Condition 1: Progressive Ataxia. Condition 2: Myopathy. Condition 3: Neuropathy. Condition 4: Leukodystrophy. Hereditary ataxia Muscle wasting and atrophy, not elsewhere classified, unspecified site Hereditary and idiopathic neuropathy Other sphingolipidosis
Conditions
Interventions
Intervention group:?After patient selection ,one or much more injections will be planned by intrathecal and/or intravenous rout based on CNS involvement . Injectable mesenchymal stem cell will be prep
Sponsors
Pasture Institute of Iran
Eligibility
Sex/Gender
All
Age
18 Years to 40 Years
Inclusion criteria
Inclusion criteria: The definitive diagnosis of neurometabolic diseases leads to ataxia, leukodystrophy,Myopathy or Neuropathy based on laboratory findings of molecular genetics Age between 18-40 years old Written consent of the patient and parents to attend the study Normal routine biochemistry, hematology and negative serology and virology tests Negative Tumor survey includes abdominal and pelvic ultrasonography and prostatic and breast and thyroid ,and occult blood in the stool test
Exclusion criteria
Exclusion criteria: A pregnant woman (positive pregnancy test) or a nursing or illness who is planning a pregnancy during the study A disease that is in addition to the involvement of a nervous system with another serious illness, such as hemodynamic disorders, homeostasis disorders, diabetes, cardiovascular / pulmonary disease, etc. Having a serious psychiatric illness or having a history of suicide Treatment with cytotoxic drugs within a month before starting the study The presence of any suspected malignancy mass Serum creatinine more than 1.7 Rised liver enzyme tests more than three times White blood cell count lower than 3000 Positive response to each of the serum tests of HTLV1,2 Ab, HIV1,2Ab, HBcAb, HBsAg,HCVAb
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Ataxia in Ataxic patients. Timepoint: At first, 1,3,6,12,18,24 months after intervention. Method of measurement: Scale of Assesment and Rating of Ataxia in Ataxic patients.;Functional Capacity in paretic patients with or without ataxia and mental problems. Timepoint: At first, 1,3,6,12,18,24 months after intervention. Method of measurement: Multiple Sclerosis Functional Capacity scoring.;Blood Sugar assessment in patients with Friedreich Ataxia diagnosis. Timepoint: At first, 1,3,6,12,18,24 months after intervention. Method of measurement: Laboratory measurements of Fasting Blood Sugar and Hemoglobin ?A1C.;Heart failure assessment in patients with Friedreich Ataxia diagnosis. Timepoint: At first, 1,3,6,12,18,24 months after intervention. Method of measurement: Ejection Fraction by Echocardiography. | — |
Countries
Iran (Islamic Republic of)
Contacts
Public ContactBita Shalbafan
Iran Social Security Organization
Outcome results
None listed