About the Study

***Thank* *you*** for your interest in our clinical study. Medical research depends on patients’ engagement, and we are immensely grateful for your support.

PRODYGY is a clinical study evaluating the potential of an investigational drug called SPVN06 for the treatment of retinitis pigmentosa (RP).

It is registered with the US online database of clinical trials. Details are available on the National Library of Medicine website.

clinicaltrials.gov

Goal of the study

The PRODYGY clinical study will help us learn whether the investigational drug SPVN06 is safe for patients and potentially identify preliminary signs of efficacy.

The study is also expected to inform on which dose level can be recommended for future clinical studies.

Our commitment to patients

We place patients and their safety at the center of our work. We have designed this clinical study in partnership with inherited retinal diseases’ specialists who have decades of experience working alongside patients with RP.

We are committed to maintaining close and open communications with patients and patient advocacy groups, to build trust within our community and make sure we incorporate patients’ needs into the design of our clinical studies.

Who can join the PRODYGY study?

Every clinical study defines rules for who can participate; only the clinical study team can decide whether you can enroll for the study after screening visits.

The main recruitment criteria for the study are listed here, please contact the study coordinators for an exhaustive list.

Clinical Coordinators

To participate in this study

Adult female or male, 18 years and older, with retinitis pigmentosa due to a variant (also known as a mutation) in the RHO, PDE6A, or PDE6B gene, that was confirmed by genetic testing, and

Either (i) have advanced severe retinitis pigmentosa, this means your best visual acuity is below or equal to 20/200 and above or equal to 20/800, and a visual field below or equal to 20 degrees.

or (ii) have advanced intermediate retinitis pigmentosa, this means that you will have a best visual acuity between 20/40 and 20/200, and a visual field below or equal to 20 degrees.

It is up to you to decide whether you would like to take part in this clinical study. Take time to discuss your participation with your doctor, family, and friends.

If you decide to participate, please also know that you can always leave the study, at any time and for any reason. Your decision to withdraw your participation will not affect your access to standard care. If you decide to participate in this study, there is also a chance that you may not receive or may not be eligible to receive the investigational drug, based on the study recruitment criteria.

What should you expect if you join the PRODYGY study?

Screening

Every clinical study defines rules for who can participate; they are called eligibility criteria.

The clinical study team will check whether this study is right for you during one or more screening visit(s).

The screening visits will involve tests to assess your vision as well as your general health.

Group assignments

If you are selected to join the clinical study, you will be assigned to a group which will either receive a certain dose of the investigational drug; or to a “no-treatment” control group, which will not undergo the surgical procedure and will not receive the investigational drug.

Regardless of group assignments, all patients participating in the study will be monitored the same way as other patients for the entire duration of the study. Note however that even If you decide to participate in this study, there is also a chance that you may not be eligible to receive the investigational drug, based on the study recruitment criteria.

More detail on the design and conduct of the clinical study will be provided by the clinical team and as part of informed consent.

Treatment

The treatment consists of a single administration of the investigational drug in your worse-seeing eye. The study team will determine which is your worse-seeing eye based on a visual assessment.

The investigational treatment will be administered under anesthesia during surgery. An injection will be made at the back of your eye, under the retina. This is called a subretinal injection. It is a procedure that is already commonly used for certain approved drugs, as well as other investigational treatments.

The dose level of SPVN06 you will receive will depend on which group you have been assigned to. Learn more about group assignments above.

Number of visits and length of study treatment

Your participation in this research study will involve 17 visits to the research site over a period of 5 years. The first 11 visits will occur during the first year of your participation.

You may also be asked to come to the clinical study site for additional visits at any time during the study, if the study doctor decides that extra tests and procedures are needed for your safety.

What happens at each study visit will vary, some visits may take longer than others.

You can be accompanied during study visit, and travel arrangements will be made for you and your caregiver.

Learn more about our travel plan.

Learn More

Protection of your safety

The safety of all patients taking part in our clinical study is our first priority, as taking part in a research study involves risks, and not all risks are identified for the investigational product yet.

We uphold the highest standards for our clinical study, following Good Clinical Practice standards defined by international and national health authorities.

We follow closely the safety of all patients joining the clinical study. Any undesirable experience and/or side effects associated with the use of the investigational drug are closely monitored during the study, along with your general health condition.

Appropriate measures to protect participants’ well-being are taken at all times during the study.

