Phase II: Autologous BM-MSC combining Rehabilitation for children with cerebral palsy

Project Title: Evaluation of the effectiveness of autologous bone marrow mononuclear cell infusion in the treatment of cerebral palsy due to cerebral hypoxia: A randomized Phase II clinical trial

Principal Investigator (PI):

– Prof. Nguyen Thanh Liem

– Prof. Pham Van Minh

Objective:

To evaluate the effectiveness of autologous bone marrow mononuclear cell infusion in the treatment of cerebral palsy due to cerebral hypoxia.

Project Description (main page – 250 words):

This is a randomized Phase II clinical trial designed to evaluate the efficacy and safety of intrathecal autologous bone marrow mononuclear cell infusion in the treatment of cerebral palsy due to cerebral hypoxia in children. The study is conducted at Vinmec International General Hospital, with coordinated rehabilitation therapy at Hanoi Rehabilitation Hospital, and is scheduled from September 2024 to January 2027.

Eligible participants are children aged 1 to 10 years of both sexes, classified as GMFCS levels II to V and diagnosed with spastic cerebral palsy due to cerebral hypoxia. The planned sample size is 58 patients, who are randomly allocated in a 1:1 ratio into two groups. Group A receives two infusions at baseline and at 6 months. Group B serves as the control for the first 9 months, after which patients receive two infusions at months 9 and 15 and are followed until month 18. Both groups receive periodic rehabilitation and supportive medications as indicated, including muscle relaxants for spasticity, vitamins, and neurotrophic agents. Treatment efficacy is assessed using the GMFM 88, GMFCS, Mini MACS and MACS, and the Modified Ashworth Scale.