measure-88 (GMFM-88)

  Evaluate the effectiveness of umbilical cord blood stem cell transplantation in children with cerebral palsy based on changes in gross motor function score after the first cell transplantation.

  Group A: Baseline; 3 months (T3±21 days); 6 months (T6±21 days); 9 months (T9±21 days)/ Group B: Baseline; 3 months (T3±21 days); 6 months (T6±21 days); 9 months (T9±21 days); 12 months (T12±21 days); 15 months (T15±21 days); 18 months (T18±21 days).

• Gross Motor Function Classification System (GMFCS)

  This tool will be used to classify the severity of motor function impairment in children with cerebral palsy.

  Group A: Baseline; 3 months (T3±21 days); 6 months (T6±21 days); 9 months (T9±21 days). Group B: Baseline; 3 months (T3±21 days); 6 months (T6±21 days); 9 months (T9±21 days); 12 months (T12±21 days); 15 months (T15±21 days); 18 months (T18±21 days).

• The Mini-Manual Ability Classification System (Mini-MACS) and Manual Ability Classification System (MACS)

  Hand function after the first cell transplantation: Mini-MACS (Manual Ability Classification System) for children aged 1 to 4 years, and MACS for children aged over 4 to 18 years.

  Group A: Baseline; 3 months (T3±21 days); 6 months (T6±21 days); 9 months (T9±21 days). Group B: Baseline; 3 months (T3±21 days); 6 months (T6±21 days); 9 months (T9±21 days); 12 months (T12±21 days); 15 months (T15±21 days); 18 months (T18±21 days).

• Modified Ashworth Scale

  Change in muscle tone after the first cell transplantation.

  Group A: Baseline; 3 months (T3±21 days); 6 months (T6±21 days); 9 months (T9±21 days). Group B: Baseline; 3 months (T3±21 days); 6 months (T6±21 days); 9 months (T9±21 days); 12 months (T12±21 days); 15 months (T15±21 days); 18 months (T18±21 days).

Study Arms (2)

BM MNC transplantation and Rehabilitation

ACTIVE COMPARATOR

Patients in group A will receive two autologous BM MNC administrations at baseline and 6 months ± 21 days (T6)

Biological: Autologous Bone Marrow Mononuclear Cell Transfusion; Other: Rehabilitation

Placebo with Rehabilitation, then BM MNC transplantation

PLACEBO COMPARATOR

Group B will receive BM MNCs at 9 months ± 21 days (T9) and 15 months ± 21 days (T15), with outcomes evaluated at 18 months ± 21 days (T18) compared to baseline.

Biological: Autologous Bone Marrow Mononuclear Cell Transfusion; Other: Rehabilitation

Interventions

Autologous Bone Marrow Mononuclear Cell Transfusion BIOLOGICAL

Bone marrow was collected under general anesthesia from both anterior superior iliac crests, taking 15-20 minutes, with a maximum volume of 350 mL for older children. Mononuclear cells were isolated using Ficoll density gradient centrifugation and prepared for infusion. The infusion, performed in the L4-L5 spinal space, lasted about 30 minutes at a rate of 20 mL per hour. Cerebrospinal fluid (CSF) was withdrawn before infusion, with the amount based on the child's weight. Patients received Rocephin for infection prevention and pain relief with alternating doses of Ibuprofen and Efferalgan for 2 days post-procedure. Seduxen was given once on the first night after bone marrow collection.

BM MNC transplantation and Rehabilitation; Placebo with Rehabilitation, then BM MNC transplantation

Rehabilitation OTHER

Rehabilitation methods involve various therapies tailored to the patient\'s mobility level. (1) Physical therapy focuses on exercises to control the head, neck, and trunk, manage muscle tone, and reduce spasticity. Patients gradually practice essential movements like rolling, sitting, crawling, standing, and walking, aligned with developmental milestones. (2) Occupational therapy aims to enhance fine motor skills and daily activities. Through hand function exercises, patients improve upper limb functionality, strengthen their ability to grasp and hold objects and refine coordination between both hands and hand-eye coordination. (3) Speech therapy improves communication, cognitive abilities, and chewing and swallowing functions. Specific exercises target lip and mouth movements, helping patients express and understand language better. Additionally, there are activities to strengthen jaw and facial muscles, which are essential for improving chewing and swallowing abilities.

BM MNC transplantation and Rehabilitation; Placebo with Rehabilitation, then BM MNC transplantation

Eligibility Criteria

• Age: 1 Year - 10 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Age from 1 to 10 years, both genders;
• Gross Motor Function Classification System (GMFCS): levels II to V;
• Spastic cerebral palsy due to brain hypoxia.

You may not qualify if:

• Coagulation disorders;
• Severe health conditions such as cachexia, heart failure, lung, liver, or kidney failure; or active infections;
• Spinal injuries prevent the placement of a catheter through the spinal cavity;
• Cancer;
• HIV positive, active viral hepatitis;
• Hemoglobin below 110 g/L.

Sponsors & Collaborators

• Vinmec Research Institute of Stem Cell and Gene Technology: lead

Study Sites (2)

Vinmec research Institute and Gene Technology

Hanoi, 100000, Vietnam

RECRUITING

Vinmec Research Institute of Stem Cell and Gene Technology

Hanoi, 100000, Vietnam

NOT YET RECRUITING

MeSH Terms

Conditions

Autism Spectrum Disorder

Interventions

Rehabilitation

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Aftercare; Continuity of Patient Care; Patient Care; Therapeutics; Health Services; Health Care Facilities Workforce and Services

Study Officials

• Liem T Nguyen, MD., PhD

  Vinmec Research Insitute of Stem Cell and Gene Technology

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Liem T Nguyen, MD, PhD

CONTACT

800-555-5555; v.liemnt@vinmec.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 3, 2024
• First Posted: October 8, 2024
• Study Start: October 15, 2024
• Primary Completion (Estimated): October 15, 2026
• Study Completion (Estimated): January 31, 2027
• Last Updated: January 22, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

VN (2)