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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03566043 | CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome) | PHASE2 | ACTIVE NOT_RECRUITING | 48 | — | — | Sep 27, 2018 | Aug 1, 2025 | Jan 28, 2025 | 5 | United States, Brazil |
| NCT04571970 | RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome) | PHASE1 | COMPLETED | 6 | — | — | Mar 11, 2021 | May 23, 2024 | Jan 28, 2025 | 2 | United States, Canada |
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...