Safety and Therapeutic Effect of Autologous Bone Marrow Mesenchymal Stem Cell Transplantation in Children with Cerebral Palsy: Clinical Trial Phase I-II
Evaluation safety and efficacy of bone marrow mesenchymal stem cells in children with cerebral palsy
Design
this study is a clinical trial with a control group, with parallel groups, non-randomized, phase I-II, on 46 patients.
Settings and conduct
46 patients with confirmed cerebral palsy in this study will be divided into control and cell therapy groups (23 patients in each groups). Patients will be screened 1, 3, 6,12 months after cell injection. The study site will be Shohada Tajrish Hospital.
Participants/Inclusion and exclusion criteria
Inclusion criteria: definitive diagnosis of cerebral palsy; no seizures or with controlled seizures; parental consent. Exclusion criteria: severe cognitive disease; hydrocephalus; coagulation diseases; diseases such as cancer, active infections and heart, kidney and liver diseases; evidence of a progressive neurological disease; evidence of a genetic disease.
Intervention groups
Intervention group: In this group, stem cells will be isolated by bone marrow aspiration. After culturing and preparing the cells, MSC cells will be transplanted at a concentration of 1 million cells per ml prepared through a puncture lumbar in the L4 / L5 region. Patients will undergo periodic examinations in neurology clinic at regular intervals after transplantation. patients' sensory and motor function, spasticity and balance will be assessed. Control group: In this group, patients do not receive cell injection and receive supportive therapies.
Main outcome variables
Motor System Functioning; Balance Assessment; Spasticity Assessment; Motor and Cognitive Disability Assessment; Side Effects of cell injection.
General information
Reason for update
Acronym
IRCT registration information
IRCT registration number:IRCT20200502047277N5
Registration date:2021-01-15, 1399/10/26
Registration timing:registered_while_recruiting
Last update:2021-01-15, 1399/10/26
Update count:0
Registration date
2021-01-15, 1399/10/26
Registrant information
Name
saeed oraee yazdani
Name of organization / entity
Country
Iran (Islamic Republic of)
Phone
+98 21 25719
Email address
saeed_o_yazdani@sbmu.ac.ir
Recruitment status
Recruitment complete
Funding source
Expected recruitment start date
2018-10-22, 1397/07/30
Expected recruitment end date
2021-10-22, 1400/07/30
Actual recruitment start date
empty
Actual recruitment end date
empty
Trial completion date
empty
Scientific title
Safety and Therapeutic Effect of Autologous Bone Marrow Mesenchymal Stem Cell Transplantation in Children with Cerebral Palsy: Clinical Trial Phase I-II
Public title
Investigation of the Effect of Stem Cells Injection in Children with Cerebral Palsy
Purpose
Treatment
Inclusion/Exclusion criteria
Inclusion criteria:
Definitive diagnosis of cerebral palsy
No seizures or with controlled seizures
Parental consent
Exclusion criteria:
Severe cognitive disease
Hydrocephalus
Coagulation diseases
Have diseases such as cancer, active infections and heart, kidney and liver diseases
Evidence of a progressive neurological disease
Evidence of a genetic disease
Age
From 1 year old to 18 years old
Gender
Both
Phase
1-2
Groups that have been masked
No information
Sample size
Target sample size:
46
Randomization (investigator's opinion)
Not randomized
Randomization description
Blinding (investigator's opinion)
Not blinded
Blinding description
Placebo
Not used
Assignment
Parallel
Other design features
Secondary Ids
empty
Ethics committees
1
Ethics committee
Name of ethics committee
Ethics committee of Shahid Beheshti University of Medical Sciences
Street address
Shahid Beheshti University of Medical Sciences, Shahid Arabi St., Yemen St., Shahid Chamran Highway, Tehran
City
Tehran
Province
Tehran
Postal code
1985717443
Approval date
2020-12-28, 1399/10/08
Ethics committee reference number
IR.SBMU.REC.1399.037
Health conditions studied
1
Description of health condition studied
Cerebral palsy
ICD-10 code
G80
ICD-10 code description
Cerebral palsy
Primary outcomes
1
Description
Motor System Functioning
Timepoint
Before injection, 3, 6 and 12 months after the intervention
Method of measurement
Physical examination and motor function will be evaluated by the Gross Motor Function Measure (GMFM-66) and Gross Motor Function Classification System (GMFCS)
2
Description
Balance Assessment
Timepoint
Before injection, 3, 6 and 12 months after the intervention
Method of measurement
Physical examination and Berg Balance Scale will be used to evaluate the balance
3
Description
Spasticity Assessment
Timepoint
Before injection, 3, 6 and 12 months after the intervention
Method of measurement
Physical examination and Grading of spasticity will be classified by the Modified Ashworth Scale which consist of 5 grades where 0 represents no increase in muscle tone and 5 is rigidity in flexion and extension in the affected part.
4
Description
Motor and Cognitive Disability Assessment
Timepoint
Before injection, 3, 6 and 12 months after the intervention
Method of measurement
Physical examination and Functional Independence Measure (FIM) and Functional Assessment Measure (FAM) will be used to measure patient motor and cognitive disability in a wide range of conditions.
5
Description
Evaluation the side effects of autologous bone marrow derived mesenchymal stem cells transplantation in cerebral palsy patients
Timepoint
1, 3, 6 and 12 months after injection
Method of measurement
Adverse effects will be assessed based on common terminology criteria for adverse events (CTCAE, version 4.3)
Secondary outcomes
empty
Intervention groups
1
Description
Intervention group: At first, the patient's personal and demographic information will be recorded completely. The necessary questionnaires and physical examinations will be performed and the results will be recorded. In this group, patients' stem cells will be isolated by bone marrow aspiration. After culturing and preparing the cells, MSC cells will be transplanted at a concentration of 1 million cells per ml prepared through a puncture lumbar in the L4 / L5 region. Patients will undergo periodic examinations in neurology clinic at regular intervals after transplantation. patients' sensory and motor function, spasticity and balance will be assessed. In addition, patients are examined for complications such as headache, stiff neck, nausea and vomiting, shortness of breath, jaundice and infection,....
Category
Treatment - Other
2
Description
Control group: In this group, patients do not receive cell injection and receive supportive therapies.