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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02716246 | Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH | PHASE2 | RECRUITING | 36 | — | — | Apr 25, 2016 | Jul 1, 2027 | Apr 24, 2026 | 5 | United States, Australia +1 |
Exposure is defined as the time-normalized area under the curve (AUC) of the percentage reduction from baseline.
| Arm | Type | Description |
|---|---|---|
| Cohort 1 Low Dose | EXPERIMENTAL | Dose of 0.5 X 10\^13 vg/kg |
| Cohort 2 Mid Dose | EXPERIMENTAL | Dose of 1 X 10\^13 vg/kg |
| Cohort 3 High Dose | EXPERIMENTAL | Dose of 3 X 10\^13 vg/kg |
| Cohort 4 High Dose (Spain Only) | EXPERIMENTAL | Dose of 3 X 10\^13 vg/kg |
| Name | Type | Description |
|---|---|---|
| UX111 | BIOLOGICAL | Self-complementary adeno-associated virus serotype 9 carrying the human SGSH gene under the control of a U1a promoter (scAAV9.U1a.hSGSH) will be delivered one time through a venous catheter inserted into a peripheral limb vein. |
| Prophylactic Immunomodulatory (IM) Therapy | DRUG | - |
| Optimized Prophylactic IM Therapy | DRUG | - |
| Adjuvant IM Therapy | DRUG | The Principal Investigator and/or caregiver, in consultation with the medical monitor, will determine whether to initiate adjuvant IM therapy. Not all participants may receive adjuvant IM therapy. |
Inclusion Criteria: * Diagnosis of MPS IIIA confirmed by the following methods: * No detectable or significantly reduced SGSH enzyme activity by leukocyte assay, and * Genomic DNA analysis demonstrating homozygous or compound heterozygous mutations in the SGSH gene (based upon review of docume...