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A Phase 1/2/3 Multicenter, Open-Label Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of RGX-121 in Pediatric Subjects With MPS II (Hunter Syndrome)

Trial Profile

A Phase 1/2/3 Multicenter, Open-Label Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of RGX-121 in Pediatric Subjects With MPS II (Hunter Syndrome)

Status: Active, no longer recruiting
Phase of Trial: Phase II/III

Latest Information Update: 05 Mar 2024

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At a glance

  • Drugs RGX 121 (Primary) ; RGX 121 (Primary)
  • Indications Mucopolysaccharidosis II
  • Focus Adverse reactions; First in man; Pharmacodynamics; Registrational
  • Acronyms CAMPSIITE
  • Sponsors REGENXBIO
  • Most Recent Events

    • 07 Feb 2024 According to a REGENXBIO media release, following an RMAT meeting held with FDA at the end of 2023, REGENXBIO continues with plans to use CSF levels of D2S6 as a surrogate endpoint for accelerated approval and is completing remaining activities in order to file a BLA in the second half of 2024. Based on an expected priority review, potential approval of the planned BLA could result in receipt of a Rare Pediatric Disease Priority Review Voucher in 2025.
    • 07 Feb 2024 According to a REGENXBIO media release, results from this trial were presented at the 20th Annual WORLDSymposium™ by Paul Harmatz, M.D., UCSF Benioff Children's Hospital and trial investigator.
    • 07 Feb 2024 Topline results from this trial presented in a REGENXBIO media release.
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