Current studies

Through national and international clinical studies at our center, we contribute to progress in the development and approval of new drugs and medical devices. All studies and investigations carried out at our center have been reviewed and approved by the Ethics Committee and Swissmedic with regard to ethical, medical-scientific and legal aspects.

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D

DRAGON study (PI Prof. Nicolas Feltgen)

Phase III, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Tinlarebant in the Treatment of Stargardt Disease in Adolescent Subjects
(Phase 3, Multicenter, Randomized, Double-Masked, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Tinlarebant in the Treatment of Stargardt Disease in Adolescent Subjects)

Status: Active

Recruitment: Completed

PI: Prof. Nicolas Feltgen

Sponsor: Belite Bio

Further information: https: //kofam.ch/de/studienportal/nach-klinischen-versuchen-suchen/233957/studie/61489

Background:

Stargardt disease 1 (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 referred to as juvenile macular degeneration. Macular degeneration is a disease that affects the central part of the retina (the macula) and leads to a loss of central vision. Loss of central vision results in the inability to see fine details, read or recognize faces. In patients with STGD1, the light-sensitive cells, called photoreceptors, die, resulting in difficulty seeing colors.
The disease typically causes a loss of vision during childhood or adolescence. Although some forms involve loss of vision, this may not be noticed until later in adulthood. Although the long-term progression of the disease is variable, unfortunately STGD1 eventually leads to very poor vision in most people. However, there is currently no approved treatment for STGD1.
The most common cause of STGD1 disease is a mutation in a gene called ABCA4, which leads to an excessive accumulation of certain lipids in the retina, causing photoreceptors (the cells in the retina that respond to light) to be destroyed (lose their function).

Study design:

Approximately 60 participants between the ages of 12 and 20 are taking part in this global study. The study is being conducted at several trial centers in Australia, Asia, Europe and North America.
The study is investigating tinlarebant at a dose of 5 mg. Tinlarebant is an investigational drug designed to slow the progression of STGD1.

Study duration: 28 months

M

MGT-RPGR-021 / MGT-RPGR-022 Study (PI Prof. Nicolas Feltgen)

Randomized, controlled Phase 3 study of AAV5-hRKp.RPGR for the treatment of X-linked retinitis pigmentosa associated with RPGR gene variants

+ Long-term gene therapy follow-up study for the treatment of patients with retinitis pigmentosa (RPGR)
(Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of Xlinked Retinitis Pigmentosa Associated with Variants in the RPGR gene)

Status: Active

Recruitment: Completed

PI: PD Prof. Nicolas Feltgen

Sponsor: Janssen Research and Development, LLC

Further information:

https://kofam.ch/de/studienportal/nach-klinischen-versuchen-suchen/233955/studie/62660
https://kofam.ch/de/studienportal/nach-klinischen-versuchen-suchen/233955/studie/62666

Background:

Retinitis pigmentosa (RP) describes a group of hereditary retinal diseases that lead to progressive death of the light-sensitive cells of the retina (photoreceptors), resulting in significant visual impairment and eventual blindness. The disease usually occurs in childhood or early adulthood and begins with night blindness, followed by a gradual narrowing of the peripheral visual field and loss of central visual acuity. X-linked retinitis pigmentosa (XLRP) is a form of RP caused by damage to the RPGR gene.
The aim of this study is to investigate whether gene therapy, which is injected into the eyes during an operation, improves vision.
Gene therapy involves injecting a healthy gene to replace a damaged gene. In this study, the gene therapy to be tested is a "healthy" version of the RPGR gene.

Study design:

Approximately 90 adult subjects and up to 6 children and adolescents worldwide will participate in this study.
There are 2 closely linked studies: Initially, patients will participate in the first study (MGT-RPGR-021) for up to 18 months. Once this is completed, they will participate in the second study (MGT-RPGR-022), which has a follow-up period of 4 years. The duration of participation depends on which treatment group the patient is assigned to in the study. It is necessary to have a group of untreated participants in order to compare benefits and risks. Therefore, some participants will receive their treatment with a delay of 12 months.

Study duration: 4-5 years

N

NAC Attack (PI PD Dr. Kristina Pfau)

Study on the efficacy and safety of oral N-acetylcysteine in patients with retinitis pigmentosa
(A Phase III, Multicenter, Randomized, Parallel, Double Masked, Placebo-Controlled Study Evaluating the Efficacy and Safety of Oral N-Acetylcysteine in Patients with Retinitis Pigmentosa)

Status: Active

Recruitment: May 2024

PI: PD Dr. Kristina Pfau

Sponsor: Johns Hopkins University

Further information: https: //clinicaltrials.gov

Background:

Retinitis Pigmentosa (RP) is an inherited retinal disease that leads to visual field loss, often progressing to tunnel vision and blindness. It is caused by a large number of mutations that lead to the death of the rod photoreceptors, the cells in the retina that are responsible for seeing in dim light. Then the cone photoreceptors (cells in the retina responsible for vision in normal or bright light needed for driving or reading) begin to slowly die, leading to a gradual loss of visual field and central vision.
A previous study suggests that the cone cells may benefit from treatment with NAC. The aim of this study is to determine whether long-term treatment with NAC could slow the death of the cones, thereby slowing the loss of the visual field and preserving central vision. It also aims to determine whether long-term use of NAC has any side effects.

Study design:

A total of 438 adult participants with RP will participate in this study. The study will be conducted at multiple sites in North and South America and Europe.
Whether a patient can participate in the study will be decided based on the results of the screening visit. Pregnancy/breastfeeding is an exclusion criterion.
The study participants are randomly assigned to one of 2 groups in a 2:1 ratio: NAC or placebo.
Study visits include clinic visits, televisits with the investigator and telephone calls with the study coordinator.

Study duration: 45 months