Nightstar Therapeutics Announces Initiation of STAR Phase 3 Registrational Trial for NSR-REP1 in Choroideremia

Nightstar Therapeutics has announced [5 March 2018] the initiation of the first ever phase III trial for a treatment of choroideremia. The trial will study the safety and efficacy of the NSR-REP1 gene therapy treatment in patients with choroideremia. Approximately 140 patients are expected to be enrolled and randomised into one of three study arms. 56 patients will receive a high-dose of NSR-REP1 in one eye, 28 patients will receive a low-dose of NSR-REP1 in one eye and 56 patients will receive no treatment.

Fight for Sight, inspired by the Salisbury family and their son Tommy, played a crucial early role in the story of the development of gene therapy for choroideremia. Back in 2005, when Tommy was diagnosed with the condition at the age of 4, his mother became aware that there was no treatment and vowed to change that. She’s fundraised tirelessly since then and recently passed the milestone of having raised £500,000. The Tommy Salisbury Choroideremia Fund at Fight for Sight supported a researcher at Imperial College London, Professor Miguel Seabra, largely focussed on modelling of the condition. This was the first chapter in the story which has now led to the announcement of the phase III clinical trial.

Michele Acton, Fight for Sight’s CEO, said: “This announcement from NightStar is incredibly exciting. Charities like Fight for Sight play a vital role in funding early stage research. We are thrilled that NightStar is now undertaking a Phase III clinical trial to help address this blinding condition.”

This story uniquely highlights how active, engaged and committed patients, working with charities, can make a real difference.

Click here to view the full NightStar announcement.

Click here for more on Tommy Salisbury.