RecruitingRare disease

Study on the Safety and Effects of ISTEM-01 and Mycophenolate Mofetil for Patients with Retinitis Pigmentosa Due to a Genetic Mutation

Registered drugNo placebo

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What is this trial about?

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

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. The study is testing a new treatment called ISTEM-01, which is a medicated patch containing cells derived from human embryonic stem cells. These cells are known as Retinal Pigment Epithelium cells, and they are implanted into one eye of the patient. The purpose of the study is to assess the safety and tolerability of this treatment over a period of 56 weeks.

Participants in the study will receive the ISTEM-01 treatment and will be monitored for any side effects or changes in their eye health. The study will involve regular eye exams and tests to check the placement of the therapeutic patch and to observe any changes in the eye's structure and function. These tests include Ocular Coherence Tomography (OCT) scans, which provide detailed images of the eye, and other assessments to measure visual acuity and eye pressure.

Throughout the study, researchers will also evaluate the functionality of the retina and the Retinal Pigment Epithelium cells using various methods. The goal is to determine if the treatment can help preserve or improve vision in patients with retinitis pigmentosa. The study will continue to follow participants for long-term effects and outcomes beyond the initial 56 weeks.

The research process

The trial runs in 5 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 genetic test to verify a diagnosis of retinitis pigmentosa due to a monogenic mutation affecting specific genes.
Eligibility criteria include being between 18 and 65 years old and having a negative test for SARS-CoV-2 during the pandemic period.
Step 2
Baseline evaluation
A comprehensive baseline evaluation is conducted. This includes ophthalmologic exams, laboratory tests, and physical examinations to establish initial health status.
Baseline measurements include visual acuity, visual field, and retinal imaging using spectral domain optical coherence tomography (OCT).
Step 3
Medication administration
The trial involves the administration of two medications: mycophenolate mofetil taken orally and ISTEM-01 administered as a sub-retinal medicated patch.
The primary objective is to assess the safety and tolerability of the ISTEM-01 patch over a period of 56 weeks.
Step 4
Follow-up visits
Regular follow-up visits are scheduled to monitor safety and efficacy. These visits include ophthalmologic exams, laboratory tests, and assessments of vital signs.
Key evaluations include changes in visual acuity, intraocular pressure, and retinal imaging at specified intervals: baseline, weeks 4, 8, 12, 24, 36, 48, and 56.
Step 5
Long-term follow-up
After the initial 56-week period, long-term follow-up continues annually to monitor the placement and position of the therapeutic patch and changes in retinal and RPE functionality.
Additional assessments include fundus fluorescein angiography, B-mode orbital ultrasound, and electroretinography (ERG) to evaluate retinal health and function.

Who can join the trial?

4 criteria

The patient must have a documented diagnosis of **retinitis pigmentosa**, which is a condition affecting the eyes. This diagnosis should be confirmed by a genetic test showing a specific type of mutation called a **monogenic mutation**. This mutation should affect a gene involved in the process of seeing, specifically at the level of **RPEs** (retinal pigment epithelium), such as **RPE65**, **LRAT**, or **MerTK**.
The patient must be between **18 and 65 years old**.
During the **SARS-CoV-2 pandemic**, the patient must test negative for the virus using a **PCR test** or any other new validated test. **PCR** stands for polymerase chain reaction, a method used to detect the virus.
For patients in the first group, their **visual acuity** (sharpness of vision) must be **20/200 or worse** in the best eye, which is considered legally blind. For patients in the second group, their visual acuity must be **better than 20/800 but worse than 20/63** in the worst eye. Additionally, their **visual field** (the area they can see) must be more than **10 degrees** in the center when tested with a **V4 test** in the worst eye. They must also have a visible **photoreceptor outer nuclear layer (ONL)** on a special eye scan called **spectral domain optical coherence tomography (OCT)**.

Who cannot join the trial?

8 criteria

Patients who do not have retinitis pigmentosa caused by a single gene mutation cannot participate. Retinitis pigmentosa is a condition that affects the eyes and can lead to vision loss.
Individuals who are not within the specified age range for the study are excluded. The age range is not specified here, but it is important for participants to be within the required age group.
People who have other serious health conditions that might interfere with the study or its results are not eligible.
Participants who are unable to follow the study procedures or attend the required visits are excluded.
Individuals who are pregnant or breastfeeding cannot take part in the study.
Anyone who has participated in another clinical trial recently may not be eligible, as it could affect the study's outcomes.
Patients who have allergies or reactions to the study medication or its components are excluded.
Individuals with a history of drug or alcohol abuse that might interfere with the study are not eligible.

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Investigated drugs

ISTEM-01 is a treatment being studied for its safety and tolerability in patients with retinitis pigmentosa. This therapy involves the implantation of retinal pigment epithelium (RPE) cells that are derived from human embryonic stem cells. The goal is to see if these cells can help improve or stabilize vision in patients who have a specific genetic mutation causing their condition. The study focuses on observing the effects of this treatment over a period of 56 weeks after the cells are implanted into one eye of the patient.

What is already known about the treatment

ISTEM-01 – This medication is administered through implantation into one eye and is currently being studied in clinical trials. It is in the early stages of research, specifically a phase 1/2 trial, focusing on its safety and tolerability. The main therapeutic indication for ISTEM-01 is for patients with retinitis pigmentosa caused by a monogenic mutation. The medication works by using human embryonic stem cell-derived retinal pigment epithelium (RPE) cells to potentially restore or improve retinal function. It falls under the pharmacological classification of cell-based therapies.

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 is caused by mutations in specific genes that are responsible for the health and function of the photoreceptor cells in the retina. The disease typically begins with night blindness, followed by a loss of peripheral vision, and eventually can lead to central vision loss. The progression of vision loss varies among individuals, with some experiencing a slow decline over many years, while others may have a more rapid progression. The condition is often diagnosed in childhood or early adulthood, and it is considered a rare disease.