Clinical trial NCT03414697: Transplantation of Umbilical Cord-derived Mesenchymal Stem Cells Via Different Routes
Official title: Transplantation of Umbilical Cord-derived Mesenchymal Stem Cells Via Different Routes for the Treatment of Cerebral Palsy in Children
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: NA
Conditions: Cerebral Palsy
Keywords: umbilical cord-derived mesenchymal stem cells
cerebral palsy
Lead sponsor: The First Affiliated Hospital of Dalian Medical University
Sponsor class: OTHER
First posted: 2018-01-30
Enrollment: 44
Sex: ALL
Minimum age (years): 2
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.

Detailed 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.

Eligibility criteria: 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

Primary outcomes:
measure: The Gross Motor Function Measure (GMFM)-88 item score
timeFrame: 12 months after 2 courses of treatment with UC-MSCs.
Secondary outcomes:
measure: Fine Motor Function Measure scale (FMFM) score
timeFrame: 1, 3, 6, 9 and 12 months after 2 courses of treatment with UC-MSCs.
measure: The Modified Ashworth scale (MAS) score
timeFrame: 1, 3, 6, 9 and 12 months after 2 courses of treatment with UC-MSCs.
measure: Gesell Development Schedule score
timeFrame: 1, 3, 6, 9 and 12 months after 2 courses of treatment with UC-MSCs.
measure: Incidence of adverse reactions
timeFrame: 1, 3, 6, 9 and 12 months after 2 courses of treatment with UC-MSCs.
Other outcomes:
Locations:
city: Dalian
country: China
facility: The First Affiliated Hospital of Dalian Medical University
state: Liaoning
Study officials:
affiliation: The First Affiliated Hospital of Dalian Medical University
name: Jing Liu, Ph.D
role: PRINCIPAL_INVESTIGATOR
References: