Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
RGX-121 · 2 trials · 1 indication
Number of participants with treatment-related adverse events and serious adverse events as assessed by CTCAE (Version 4.03).
CSF GAG levels (as measured by D2S6)
Neurodevelopmental function as measured by the Bayley Scales of Infant and Toddler Development, 3rd Edition (BSID-III) or Mullen Scales of Early Learning (MSEL). The Bayley Scales of Infant Development, or the BSID-III is an individually administered test, designed to evaluate the developmental functioning of infants and small children, between 1 and 42 months of age. The purpose of the test is to identify infants and children with developmental delay. The Mullen Scales of Early Learning (MSEL) is a developmental test to measure cognitive ability, language and motor development. The test has five scales: gross motor, visual reception, fine motor, receptive language, and expressive language. An increase in raw and age equivalent scores indicates neurodevelopmental skill acquisition. Standard scores compare function to age matched normative data.
Number of participants with treatment-related adverse events and serious adverse events as assessed by CTCAE (Version 5.0)
| Arm | Type | Description |
|---|---|---|
| Part 1: RGX-121 Dose 1 | EXPERIMENTAL | 1.3x10\^10 GC/g brain mass of RGX-121 |
| Part 1: RGX-121 Dose 2 | EXPERIMENTAL | 6.5x10\^10 GC/g brain mass of RGX-121 |
| Part 1: RGX-121 Dose 2 Expanded Cohort | EXPERIMENTAL | 6.5x10\^10 GC/g brain mass of RGX-121 |
| Part 1: RGX-121 Dose 3 | EXPERIMENTAL | 2.0x10\^11 GC/g brain mass of RGX-121 |
| Part 1: RGX-121 Dose 3 Expanded Cohort | EXPERIMENTAL | 2.9x10\^11 GC/g brain mass of RGX-121 (transgene-specific PCR assay) equivalent to, 2.0x10\^11 GC/g brain mass of RGX-121 (Poly-A-specific PCR assay) |
| Part 2: RGX-121 Pivotal Expansion | EXPERIMENTAL | 2.9x10\^11 GC/g brain mass of RGX-121 (transgene-specific PCR assay) |
| Single Arm | EXPERIMENTAL | 6.5 × 10\^10 GC/g brain mass of RGX-121 |
| Name | Type | Description |
|---|---|---|
| RGX-121 | GENETIC | Recombinant adeno-associated virus serotype 9 capsid containing human iduronate-2-sulfatase expression cassette |
Part 1 Inclusion Criteria: * The subject'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 research-related procedures * Is a male ≥4 months to \< 5 years of age on Day 1 * Must meet any of th...
RGX-121 is an investigational gene therapy being developed for the treatment of Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome. It is designed for patients with this rare inherited metabolic disorder, which primarily affects males. The therapy is currently in Phase 2 clinical development.
RGX-121 is a gene therapy designed to deliver a functional copy of a gene to address the underlying cause of MPS II. The therapy aims to provide a lasting source of the missing enzyme by introducing the gene into cells, potentially reducing the accumulation of harmful substances associated with the disease.
RGX-121 is being developed by REGENXBIO Inc., a biotechnology company focused on gene therapies. REGENXBIO is publicly traded under the ticker symbol RGNX on the NASDAQ stock exchange. The company is conducting clinical trials to evaluate the safety and efficacy of RGX-121 in patients with MPS II.
RGX-121 is currently in Phase 2 clinical development. It has received multiple FDA designations, including Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug, Rare Pediatric Disease, Fast Track, Accelerated Approval, and Priority Review. These designations reflect the potential of the therapy to address a serious unmet medical need.
RGX-121 is being studied in two clinical trials. The first, NCT03566043, is a Phase 2 study in subjects with MPS II, with an enrollment of 48 participants, and is active but not recruiting. The second, NCT04571970, is a Phase 1 study in children aged 5 years and over, with 6 participants, and has been completed.
RGX-121 is also known as CAMPSIITE, as indicated by the title of one of its clinical trials. The Phase 2 trial is named 'CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome).' Both names refer to the same investigational gene therapy being developed by REGENXBIO.