Either you have JavaScript disabled or your browser does not support Javascript . To work properly, this page requires JavaScript to be enabled.
How to enable JavaScript in your browser?

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)

Next Previous
X
Search
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: 11 Sep 2024

Price : $35 *
  • Adis is an information provider.
  • Final gross price and currency may vary according to local VAT and billing address.
  • Your purchase entitles you to full access to the information contained in our trial profile at the time of purchase.
  • A link to download a PDF version of the trial profile will be included in your email receipt.

At a glance

  • Drugs RGX 121 (Primary) ; RGX 121 (Primary)
  • Indications Mucopolysaccharidosis II
  • Focus Adverse reactions; Biomarker; First in man; Registrational; Therapeutic Use
  • Acronyms CAMPSIITE
  • Sponsors REGENXBIO

    • Most Recent Events

    • 03 Sep 2024 According to a REGENXBIO media release, company is on track to initiate a rolling Biologics License Application (BLA) submission using the accelerated approval pathway in the third quarter of 2024 using CSF D2S6 as a surrogate endpoint reasonable likely to predict clinical benefit. Approval of the planned BLA could result in receipt of a Priority Review Voucher in 2025.
    • 03 Sep 2024 According to a REGENXBIO media release, positive results from this study were presented at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium 2024.
    • 27 Aug 2024 According to a REGENXBIO media release, data from this trial will be presented at Inborn Errors of Metabolism (SSIEM) 2024 Annual Symposium taking place in Porto, Portugal from September 3-6, 2024.

You need to be a logged in or subscribed to view this content Request demo

If your organization or you do not have a subscription, try one of the following:
  • Contacting your organization’s admin about adding this content to your AdisInsight subscription
  • Buying a PDF version of any individual profile
  • Request a free trial
If your organization has a subscription, there are several access options, even while working remotely:
  • Working within your organization’s network
  • with username/password or try to via your institution
  • Persisted access using your organization’s identifier stored in your user browser for 90 days
Back to top