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IRCT20180228038899N1 IRCT 2018-07-16 Pasture Institute of Iran Effect of Allogenic Stem Cell in Neurometabolic disorders Evaluation of safety and efficacy of Allogenic Mesenchymal Stem Cell transplantation (core matrix extracted)in Late-Onset Neurometabolic disorders: an open label study 2017-02-21 anticipated 25 Complete https://irct.ir/trial/30396 interventional Randomization: N/A, Blinding: Not blinded, Placebo: Not used, Assignment: Single, Purpose: Treatment. 2 Condition 1: Progressive Ataxia. Condition 2: Myopathy. Condition 3: Neuropathy. Condition 4: Leukodystrophy. 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 prepared in clean room by Sinacell company, 3-4 million cell per kilogram more than 90% viability in 20-50 milliliter injectable human albumin as preservative in each intravenous injection and one million per kilogram in 5-10 milliliter in each intrathecal injection.Interval of these injections is between two days to two weeks. The hospitalization time will be about 6 hours for intravenous injection and 24 to 24 48 hours for intrathecal injection. Patients will undergo a clinical and laboratory evaluation for a period of 2 years at defined intervals.. Yes - There is a plan to make this available What will be shared: After keeping samples privacy , we will share their demographic data and all of findings. When: We will publish our findings as an article after 6 months. To whom: Somebody are interesting to research in the field of Stem cell. Conditions: For researchers in the field of stem cells, statistical analyzes can be done. Where to obtain: 1-Dr. Bita Shalbafan +989161119656 shalbafan.b@gmail.com 2-Dr. Mandana Mohyeddin Bonab +989122024962 mohyeddin@sina.tums.ac.ir How to obtain: Six months after the final article is published, the applicants will submit their application by email or telephone and receive data within a maximum of one month by email. Comments: no
public Bita Shalbafan
No.133,9th Boustan Ave.,Pasdaran Ave.
Tehran Iran (Islamic Republic of) 1666659534 +98 21 2278 3140 shalbafan.b@gmail.com Iran Social Security Organization scientific Bita Shalbafan
No.133,9th Boustan Ave.,Pasdaran Ave.
Tehran Iran (Islamic Republic of) 1666659534 +98 21 2278 3140 shalbafan.b@gmail.com iran social security Organization Iran (Islamic Republic of) 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 18 years 40 years Both 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 G11 M62.5 G60 E75.2 Hereditary ataxia Muscle wasting and atrophy, not elsewhere classified, unspecified site Hereditary and idiopathic neuropathy Other sphingolipidosis Treatment - Drugs 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 prepared in clean room by Sinacell company, 3-4 million cell per kilogram more than 90% viability in 20-50 milliliter injectable human albumin as preservative in each intravenous injection and one million per kilogram in 5-10 milliliter in each intrathecal injection.Interval of these injections is between two days to two weeks. The hospitalization time will be about 6 hours for intravenous injection and 24 to 24 48 hours for intrathecal injection. Patients will undergo a clinical and laboratory evaluation for a period of 2 years at defined intervals. 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. Pasture Institute of Iran Approved 2017-02-21 Ethics committee of Shahid Beheshti University of Medical Sciences Research Management office, 6th floor, building No.2, Shahid Beheshti Medical University, Arabi Ave., Daneshjoo Blvd., Velenjak Town Tehran Tehran Iran (Islamic Republic of)