Spinal Muscular Atrophy Type 1-2 3. Spinal muscular atrophy and related syndromes
Conditions
Interventions
Intervention 1: Intervention group 1: Type 1 SMA patients that will receive three intrathecal injections of mesenchymal stem cells derived from umbilical cord, prepared by Royan stem cell Technology
Sponsors
Tehran University of Medical Sciences
ROYAN stem cell technology Co
Eligibility
Sex/Gender
All
Age
6 Months to 16 Years
Inclusion criteria
Inclusion criteria: Genetically confirmed deletion of SMN1 gene for the diagnosis of SMA disease, SMN2 gene copy number count for the diagnostic classification of disease type, Minimum age of 6 months in type I, Ventilator independent patients at the beginning of treatment in type I, Maximum age of 16 years in type II and III, Ventilator independent patients in type II and III, Absence of brain damage, Absence of liver disease, Absence of renal disease, Absence of hematological disease, Informed consent of patients and their parents
Exclusion criteria
Exclusion criteria: Acquired brain damage including hypoxia, ?Structural and functional brain disorders, Acute infections such as( HCV, HIV,HBV), Malignancies, Hemorrhagic diathesis, Severe anemia ( Hb less than 8 gram/dl), Renal dysfunction, Hepatic dysfunction
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Evaluation of effectiveness of umbilical cord derived mesenchymal stem cells in increasing life expectancy of SMA type 1. Timepoint: Before intervention and then every 2 months till 1 year. Method of measurement: Questionnaire and clinical exams.;Evaluation of effectiveness of umbilical cord derived mesenchymal stem cells in increasing muscle strength of SMA type 1. Timepoint: Before intervention and then every 2 months till 1 year. Method of measurement: Hammersmith Infant Neurological Examinations (HINE) ,Children's Hospital of Philadelphia Questionnaire (CHOP intent ).;Evaluation of effectiveness of umbilical cord derived mesenchymal stem cells in increasing muscle strength of SMA type 2. Timepoint: Before intervention and then every 4 months till 1 year. Method of measurement: Expanded Hammersmith Functional Motor Scale(HFMSE) WHO Motor Mile stones (WMM) questionnaire , Upper limb Module Scale (ULMS).;Evaluation of effectiveness of umbilical cord derived mesenchymal stem cells in increasing muscle strength of SMA type 3. Timepoint: Before intervention and then every 4 months till 1 year. Method of measurement: Expanded Hammersmith Functional Motor Scale(HFMSE) WHO Motor Mile stones (WMM) questionnaire, Upper limb Module Scale (ULMS) questionnaire, 6 Minute Walk Test (6MWT). | — |
Secondary
| Measure | Time frame |
|---|---|
| Improvement of Electrodiagnosic evaluative indexes of SMA patients. Timepoint: Before intervention , 2 months after last injection and then every 4 months till 1 year of first injection. Method of measurement: Motor unit number estimation (CMAP scan).;Number of participants experiencing adverse effects and serious adverse effects. Timepoint: First 24 hours after injection and then any time if occurred. Method of measurement: Questionnaire , parents report and periodic planned clinical evaluations. | — |
Countries
Iran (Islamic Republic of)
Contacts
Public ContactMorteza Heidari
Tehran University of Medical Sciences
Outcome results
None listed