Study identification

PURI

https://redirect.ema.europa.eu/resource/46265

EU PAS number

EUPAS31153

Study ID

46265

Official title and acronym

A Post-Authorization, Multicenter, Multinational, Longitudinal, Observational Safety Registry Study for Patients Treated with Voretigene Neparvovec

DARWIN EU® study

No

Study countries

Argentina
Austria
Brazil
Canada
Denmark
Finland
France
Germany
Israel
Italy
Korea, Democratic People's Republic of
Mexico
Netherlands
Norway
Russian Federation
Saudi Arabia
Singapore
Sweden
Switzerland
Taiwan
Türkiye
United Arab Emirates
United Kingdom

Study description

This is a global (ex-US) non-interventional registry-based, post-authorization study (PASS) in pediatric and adult patients who have received Luxturna® sub-retinal injections in a real-world setting. Patients will be treated according to the local prescribing information and routine medical practice. The study will collect all AEs and SAEs including AEs of special interest (AESIs), information about pregnancy occurrence and outcomes, and ophthalmic examination results. A five year enrollment duration is expected to provide a minimum of 40 participants who are to be followed annually for 5 years.

Study status

Ongoing
Research institution and networks

Institutions

Contact details

Novartis Clinical Disclosure Officer

Primary lead investigator
Study timelines

Date when funding contract was signed

Planned:
Actual:

Study start date

Planned:
Actual:

Data analysis start date

Planned:

Date of interim report, if expected

Planned:
Actual:

Date of final study report

Planned:
Sources of funding
Pharmaceutical company and other private sector 

More details on funding

Novartis
Study protocol
Initial protocol

LTW888A12401 PASS Protocol_July 2019_Redacted_21 Aug 2019

English (861.51 KB - PDF)View document
Updated protocol

CLTW888A12401-v02--protocol_Redacted

English (980.31 KB - PDF)View document
Regulatory

Was the study required by a regulatory body?

Yes

Is the study required by a Risk Management Plan (RMP)?

EU RMP category 1 (imposed as condition of marketing authorisation)