RESCUE and REVERSE Long-term Follow-up

Status:	Recruiting
Conditions:	Ocular, Ocular
Therapuetic Areas:	Ophthalmology
Healthy:	No
Age Range:	15 - Any
Updated:	4/17/2018
Start Date:	January 9, 2018
End Date:	August 2022
Contact:	Barrett Katz, MD
Email:	bkatz@gensight-biologics.com
Phone:	+1-646-831-3799

Long-term Follow-up of ND4 LHON Subjects Treated With GS010 Ocular Gene Therapy in the RESCUE or REVERSE Phase III Clinical Trials

The goal of this clinical trial is to assess the long-term safety and efficacy of GS010, a
gene therapy, and assess the quality of life in subjects with LHON due to the G11778A ND4
mitochondrial mutation and who were treated in the Rescue or Reverse studies.

Inclusion Criteria Subjects must meet all the following criteria at Inclusion period to be
eligible for inclusion.

- Subject was treated with GS010 IVT injection in either of the RESCUE or REVERSE Phase
III clinical studies

- Subject of legal consent age has provided informed consent; subjects that are not of
legal consent age have undergone their country-approved clinical trial enrollment
consent process

Non-Inclusion Criteria Subjects meeting any of the following criteria at Inclusion period
will be excluded from eligibility.

- Subject is unwilling or unable to comply with the protocol requirements

- Subject has any medical or psychological condition that, in the opinion of the
Investigator, may compromise his or her safe participation in the study

- Subject is taking or intending to take idebenone during the long-term follow-up study
period

We found this trial at

sites

Emory University Hospital

1364 Clifton Rd NE
Atlanta, Georgia 30322

(404) 712-2000