Risks associated with your participation in the study

It is the first time that the investigational drug is administered to patients in this study, so the exact risks that you will be taking as a participant are not known yet. The investigational drug has previously been administered and evaluated in animals and has received approval in the USA and France to be tested for the first time in patients.

A list of the potential risks associated with participation in the study will be given to you before you join the clinical study, as part of the informed consent documentation.

Protection of your privacy

We are committed to protecting your privacy and keeping your personal health information collected as part of the study strictly confidential.

All information collected is coded and recorded safely. We will use your information exclusively for the needs of this study and to analyze the study results. Your identity will always be kept private in any publication or presentation about the study.

More information about the protection of your privacy will be provided to you before joining the clinical study as part of informed consent.

Informed Consent

Clinical trial participants must provide informed consent before they are enrolled into a clinical trial.

Patients are provided with an Informed Consent Form approved by an independent Institutional Review Board (IRB) or Ethics Committee (EC) that explains the details of the trial, including its possible risks and benefits.

Learn more about gene therapy for retinal diseases

You should know as much as possible before making the decision to join a clinical trial. Below are some frequently asked questions and answers, which you may find useful.

How does vision work?

The retina is the light-sensitive layer of tissue located in the back of the eye, where vision originates. The cells of the retina that are sensitive to light are called photoreceptors. There are two types of photoreceptors: cones and rods.

The rods are located mainly on the edges of the retina, they are responsible for vision in dim light and peripheral vision (i.e., what you see off to the side of your vision). The cones are concentrated in the center of the retina, they are responsible for color vision, daylight vision, and detailed vision allowing us to read and recognize faces.

In rod-cone dystrophies like retinitis pigmentosa, rods gradually stop functioning properly. This starts first with difficulty seeing in dim-light, and slowly progresses to losing vision in the periphery, as if looking through a tunnel or a straw. Subsequently, cones also start to malfunction, causing a progressive loss of central vision and color perception.

What is gene therapy?

Gene therapy is a medical solution that modifies a person’s genes to treat or cure a disease. It consists in delivering therapeutic human genes (made of DNA) into malfunctioning cells to restore or enhance their function.

The therapeutic genes are delivered by a viral vector, a tool that helps bring genetic material into cells that is unable to replicate inside of cells, which means that it cannot cause an infectious disease. This viral vector is derived from a non-pathogenic virus (i.e., it does not cause disease). Once the therapeutic genes are transferred to cells, they are expected to remain inside the cells in theory for life.

What is a gene-independent gene therapy approach?

Traditional gene therapies for inherited retinal diseases aim to bring to the retina a functional copy of the gene that is misfunctioning and causing the disease. As such, traditional gene therapy can only be indicated for patients who carry a specific genetic variant.

So far, researchers have found genetic variants responsible for retinitis pigmentosa in more than 70 different genes, and the development of treatments specific to each gene will probably take decades and may not be possible for all.

Consequently, gene-independent approaches are currently being developed to offer treatment to most patients with RP, regardless of their genetic background.

What is genetic testing?

Genetic testing is a medical test that examines a person’s DNA – the genetic material that contains instructions for the functioning of the body – to identify changes or variations in the genes that may be associated with certain diseases. Genetic testing can increase the accuracy of medical diagnosis by providing information that would not be otherwise available.

Genetic testing is usually conducted by collecting a sample of tissue or fluid containing DNA (such as blood or saliva). The DNA is analyzed to look for looks for changes, sometimes called mutations or variants, in your DNA.

How does the investigational drug SPVN06 work?

SPVN06 is an investigational gene therapy for the treatment of RP. It aims to slow down disease progression by prolonging the survival of cone photoreceptors, regardless of the genetic variant causing your disease.

What is an investigational drug?

An investigational drug is a treatment that has not been commercially approved by the U.S. Food and Drug Administration or any regulatory authority in any country. It is being tested in humans as part of a clinical study to determine whether it is safe and/or efficacious.

What is informed consent?

Clinical trial participants must provide informed consent before they are enrolled into a clinical trial. Patients are provided with an Informed Consent Form approved by an independent Institutional Review Board (IRB) or Ethics Committee (EC) that explains the details of the trial, including its possible risks and benefits.

Whether you are considering joining the PRODYGY study, or you would just like information on the study, we hope you will find here what you are looking for.

If you didn’t find all the necessary information or have any questions, please do not hesitate to contact the study coordinators below.