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The mesenchymal stem cell therapy for Hutchinson-Gilford Progeria syndrome

Not Applicable
Conditions
Endocrine, nutritional and metabolic diseases
Registration Number
KCT0008336
Lead Sponsor
Inha University Hospital
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
ot yet recruiting
Sex
All
Target Recruitment
1
Inclusion Criteria

1. Genetically confirmed Hutchinson-Gilford progeria syndrome (progeria in childhood)
2. Typical clinical symptoms of progeria identified by medical staff in this clinical study
3. Aspartate aminotransferase (AST) and alanine aminotransfers (ALT) at the time of enrollment/screening < 5× upper limit of normal
4. Research subjects and legal representatives who must be able to visit the center according to this research plan and who are willing to participate voluntarily

Exclusion Criteria

Research subjects who fall under any of the following are excluded from the study.
1. Severe renal impairment (GFR < 30ml/min/1.73m2)
2. Other clinical study medications received 90 days prior to the first treatment visit
3.Persons with serious adverse reactions during stem cell treatmen
4. In the case of a medical condition that prevents safe participation in the study according to the opinion of the research director

Study & Design

Study Type
Interventional Study
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Assessment of cerebrovascular and carotid artery conditions
Secondary Outcome Measures
NameTimeMethod
? Joint contracture evaluation ? Perform serological test and cytokine analysis ? Cytokine: analysis of proinflammatory cytokines at the mRNA level (IL-1ß, TNF-a, CRP, TLR4, IL-8, ICAM-1, MCP-1. 18s) ? Bone density evaluation ? Echocardiography ? Eye examination ? Otolaryngology examination ? IQ test
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