Study on the Safety and Effects of CPK850 Gene Therapy for Patients with Retinitis Pigmentosa Due to RLBP1 Gene Mutations

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What is this study about?

This clinical trial is focused on studying a condition called Retinitis Pigmentosa, which is a genetic disorder that affects the eyes and can lead to vision loss. Specifically, the trial is looking at cases of Retinitis Pigmentosa caused by mutations in the RLBP1 gene. The treatment being tested is a gene therapy called CPK850, which is administered as a solution for injection directly into the eye. This therapy uses a special type of virus, known as an adeno-associated viral vector, to deliver a healthy version of the gene to the cells in the retina, which is the part of the eye that detects light.

The purpose of the study is to explore the safety and effectiveness of this new treatment. Participants in the trial will receive a single dose of CPK850, and researchers will monitor them to see how well they tolerate the treatment and whether it helps improve their vision. The study will involve regular eye examinations and tests to assess changes in vision and eye health over time. These tests will include checking visual acuity, which is how clearly one can see, and examining the retina using imaging techniques.

Throughout the study, participants will be closely observed for any side effects or changes in their overall health. The trial aims to determine if CPK850 can help restore some of the lost vision in patients with this specific genetic form of Retinitis Pigmentosa. The study will continue for several years to gather comprehensive data on the long-term effects and benefits of the treatment.

1 initial assessment

Upon joining the study, an initial assessment is conducted to ensure eligibility. This includes a review of medical history and a series of eye examinations to confirm the diagnosis of retinitis pigmentosa caused by mutations in the RLBP1 gene.

A pregnancy test is required for female participants. The study requires participants to be between 18 and 70 years old, with specific visual acuity criteria.

2 pre-treatment evaluations

Before receiving the treatment, several evaluations are performed. These include measuring best corrected visual acuity (BCVA), checking intraocular pressure (IOP), and conducting eye examinations using techniques like optical coherence tomography (OCT) and color fundus photography (CFP).

Blood tests and other systemic evaluations are conducted to ensure overall health and safety.

3 treatment administration

The treatment involves a single dose of CPK850, administered as a solution for injection directly into the subretinal space of the eye. This procedure is performed under surgical conditions.

The goal of the treatment is to explore the safety and potential benefits of the gene therapy for improving vision.

4 post-treatment monitoring

After the treatment, regular follow-up visits are scheduled to monitor the safety and effectiveness of the therapy. These visits include repeated eye examinations and assessments of visual function.

Participants are observed for any adverse events or changes in vision, with specific attention to the recovery of the rod system in the treated eye.

5 long-term follow-up

The study includes long-term follow-up visits to assess the sustained effects of the treatment. These visits occur over a period of up to one year after the treatment.

During these visits, various tests are repeated to evaluate changes in visual acuity, contrast sensitivity, and other aspects of visual function.

Who Can Join the Study?

Provide written informed consent before any assessment is performed. This means you agree to participate in the study after understanding all the details.
Be a male or female aged between 18 and 70 years. Women must have a negative pregnancy test before the study starts and just before the treatment day.
Have clear enough eyes and pupils that can be dilated to allow for clear eye photographs and certain eye procedures.
Meet the surgical requirements for the study. You must weigh at least 40 kg (about 88 pounds) and have a body mass index (BMI) of less than 40. BMI is a measure of body fat based on your weight and height.
Have a certain level of visual acuity (clarity of vision) in the study eye. This will be measured using a specific eye chart. Different levels are required for different groups in the study.
Have a clinical diagnosis of certain types of retinitis pigmentosa with specific genetic mutations, confirmed by specialized genetic testing.
Have visible layers of photoreceptor and retinal pigment epithelium in the study eye, as seen on a standard eye scan. This will be confirmed by a central reading center.
Show a certain level of dark adaptation in the study eye, which is the eye’s ability to adjust to darkness, measured before treatment.

Who Cannot Join the Study?

Patients with any other eye disease that could affect the study results.
Patients who have had eye surgery within the last 6 months.
Patients who are currently using any medication that could affect the eyes.
Patients with a history of severe allergic reactions.
Patients who are pregnant or breastfeeding.
Patients with any serious health condition that could interfere with the study.
Patients who have participated in another clinical trial within the last 30 days.
Patients who are unable to follow the study procedures or attend all study visits.

Where you can join this trial?

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Verified Sites

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Other Sites

Site Name	City	Country	Status
St Erik Eye Hospital	Solna	Sweden

Trial status

Country	Status	Recruitment Start
Sweden	Not recruiting	16.04.2018

Trial locations

Investigated drugs:

Cpk850

CPK850 is a gene therapy being tested for its safety and effectiveness in treating a specific type of eye disease called retinitis pigmentosa, which is caused by mutations in the RLBP1 gene. This therapy is administered directly into the eye, specifically under the retina, to potentially help improve vision. The goal of this treatment is to see if it can help the eye recover its ability to adapt to darkness, which is often impaired in people with this condition. The study is also looking at how safe this treatment is for both the eyes and the rest of the body by monitoring various health parameters and any side effects that may occur.

Investigated diseases:

Retinitis pigmentosa

Retinitis pigmentosa caused by biallelic mutations in the RLBP1 gene – Retinitis pigmentosa is a genetic disorder that affects the retina’s ability to respond to light, leading to a gradual loss of vision. It is characterized by the progressive degeneration of the photoreceptor cells in the retina, specifically the rods and cones. The condition begins with night blindness, as rod cells, which are responsible for vision in low light, are affected first. Over time, individuals experience a narrowing of the visual field, often described as tunnel vision. As the disease progresses, cone cells, which are responsible for color vision and central vision, may also degenerate, leading to further vision impairment. The specific form caused by biallelic mutations in the RLBP1 gene is a rare variant of retinitis pigmentosa, with symptoms and progression similar to other forms of the disease.

Trial ID:

2023-508688-54-00

Protocol code:

CCPK850X2202

NCT ID:

NCT03374657

Trial Phase:

Phase I and Phase II (Integrated) – First administration to humans

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Study on the Safety and Effects of CPK850 Gene Therapy for Patients with Retinitis Pigmentosa Due to RLBP1 Gene Mutations

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?