RESCUE and REVERSE Long-term Follow-up

Plain-language summary

This Phase 3 follow-up study assesses the long-term safety and efficacy of the gene therapy GS010 (lenadogene nolparvovec), as well as quality of life, in subjects with LHON due to the G11778A ND4 mitochondrial mutation who were treated in the RESCUE or REVERSE studies. Participants must have received a GS010 intravitreal injection in either of those Phase 3 trials. Those of legal consent age provide informed consent, while younger subjects follow their country-approved trial enrollment consent process.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Phase 3

Status

Completed

Sponsor

GenSight Biologics

Intervention

GS010 (Lenadogene Nolparvovec), Sham Intravitreal Injection

Start date

1/9/2018

Estimated completion

7/4/2022

Countries

United States, France, Germany, Italy, United Kingdom

Locations

• Pasadena, United States
• Atlanta, United States
• Philadelphia, United States
• Paris, France
• Munich, Germany
• Bologna, Italy
• London, United Kingdom

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria

* 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

Exclusion Criteria

* 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