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RGX-121-3102 · 1 trial · 2 indications
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 ...
RGX-121-3102 is an investigational gene therapy being developed for the treatment of MPS II, also known as Hunter syndrome. It is designed to address the underlying genetic cause of this rare disease. The therapy is currently in Phase 3 clinical development.
RGX-121-3102 is being developed by REGENXBIO Inc. (NASDAQ: RGNX). The company is conducting a Phase 3 clinical trial to evaluate the therapy in participants with MPS II (Hunter syndrome).
RGX-121-3102 is in Phase 3 clinical development. It is an investigational gene therapy and has not been approved by the FDA. The therapy has received several FDA designations, including Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug, Rare Pediatric Disease, Fast Track, Accelerated Approval, and Priority Review.
RGX-121-3102 is being studied in a Phase 3 clinical trial with the identifier NCT07236606. This trial is titled 'RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)' and is currently active but not recruiting. The trial is enrolling male participants aged 4 months and older in the United States.
RGX-121-3102 is a specific formulation or version of the gene therapy being developed by REGENXBIO. The clinical trial identifier NCT07236606 refers to this specific therapy. It is important to note that RGX-121-3102 is distinct from other RGX-121 products, and its development is focused on MPS II.