JPRN-UMIN000017802
Recruiting
未知
Clinical research of the gene therapy for AADC deficiency
ConditionsAADC deficiency
Overview
- Phase
- 未知
- Intervention
- Not specified
- Conditions
- AADC deficiency
- Sponsor
- JIchi Medical University
- Enrollment
- 5
- Status
- Recruiting
- Last Updated
- 2 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •1\.Typical AADC deficiency patients who was unable to stand with motor disturbance and dystonia. Diagnosis was confirmed from the findings of CSF analysis, enzyme activity or genetic analysis.
- •2\.Age; \=\> 4 years at the time of medical treatment. No restriction of age upper limit.
- •3\.No findings suggestive of CNS Degenerative Disease are found.
- •4\.To be able to comply with the requirements of this study, including the frequent clinical examination after medical treatment.
- •5\.To keep the therapeutic medicine for AADC deficiency for at least 2 months prior to participation in this study.
- •6\.Written informed consent from patient' s parental authorities.
Exclusion Criteria
- •1\.Mild AADC deficiency patients who can stand and walk.
- •2\.Patients with history of significant cardiovascular disease including cerebrovascular accident.
- •3\.Malignant neoplasm in the brain, clinically significant neurological disease.
- •4\.History of malignancy, with the exception of treated carcinoma cutaneum, within 5 years.
- •5\.Uncontrolled hypertension: systolic blood pressure over 160 mmHg.
- •6\.Coagulopathy or need for anticoagulant therapy.
- •7\.Clinically significant immune dysfunction (for example, the case who require the use of immunosuppresive drugs).
- •8\.Unable to scan MRI.
- •9\.Cases without abnormal finding in FMT\-PET.
- •10\.Past medical history of serious drug allergy.
Outcomes
Primary Outcomes
Not specified
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