Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01395641 | A Phase I/II Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC | PHASE1 | COMPLETED | 10 | — | — | Oct 22, 2014 | Mar 7, 2022 | Feb 10, 2023 | 1 | Taiwan |
1. At one year post-surgery, neurotransmitter metabolites (HVA or HIAA) is detectable in CSF (higher than that at pre-surgery) 2. At one year post-surgery, PDMS-II score is higher than that at pre-surgery, with an improvement over 10 points
| Arm | Type | Description |
|---|---|---|
| Gene therapy | EXPERIMENTAL | Intracerebral infusion of AAV2-hAADC viral vector will be performed |
| Name | Type | Description |
|---|---|---|
| gene therapy | DRUG | AAV2-hAADC viral vector will be injected into bilateral putamen by stereotactic surgery. |
Inclusion Criteria: 1. With a confirmed diagnosis of AADC, including cerebrospinal fluid analysis to show reduced levels of neurotransmitter metabolites, HVA and 5-HIAA, and higher L-Dopa, together with more than one mutation within AADC gene. 2. Classical clinical characteristics of AADC deficienc...