Active not recruitingPHASE1, PHASE2INTERVENTIONAL

Interventional Study of Implantation of hESC-derived RPE in Patients With RP Due to Monogenic Mutation

This study is investigating a new treatment for retinitis pigmentosa (RP), an inherited eye disease that causes vision loss. Researchers are testing a special type of cell therapy, using human embryonic stem cell-derived retinal pigment epithelium (RPE) cells, implanted into one eye. The main goal is to check if these cells are safe and tolerated by the body. They also want to see if the treatment might help preserve or improve vision. This early-stage study will involve 12 patients in total, with different levels of vision loss, and will follow them closely for nearly a year, with extra long-term follow-up.

At a glance

Status

Active not recruiting

Phase

PHASE1, PHASE2

Sponsor

Centre d'Etude des Cellules Souches

Enrolment target

Start

19 Aug 2019

Estimated completion

15 Dec 2026

Retinitis Pigmentosa

What is this study about?

This study is exploring a new way to treat retinitis pigmentosa (RP), an inherited eye condition that gradually causes people to lose their sight. RP happens when certain cells in the back of your eye, called retinal pigment epithelium (RPE) cells, don't work properly due to a faulty gene. These RPE cells are vital for the health of the light-sensing cells (photoreceptors) in your eye, and when they fail, vision begins to decline.

The treatment being tested involves implanting healthy RPE cells, grown from human embryonic stem cells, into one eye. The hope is that these new, working cells will support the existing light-sensing cells, potentially slowing down or even improving vision loss. This is an early-stage study (Phase 1/2), which means the main focus is to make sure the treatment is safe and doesn't cause serious side effects. They will also be gently looking for any signs that the treatment might be working to help vision.

Researchers will carefully watch how the body reacts to the new cells and whether they settle into the correct place in the eye. They'll also perform various eye tests to see if there are any changes in vision or the health of the back of the eye. This study is an important step in developing new treatments for RP, aiming to give people with this condition hope for preserving their sight.

Key takeaways

Tests a new cell therapy for specific types of retinitis pigmentosa.
Aims to check the safety and early effectiveness of implanted RPE cells.
Involves a single operation on one eye, followed by close monitoring.
Participation requires regular visits to a hospital in Paris for up to 2 years, plus long-term follow-up.
This is an early-stage study, so the main focus is on safety.
Eligibility depends on your age, specific genetic mutation, and current vision level.

Who may be eligible?

To be considered for this study, you generally need to be between 18 and 65 years old and have a confirmed diagnosis of retinitis pigmentosa (RP) caused by a specific faulty gene (RPE65, LRAT, or MerTK).

If you have very advanced vision loss (legally blind), you might be considered for the first group of patients. If your vision loss is not as advanced, you might be eligible for the second group. For this second group, doctors would also need to see that you still have certain light-sensing cells in your eye according to a special scan.

There are also some important safety requirements. For women, this includes a negative pregnancy test and a commitment to using two forms of contraception if you could become pregnant. For men, if you are sexually active, you would need to use condoms during certain periods of the study. You also cannot have certain other medical conditions, like chronic hepatitis, or have had other gene or cell therapies in the past.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you between 18 and 65 years old?
Do you have a confirmed genetic test showing RP caused by RPE65, LRAT, or MerTK gene mutations?
Are you able to travel to Paris for hospital visits?
Are you willing and able to use specific contraception methods if needed?
Do you have any serious allergies or medical conditions that might exclude you (e.g., chronic hepatitis, prior gene therapy)?
Are you comfortable with the possibility of being legally blind or having other specific vision criteria depending on the study group?

Answer every question to see your result.

What does participation involve?

If you join this study, it will involve several visits to a specialist hospital in Paris over about 106 weeks in total. Initially, there will be screening tests for about 8 weeks to check if you are suitable. If you are, you will have the treatment implanted into one eye. After the procedure, you will have regular check-ups for 56 weeks to monitor your safety and vision. This includes various eye exams to check the position of the implanted cells, how well your vision is working, and the overall health of your eye. After these 56 weeks, you will be asked to continue in a separate long-term follow-up study for another four years to keep monitoring your progress and safety over time.

Potential risks and benefits

The potential benefits of this study include the possibility that the implanted cells could help protect or improve your vision, offering hope for a condition that currently has limited treatments. However, as this is a new type of treatment, there are also potential risks, such as surgery-related complications, side effects from the medication you'd take, or your body reacting to the implanted cells. Your safety will be closely monitored throughout the study. Remember, joining a study is always your choice, and you have the right to withdraw at any time without affecting your usual medical care.

Locations (1)

•Centre Hospitalier National d'Ophtalmologie (CHNO) des Quinze-Vingts
Verified postcode
Paris, France

Common questions

What is retinitis pigmentosa?

It's an inherited eye disease that causes vision to slowly get worse over time, often inherited through family genes.

What are RPE cells?

These are special support cells at the back of your eye that help keep your light-sensing cells healthy and working properly.

What does 'Phase 1/2' mean for a study?

It means this is an early study primarily focused on checking if the treatment is safe and well-tolerated, rather than fully proving how effective it is yet.

Will my vision definitely improve?

There's no guarantee. The main goal is safety, but researchers will also explore if there are any early signs the treatment helps protect or improve vision.

How long will I be involved in the study?

The active part of the study is about 106 weeks, but you'll be asked to participate in a long-term follow-up for an additional four years after that.