Clinical trials play a central role in improving our understanding of eye diseases and developing new treatments for vision loss. Check out our list of current clinical trials and register your interest in participating.

1. Belite Study:

Status: No longer recruiting

PI: PD Dr. med Maximilian Pfau

Research Coordinators: clinicaltrialcenter@iob.ch

For eligibility and more information, please see: https://clinicaltrials.gov/ct2/show/NCT05244304

Background: STGD1 is the most common form of inherited retinal disease passed from parents to their children. STGD1 is a form of macular degeneration and is often also known as juvenile macular degeneration.

The most common cause of STGD1 disease is a mutation in a gene called ABCA4, which leads to excessive accumulation of certain lipids in the retina, causing photoreceptors (the cells in the retina that respond to light) to break down (lose their function).

Design: Approximately 60 participants between the ages of 12 and 18 will participate in this global study. The study will be conducted at multiple trial sites in Australia, Asia, Europe and North America. The study will evaluate tinlarebant at a dose of 5 mg. Tinlarebant is an investigational drug being developed to slow the progression of STGD1.

The study will last more than 2 years.

2. Meira GTx Study:

Status: No longer recruiting

PI: PD Dr. med Maximilian Pfau

Research Coordinators: clinicaltrialcenter@iob.ch

For eligibility and more information, please see: https://clinicaltrials.gov/ct2/show/NCT03252847

Background: Retinitis pigmentosa (RP) is a group of inherited retinal diseases characterized by a progressive reduction in vision that first manifests as nyctalopia (night blindness) and usually occurs in childhood or early adulthood and progresses throughout life (Tee 2016). Due to a number of X-linked forms, RP affects more males than females.

Currently, there is no approved therapeutic treatment for XLRP caused by mutations in RPGR (RPGR-XLRP). Among a number of new experimental strategies currently under investigation, gene therapy is considered the most promising.

This gene therapy study aims to determine whether a healthy version of the RPGR gene can correct this inherited error (defect) when delivered by injection into the retina during surgery.

Design: Approximately 90 adult subjects and up to 6 children and adolescents worldwide will participate in this study.

As part of this study, patients will be asked to give their consent to participate in 2 closely related studies. All participants must read the information about both studies and decide if they want to consent to participate. Initially, patients will participate in the first study (MGT-RPGR-021) for up to 18 months. Once patients have completed their participation in this study, they will proceed to the second study (MGT-RPGR-022), which has a follow-up period of 4 to 5 years.

The length of participation will depend on which treatment group the patient is assigned to in the study. It is necessary to have a group of non-treated participants to compare benefits and risks. Therefore, some participants will receive their treatment with a delay of 12 months. All participants will receive either the RPGR2e11 or RPGR4e11 dose of gene therapy tested in both eyes, either in the MGT-RPGR-021 study or 12 months later in the MGT-RPGR-022 study.

3. Nac Attack:

Status: Active

PI: PD Dr. med Kristina Pfau

Research Coordinators: clinicaltrialcenter@iob.ch

For eligibility and more information, please see: https://clinicaltrials.gov/ct2/show/NCT05537220

Background: NAC Attack is a Phase III, multicenter, randomized, placebo-controlled study to evaluate the efficacy and safety of oral NAC in patients with retinal pigmentosa.

Retinitis pigmentosa (RP) is an inherited retinal disease resulting from one or more genetic mutations. The mutations cause photoreceptor cells to die, resulting in loss of night vision as the first symptom. Gradually, patients then notice narrowed visual fields, followed by a loss of visual acuity over years or decades, culminating in blindness.

Design: The study will enroll a total of 438 participants with RP and randomize them to approximately 30 clinical sites in the Americas and Europe. Patients will be eligible if both eyes have an RP phenotype consisting of severe loss of rod function followed by progressive visual field loss and maintenance of visual acuity.

During the screening phase, inclusion and exclusion criteria are assessed and eligibility for participation in the study is determined. Patients eligible to participate in the study will be randomized in a 2:1 ratio to one of two arms stratified by the clinical site of the study:

- Intervention of NAC 1800 mg bid

- Placebo

Each participant will be followed for 45 months after randomization. Study visits, including clinic visits, tele-visits with the on-site investigator, and phone calls with the on-site coordinator.