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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07236606 | RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome) | PHASE3 | ACTIVE NOT_RECRUITING | 2 | — | — | Nov 25, 2025 | May 30, 2031 | Jun 4, 2026 | 1 | United States |
To evaluate the effect of RGX-121 on the neurodevelopmental function as measured by the Bayley Scales of Infant and Toddler Development, 3rd Edition (BSID-III).
To evaluate the effect of RGX-121 on the neurodevelopmental function as measured by the Kaufman Assessment for Children, 2nd edition (KABC-II). The KABC-II is only given if the participant achieved the max ceiling on the BSID-III Cognitive scale at two consecutive administrations.
To evaluate the safety of RGX-121 for up to five years including serious and adverse events as assessed by Common Terminology Criteria for Adverse Events (CTCAE) (Version 5.0)
| Arm | Type | Description |
|---|---|---|
| 2.9×1011 GC/g brain mass of RGX 121 | EXPERIMENTAL | AAV9.CB7.hIDS |
| Name | Type | Description |
|---|---|---|
| RGX-121-3102 | GENETIC | Recombinant adeno-associated virus serotype 9 \[AAV9\] capsid containing human iduronate-2-sulfatase (hIDS) expression cassette |
Inclusion Criteria: * The participant's legal guardian(s) is (are) willing and able to provide written, signed informed consent after the nature of the study has been explained, and prior to any study-related procedures being performed. * Is a male ≥ 4 months to \< 5 years of age on Day 1. * Has a ...