A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects with Choroideremia Previously Treated with Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) and in Subjects with X-Linked Retinitis Pigmentosa Previously Treated with Adeno-Associated Viral Vector Encoding RPGR (AAV8-RPGR) in an Antecedent Study (SOLSTICE)
Choroideremia and X-linked retinitis pigmentosa are rare genetic eye conditions that mainly affect men, causing gradual vision loss over time. In earlier clinical trials, some patients received gene therapy designed to slow down or prevent this sight loss by delivering a working copy of a faulty gene to the back of the eye. This follow-up study, known as SOLSTICE, invites men who received those earlier treatments to return for regular check-ups. Because gene therapy is a newer type of medicine, researchers need to monitor participants over a long period. The study team will track how safe the therapy is in the long term, check whether any improvements in vision last, and see if the treatment continues to protect remaining sight. No new surgical treatments are given in this study; it focuses entirely on monitoring.
Auf einen Blick
Worum geht es in dieser Studie?
Choroideremia and X-linked retinitis pigmentosa are inherited conditions affecting the retina, the light-sensitive layer at the back of the eye. Both conditions cause cells in the retina to stop working properly, leading to night blindness, loss of side vision, and eventually loss of central vision. Because the genes responsible are on the X chromosome, these diseases mostly affect men.
Gene therapy aims to treat the root cause of these conditions rather than just managing symptoms. In previous clinical trials, participants received an injection into the eye containing a healthy copy of the missing or faulty gene. The goal was to help the retinal cells survive and function better.
This study is a long-term monitoring programme for people who received that gene therapy in earlier research studies. Long-term studies are vital because they show whether the benefits of a one-off gene therapy last for years, and they ensure that no unexpected safety issues develop over time. By looking closely at how participants are doing, researchers can gather essential evidence to help improve care for people with genetic eye diseases in the future.
Wichtige Erkenntnisse
- This is a long-term follow-up study, not a test of a new treatment.
- It is only for men aged 18 and older who already had gene therapy in a previous trial.
- The main purpose is to see if earlier gene therapy treatments remain safe and keep protecting vision over time.
- Appointments involve routine, non-invasive eye tests, scans, and questionnaires.
- Taking part is voluntary, and you can withdraw at any point.
Wer könnte berechtigt sein?
This study is specifically designed for adult men who have already received gene therapy in an earlier trial for either choroideremia or X-linked retinitis pigmentosa.
You cannot join this study if you have not had this specific gene therapy before. It is not open to people who are looking to receive gene therapy for the first time.
Because these conditions are linked to the X chromosome and the earlier trials focused on men, you must be male and aged 18 or older to take part.
Könnte diese Studie für Sie geeignet sein?
Beantworten Sie diese kurzen Fragen, um zu sehen, ob Sie möglicherweise berechtigt sind. Dies ist nur ein Leitfaden – das Forschungsteam trifft die endgültige Entscheidung.
- I am a man aged 18 or older.
- I have choroideremia or X-linked retinitis pigmentosa.
- I previously received gene therapy in an earlier trial for one of these conditions.
- I am able and willing to attend follow-up eye appointments.
Was beinhaltet die Teilnahme?
Taking part in this study does not involve having any new operations or receiving new medication. Instead, you will attend regular eye clinic visits so the team can monitor your vision and overall eye health. At these visits, you will read letter charts in different lighting conditions, answer questions about how your sight affects your daily life, and have detailed, painless pictures taken of the back of your eye. These tests include standard eye scans, photos, and checks of your visual field. The study will follow you over several years to track any changes in your sight.
Potenzielle Risiken und Vorteile
Standorte (2)
- —UnbestätigtFrankreich
- —UnbestätigtDeutschland
Häufige Fragen
Will I receive an injection or new gene therapy in this study?
No. This study is purely an observational follow-up to check how well your earlier gene therapy is performing over the long term.
Can I join if I have never had gene therapy before?
No. Only people who were treated in specific earlier gene therapy trials for choroideremia or X-linked retinitis pigmentosa can join.
Do the eye scans and tests hurt?
No. The scans and photographs are painless. You may have drops put in your eyes to widen your pupils, which can sting briefly and make your sight blurry for a few hours.
How often will I need to visit the hospital?
The study team will give you a schedule of visits. These are spaced out over months or years, similar to specialised follow-up appointments.
Will travel costs be covered?
Clinical trials often provide help with travel expenses for study visits. You should check the exact details with the study clinic team.
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