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 Safety Study in Subjects With Leber Congenital Amaurosis (LCA) Using Adeno-Associated Viral Vector to Deliver the Gene for Human RPE65 Into the Retinal Pigment Epithelium (RPE) [AAV2-hRPE65v2-101]

Next Previous
Trial Profile

A Phase 1 Safety Study in Subjects With Leber Congenital Amaurosis (LCA) Using Adeno-Associated Viral Vector to Deliver the Gene for Human RPE65 Into the Retinal Pigment Epithelium (RPE) [AAV2-hRPE65v2-101]

Status: Completed
Phase of Trial: Phase I

Latest Information Update: 13 Mar 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 Voretigene neparvovec (Primary)
  • Indications Leber congenital amaurosis
  • Focus Adverse reactions
  • Acronyms LCARPE
  • Sponsors Spark Therapeutics

    • Most Recent Events

    • 30 Oct 2020 Status changed from active, no longer recruiting to completed.
    • 23 Nov 2018 According to a Novartis media release, the EC decision for approval of Luxturna, is based on a positive CHMP opinion opinion, that looked at the data from this phase 1 clinical trial (NCT00516477), its follow-up trial (NCT01208389) and a phase 3 trial (NCT00999609).
    • 23 Nov 2018 According to a Spark Therapeutics media release,European commission has granted a marketing authorization for Luxturna (voretigene neparvovec), for the treatment of adult and pediatric patients with vision loss due to inherited retinal dystrophy, this authorization will be valid in all 28 member states of the EU, as well as Iceland, Liechtenstein and Norway.
Subscriber content

You need to be a logged in subscriber to view this content.

If your organization has a subscription then there are several options available to help you access AdisInsight, even while working remotely.

  • IP authentication when working within your organization’s network.
  • with username/password or try via your institution
  • Persisted access using your organization’s identifier stored in your user browser for 90 days.
  • Contact us at AsktheExpert.AdisInsight@springer.com for assistance.

If your organization does not have a subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Buy a PDF version of the profile.
  • Request a free trial
Legal
Back to top