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 I/II single center, open-label study to evaluate the safety, tolerability, pharmacodynamics and preliminary efficacy of intracisternal RGX-111 gene therapy in subjects with severe mucopolysaccharidosis type I

Next Previous
Trial Profile

A phase I/II single center, open-label study to evaluate the safety, tolerability, pharmacodynamics and preliminary efficacy of intracisternal RGX-111 gene therapy in subjects with severe mucopolysaccharidosis type I

Status: Not yet recruiting
Phase of Trial: Phase I/II

Latest Information Update: 28 Jan 2020

Price :
$35 *
Note:
  • 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 111 (Primary)
  • Indications Mucopolysaccharidosis I
  • Focus Adverse reactions
  • Sponsors REGENXBIO

Most Recent Events

  • 15 Nov 2018 New trial record
  • 07 Nov 2018 According to a REGENXBIO media release, the company has ubmitted an application to the Brazilian Health Surveillance Agency (ANVISA) to proceed with a Phase I clinical trial evaluating RGX-111 for treatment of MPS I in patients under the age of 3 which it expects will allow the company to activate an initial site in Brazil during the first half of 2019. Dosing of first subject is expected in the first half of 2019.

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

Request demo (opens in a new window)
If your organization or you do not have a subscription, try one of the following: 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