Clinical trial NCT03005249: Neural Stem Cells Therapy for Cerebral Palsy
Official title: Neural Stem Cells Therapy for Cerebral Palsy: a Prospective, Randomized, Parallel-controlled Trial
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: NA
Conditions: Cerebral Palsy
Lead sponsor: The First Affiliated Hospital of Dalian Medical University
Sponsor class: OTHER
First posted: 2016-12-29
Enrollment: 20
Sex: ALL
Minimum age (years): 1
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: To evaluate the safety and efficacy of neural stem cells (NSCs) therapy for cerebral palsy.

Detailed description: Cerebral palsy is the most severe lifelong disability disease in childhood. In China, an epidemiological investigation of cerebral palsy in 2004 has reported that 2.74‰ (about 6.50-9.75 million) of children suffered from cerebral palsy. The costs of medical expenses, living allowance and nursing care can go up to RMB 20000-50000 yuan per year for each child with cerebral palsy and thus China will expend RMB 120-300 billion yuan per year for children with cerebral palsy. With full liberalization of two-child policy, there is an increasing number of second babies born to women who are 35 years or older. Thus, the incidence of cerebral palsy tends to increase. There is currently no cure for cerebral palsy, resulting in a heavy burden for families and on society. Recent clinical trials performed in countries outside China have demonstrated that stem cell therapy is likely the most effective method to treat cerebral palsy. Fifteen trials regarding stem cell therapy for cerebral palsy have been registered with ClinicalTrials.gov. In China, there have been no standardized registered trials regarding stem cell therapy for cerebral palsy, which greatly restricts its clinical application. In October 2016, the National Health and Family Planning Commission and the Food and Drug Administration of China jointly announced the premier stem cell clinical research institutions, making performing standardized trials regarding stem cell therapy for cerebral palsy in China possible.

Eligibility criteria: Inclusion Criteria:

* Neonates with ischemic/hypoxic cerebral palsy (mainly including neonate with asphyxia and premature infants);
* Clinical manifestation of spastic tetraplegia;
* With moderate to severe cerebral palsy, The Gross Motor Function; Classification System (GMFCS) levels IV-V;
* Age 1-5 years, of either sex;
* Provision of signed informed consent by one of his/her parents or legal guardians prior to commencement of this study;

Exclusion Criteria:

* Systemic diseases that possibly influence treatment or patient's compliance;
* Potentially life-threatening diseases involving various organ systems;
* Brain deformity;
* Abnormal behaviors or mood disorder;
* Allergies from blood products;
* Suffering from infectious disease;
* Subjected to craniocerebral operations prior to screening.

Primary outcomes:
measure: GMFM-88 score
timeFrame: changes of month 1, month 3, month 6, month 9 and month 12 after therapy
Secondary outcomes:
measure: Fine Motor Function Measure (FMFM) score
timeFrame: changes of month 1, month 3, month 6, month 9 and month 12 after therapy
measure: Modified Ashworth Scale score
timeFrame: changes of month 1, month 3, month 6, month 9 and month 12 after therapy
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: