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 Open-label Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacodynamics, and Pharmacokinetics of Intravenous RGX-202 Gene Therapy in Males with Duchenne Muscular Dystrophy (DMD)

Next Previous
X
Search
Trial Profile

A Phase 1/2/3 Open-label Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacodynamics, and Pharmacokinetics of Intravenous RGX-202 Gene Therapy in Males with Duchenne Muscular Dystrophy (DMD)

Status: Recruiting
Phase of Trial: Phase II/III

Latest Information Update: 14 Mar 2025

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 202-REGENXBIO (Primary)
  • Indications Duchenne muscular dystrophy
  • Focus Adverse reactions; First in man; Pharmacodynamics
  • Acronyms AFFINITY DUCHENNE
  • Sponsors REGENXBIO

    • Most Recent Events

    • 13 Mar 2025 According to a REGENXBIO media release, company expects to share additional Phase I/II biomarker data at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, including the first biomarker data from the cohort of patients aged 1-3. The company expects to share additional efficacy and safety data, including additional functional data, in the first half of 2025.
    • 13 Mar 2025 According to a REGENXBIO media release, In November 2024, REGENXBIO announced the first patient had been dosed in the pivotal phase. Trial will enroll approximately 30 patients aged 1+ in the U.S. and Canada, the pivotal trial is nearly 50% enrolled, and REGENXBIO expects to complete enrollment in the study in 2025, share top line data in the first half of 2026, and submit a BLA under the accelerated approval pathway in mid-2026.
    • 24 Jan 2025 According to ClinicalTrials.gov record, The protocol of this study has been amended to change the primary endpoints due to which overall trial focus was also changed to AR and PD. There was also a change in phase of the study to Phase II/III and planned patient number to 65. Arms of the study were also changed.

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