Effect of Allogenic Stem Cell in Neurometabolic disorders

Phase 2

Recruiting

• Conditions: 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

• Registration Number: IRCT20180228038899N1
• Lead Sponsor: Pasture Institute of Iran

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Last Update Posted: 11 September 2018

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 25

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

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

Study & Design

• Study Type: interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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.