Transplantation of Umbilical Cord-derived Mesenchymal Stem Cells Via Different Routes

The First Affiliated Hospital of Dalian Medical University

Status: Unknown

Conditions: Cerebral Palsy

Treatments: Other: Control group; Procedure: UC-MSCs

Study type: Interventional

Funder types: Other

Identifiers

NCT03414697

FirstHospitalDalianMU004

Details and patient eligibility

About

To preliminarily evaluate the efficacy and safety of umbilical cord-derived mesenchymal stem cells (UC-MSCs), and compare the efficacy of UC-MSCs administered through the intravenous, intrathecal, and intranasal routes, in the treatment of cerebral palsy in children.

Full description

Cerebral palsy refers to a neurological disorder caused by a non-progressive brain injury or malformation that occurs in early childhood. It can result in central motor deficits, developmental retardation, abnormal posture, abnormal muscular strength, abnormal muscle tone and/or dysreflexia. It has a high disability rate. There is currently no effective treatment for cerebral palsy. Conventional treatments for cerebral palsy are often tiresome and expensive and have a slow onset of action. Stem cells have been recently used in the treatment of cerebral palsy. This provides a novel method for the treatment of cerebral palsy. According to the existing clinical studies, neural stem cells, bone marrow-derived mesenchymal stem cells, and umbilical cord-derived mesenchymal stem cells (UC-MSCs) are mainly used as the seed cells for the treatment of cerebral palsy. UC-MSCs are the most promising seed cells for the treatment of cerebral palsy because of the advantages including rich sources, ease of harvesting, short doubling time, low immunogenicity, long-time survival post-transplantation, and no ethical issues. UC-MSCs have been widely used to treat Parkinson's disease, rheumatoid arthritis, traumatic brain injury, aplastic anemia, and decompensated liver disease. However,only two studies, and performed only in China, are reported on the treatment of cerebral palsy with UC-MSCs. One from the Hospital 463 of PLA reported 51 patients with cerebral palsy whose symptoms had not been obviously improved after 1 year of rehabilitative treatment. These patients received intrathecal injection of UC-MSCs for three times, once a week, followed by one injection of UC-MSCs via the peripheral vein in the fourth week. Four UC-MSCs injections, once per week, were considered as one course of treatment. Activities of daily living scale score was compared between before and after treatment to evaluate efficacy. Another study is a case report from China. In this report, a combined intravenous and intrathecal injection of UC-MSCs was used to treat cerebral palsy in a 5-year-old child. 28-month follow-up results revealed that the child's gross motor function, immune function, muscle strength, and language ability improved and adverse reactions were not obvious.

Enrollment: 44 estimated patients

Sex: All

Ages: 2 to 18 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Patients diagnosed with hypoxic/ischemic cerebral palsy, primarily including birth asphyxia and premature children
• Patients with spastic quadriplegia
• Patients with moderate to severe cerebral palsy, GMFM scores of 2-3
• Age at 2-18 years
• Provision of signed informed consent by legal representatives of the child prior to start of the study

Exclusion criteria

• Those with systemic diseases that likely interfere with the treatment or child's compliance
• Those complicated by life-threatening diseases of any organ
• Those with brain deformity
• Those with uncontrolled epilepsy
• Those with abnormal behavior or mood disorders
• Those with allergies especially those who are allergic to blood products
• Those are infected with infectious diseases
• Those who had underwent a craniocerebral surgery in other clinical trials

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Single Blind

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44 participants in 4 patient groups

Control group

Other group

Description:

Routine rehabilitation treatments

Treatment:

Other: Control group

Intravenous UC-MSCs group

Experimental group

Description:

Injection of UC-MSCs via the peripheral vein.

Treatment:

Procedure: UC-MSCs

Intrathecal UC-MSCs group

Experimental group

Description:

Injection of UC-MSCs via the intrathecal route.

Treatment:

Procedure: UC-MSCs

Intranasal UC-MSCs group

Experimental group

Description:

Injection of UC-MSCs via the nasal route.

Treatment:

Procedure: UC-MSCs