Not recruitingRare disease

Study of HORA-PDE6B's Safety in Retinitis Pigmentosa Patients with PDE6B Gene Mutations

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What is this trial about?

A plain-language summary of the goals, design and what participants do

The clinical study focuses on patients with a condition called retinitis pigmentosa, which affects the retina, a part of the eye. This disease is characterized by mutations in the PDE6B gene, leading to eyesight problems. The study investigates the safety and effectiveness of treating this condition using a gene therapy method. This involves a treatment called HORA-PDE6B, which is introduced into the eye via a technique known as subretinal injection. This method aims to correct the genetic defect and improve vision.

Several other treatments are also being evaluated in the study for different conditions, but they are not the primary focus for this particular investigation. These include atropine, dexamethasone, neomycin, and indometacin, all formulated as eye drops or solutions for eye treatment. Additionally, phenylephrine hydrochloride and tropicamide are used in an ophthalmic insert form.

The main goal of the study is to determine whether the HORA-PDE6B therapy is safe for use in patients with retinitis pigmentosa due to specific genetic changes. This involves careful monitoring for any adverse effects post-treatment, alongside regular eye exams and other medical assessments to ensure the well-being of participants.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial assessment
The trial begins with an initial assessment to confirm eligibility. This includes a review of medical history and specific tests to confirm the presence of retinitis pigmentosa caused by mutations in the PDE6B gene.
Tests may include a detailed eye examination, visual acuity tests, and genetic testing to confirm the presence of the mutation.
Step 2
Pre-treatment preparation
Before the treatment, a series of eye drops are administered to prepare the eye. These include atropine sulfate and phenylephrine hydrochloride, tropicamide for ophthalmic use.
These medications help dilate the pupil and prepare the eye for the procedure.
Step 3
Treatment administration
The main treatment involves a subretinal injection of a solution containing an adenovirus associated viral vector with the human PDE6B gene.
This procedure is performed under local anesthesia and involves a short hospital stay.
Step 4
Post-treatment care
After the treatment, dexamethasone phosphate is administered as an injection to reduce inflammation.
Regular follow-up visits are scheduled to monitor the eye's response to the treatment and to assess any side effects.
Step 5
Follow-up assessments
Follow-up assessments include routine eye examinations, visual acuity tests, and other functional tests such as color vision and visual fields.
These assessments help evaluate the safety and effectiveness of the treatment over time.
Step 6
Long-term monitoring
Long-term monitoring continues for several years to assess the ongoing safety and efficacy of the treatment.
This includes periodic visits for eye examinations and additional tests as needed.

Who can join the trial?

12 criteria

Patients must have retinitis pigmentosa (RP) caused by a defect in a specific gene called PDE6B. This means they have a genetic condition affecting their vision.
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Patients should have a clinical status confirmed by:
- A history of the disease.
- Presence of typical features of retinitis pigmentosa in the eye.
- Reduced responses in a test called ERG (electroretinogram), which measures how the eye responds to light, with more impact on the rod cells, which are responsible for vision in low light.
- Specific vision requirements based on different groups in the study, such as a certain level of visual acuity or a preserved central visual field.
- Patients must have a confirmed genetic mutation in the PDE6B gene, verified by a certified laboratory.
- Adult patients must provide informed consent. Patients under 18 need to give their assent, and their parents or legal representatives must also consent after being informed about the study details.
- Patients should be at least 18 years old for certain groups in the study, and at least 13 years old for another group.
- Patients must still have some central vision that allows them to move around independently.
- Females who can have children must have a negative pregnancy test before starting the study and use effective birth control during the study and for six months after.
- Males who can father children must agree to use effective birth control for six months after receiving the study treatment.
- Patients must be part of a health security system or have national healthcare insurance if they are not from France.

Who cannot join the trial?

7 criteria

Patients who have any other eye diseases besides retinitis pigmentosa. This is a condition that affects the retina, the part of the eye that helps you see.
Patients who have had any eye surgery in the past 6 months.
Patients who are currently participating in another clinical trial.
Patients who have any serious health conditions that might affect their ability to participate safely in the study.
Patients who are pregnant or breastfeeding.
Patients who have allergies to any of the substances used in the study.
Patients who are unable to follow the study procedures or attend the required visits.

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Centre Hospitalier Universitaire De Nantes
Nantes, France

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Investigated drugs

HORA-PDE6B is a treatment being studied for its safety and effectiveness in patients with a specific type of eye condition called Retinitis Pigmentosa. This condition is caused by mutations in the PDE6B gene, which leads to problems with vision. The therapy involves administering HORA-PDE6B under the retina in one eye to see if it can help improve or stabilize vision in affected patients.

What is already known about the treatment

HORA-PDE6B – This medication is administered through a subretinal injection, specifically targeting the eye. It is currently being studied in clinical trials for its safety and effectiveness in treating retinitis pigmentosa, a genetic disorder that affects vision. The main therapeutic indication is for patients with mutations in the PDE6B gene, which leads to a defect in PDE6ß expression. At the molecular level, HORA-PDE6B works by addressing the underlying genetic defect, potentially restoring normal function to the affected cells. It is classified pharmacologically as a gene therapy product.

Investigated diseases

Retinitis Pigmentosa – Retinitis pigmentosa is a group of genetic disorders that affect the retina's ability to respond to light, leading to a gradual decline in vision. It typically begins with night blindness, followed by a loss of peripheral vision, and can eventually result in tunnel vision. The disease progresses slowly, often over many years, and can vary significantly in severity among individuals. As the condition advances, the central vision may also be affected, making it difficult to perform tasks that require detailed vision, such as reading or recognizing faces. The progression of retinitis pigmentosa can be influenced by genetic factors, and it is considered a rare disease.