Autologous bone marrow cell transplant for spastic cerebral palsy from brain hypoxia

Evaluation of the Efficacy of Autologous Bone Marrow-Derived Mononuclear Cell Transplantation in the Treatment of Cerebral Palsy Due to Brain Hypoxia: A Phase II Randomized Clinical Trial

This trial will try giving children aged 1 to 10 with spastic cerebral palsy caused by brain hypoxia an infusion of their own bone marrow mononuclear cells to see if their movement and muscle tightness improve.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 58 (estimated)
• Ages: 1 Year to 10 Years
• Sex: All
• Sponsor: Vinmec Research Institute of Stem Cell and Gene Technology (Academic / other)
• Locations: 2 sites (Hanoi and 1 other locations)
• Trial ID: NCT06629324 on ClinicalTrials.gov

What this trial studies

This Phase II randomized trial will enroll 58 children aged 1–10 with spastic cerebral palsy due to brain hypoxia and GMFCS levels II–V at Vinmec in Hanoi. Participants will be randomly assigned to receive autologous bone marrow mononuclear cell transplantation plus rehabilitation or a control regimen, with treatments delivered per protocol and follow-up visits over nine months after the first transplantation. Safety outcomes include monitoring adverse events and serious adverse events, and efficacy outcomes include changes in GMFM-88, GMFCS, Modified Ashworth Scale, and MACS/Mini-MACS scores. The trial builds on a Phase I safety study and aims to quantify functional gains and tone changes under standardized rehabilitation.

Who should consider this trial

Good fit: Children aged 1 to 10 with spastic cerebral palsy caused by brain hypoxia and GMFCS level II–V who are medically stable and meet laboratory and safety criteria are ideal candidates.

Not a fit: Children with non-hypoxic or non-spastic cerebral palsy, severe organ failure, active infections, coagulation disorders, spinal conditions preventing intrathecal procedures, or who fail hemoglobin and other safety thresholds are unlikely to benefit or be eligible.

Why it matters

Potential benefit: If successful, this approach could reduce muscle spasticity and improve gross motor function and daily mobility for children with hypoxic spastic cerebral palsy, potentially improving quality of life.

How similar studies have performed: Small early-phase trials and the investigators' Phase I study have reported safety and some motor improvements with autologous bone marrow mononuclear cell therapy, but randomized evidence in larger samples is still limited.

Eligibility criteria

Inclusion Criteria:

* 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.

Exclusion Criteria:

* 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.

Where this trial is running

Hanoi and 1 other locations

• Vinmec research Institute and Gene Technology — Hanoi, Vietnam (Recruiting)
• Vinmec Research Institute of Stem Cell and Gene Technology — Hanoi, Vietnam (Not_yet_recruiting)

Study contacts

• Principal investigator: Liem T Nguyen, MD., PhD — Vinmec Research Insitute of Stem Cell and Gene Technology
• Study coordinator: Liem T Nguyen, MD, PhD

Conditions Autism Spectrum Disorder