Gene therapy trial expanded to include teenagers with LHON

Regulatory agencies and ethics committees in the United States, France and the United Kingdom will allow Gensight Biologics to amend its ongoing phase 3 trials to include teenage patients, aged 15 to 18, with Leber hereditary optic neuropathy (LHON).

The initial protocols for GS010, an adeno-associated virus (AAV) targeting the ND4 mutation, were designed to enroll adult patients. However, available epidemiologic data suggests that teenage patients may represent between 14% to 22% of all LHON patients (all mutations included).

“Considering that affected adolescents with LHON present in a similar fashion to adults with the disease, and the current good safety profile and potential prospect for benefit, it is extremely important to be able to include these teenage patients with LHON,” said Nancy J. Newman, MD, director of Neuro-Ophthalmology at Emory University School of Medicine.

The trials, RESCUE and REVERSE, are 2 separate randomized, double-masked, sham-controlled trials designed to evaluate the efficacy of a single intravitreal injection of GS010 in subjects affected by LHON due to the G11778A mutation in the mitochondrial ND4 gene.

The trials are ongoing in 7 centers across the United States, the UK, France, Germany and Italy. Topline results at 48 weeks are expected in early 2018.