New patients required for pioneering X-linked RP clinical trial

10 May 17

written by:

Press Office

(more articles)

Following the recent announcement of a new trial at the University of Oxford to treat retinitis pigmentosa (RP), lead researcher Professor Robert MacLaren has issued a call for more patients to volunteer and be part of this ground breaking research project. It was previously reported that the first patient underwent successful surgery on 16 March 2017.

The clinical trial to treat X-linked RP, which is currently untreatable and leads to progressive and irreversible sight loss, is sponsored by NightstaRx Ltd, a University of Oxford spin out company set up to develop treatments for RP using gene therapy. The current trial uses gene therapy to restore the full length RPGR gene to photoreceptor cells. The preliminary work funded by the Medical Research Council showed that small modifications to the vector can make the gene stable so that the correct RPGR sequence is restored (Fischer et al., Molecular Therapy 2017). Until now, other gene therapy approaches have only been able to generate mutated versions of the gene, which may not be effective in patients.

Now more volunteers are needed and anyone interested in joining the gene therapy trial should arrange for a routine GP referral to see Professor Robert MacLaren for review at the Oxford Eye Hospital. Please note that this trial is only for UK-based male patients with X-linked RP caused by a confirmed mutation in the RPGR gene. Patients who are under review at other hospitals are also welcome, but should first discuss their eligibility with their regular consultant. The main purpose of the trial is to assess the safety and tolerability of the gene therapy over a 12 month period and it is hoped that at least 24 patients will be enrolled in the trial.

A second RPGR gene therapy trial centre in Manchester will shortly be opened. Anyone who is unsure about eligibility or who would like further information should contact NightstaRx at

Fight for Sight has been a key funder of the gene therapy research in Oxford that has enabled this trial to commence. In particular, this trial would not have happened without the ground breaking research that has led to trials of gene therapy to treat choroideremia (a rare inherited disorder that also causes progressive sight loss) which were greatly facilitated by the Tommy Salisbury Choroideremia Fund at Fight for Sight.

Michele Acton, CEO of Fight for Sight, said: “This is the world’s first trial to test a treatment for retinitis pigmentosa caused by the RPGR gene which is essential for the functioning of the light sensitive cells at the back of the eye. We are very proud that Fight for Sight’s support for research in this area has helped lead to the start of another clinical trial – but funding is just part of the story. The role of volunteer patients is vital to the success of any clinical trial and I would urge people to come forward as this could make a significant difference to the lives of so many. We wish the team in Oxford continued success in the coming months”. 

New trial starts to treat X-linked Retinitis Pigmentosa