Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Verified
Rome, Italy
Not recruitingOptic gliomaRare disease
Study on the Safety and Effectiveness of Nerve Growth Factor CHF6467 for Children and Young Adults with Optic Pathway Glioma
Verified siteInvestigational
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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 the effects of a new treatment for a condition known as Optic Pathway Glioma (OPG). This is a type of brain tumor that affects the optic nerves, which are responsible for vision. The treatment being tested is an eye drop formulation containing a substance called Nerve Growth Factor, Recombinant (R100E), also known by its code name CHF6467. The study aims to evaluate the safety and effectiveness of these eye drops in improving the visual function of children and young adults who have this condition.
The purpose of the study is to assess how well the CHF6467 eye drops work in treating visual problems caused by Optic Pathway Glioma, whether or not the condition is associated with a genetic disorder called type 1 neurofibromatosis. Participants in the study will receive multiple doses of the eye drops over a period of time. Some participants will receive the actual medication, while others will receive a placebo, which looks like the medication but does not contain the active ingredient.
The study will follow participants to monitor any changes in their vision and to ensure the treatment is safe. The trial is designed to provide valuable information about the potential benefits of using CHF6467 eye drops for individuals with Optic Pathway Glioma. This research could lead to new treatment options for those affected by this condition, improving their quality of life and visual health.
The research process
The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.
Who can join the trial?
4 criteria
- Participants must be between the ages of 3 and 40 years old.
- Participants must have a diagnosis of **Optic Pathway Glioma (OPG)**, which is a type of brain tumor that affects the nerves related to vision.
- Participants can have **type 1 neurofibromatosis (NF-1)**, a genetic condition that can cause tumors to grow on nerves, but it is not required.
- The disease must be stable, meaning it hasn't changed, as shown by two brain MRI scans done at least 6 months before joining the study.
Who cannot join the trial?
5 criteria
- Patients who are not diagnosed with Optic Pathway Glioma (OPG) cannot participate. OPG is a type of brain tumor that affects the optic nerve, which is responsible for vision.
- Patients who are not within the specified age range for the study cannot participate. The study is for children or young adults.
- Patients who are not able to use the study medication, which is a type of eye drop, cannot participate.
- Patients who have other medical conditions that might interfere with the study cannot participate.
- Patients who are unable to follow the study procedures or attend the required visits cannot participate.
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Investigated drugs
PAINLESS Nerve Growth Factor CHF6467: This is an investigational eye drop treatment being studied for its potential to improve visual function in children or young adults who have optic pathway gliomas. These are tumors that affect the nerves responsible for vision. The study aims to determine if these eye drops are safe to use and if they can effectively help in managing the condition.
What is already known about the treatment
CHF6467 – This medication is administered as eye drops and is currently being studied in clinical trials for its safety and effectiveness in treating optic pathway glioma, a type of brain tumor affecting the optic nerves. The main therapeutic indication is to improve visual function in children and young adults with this condition. CHF6467 works by utilizing a form of nerve growth factor to potentially support nerve health and function. It is classified pharmacologically as a nerve growth factor therapy.
Investigated diseases
Optic Pathway Glioma – Optic pathway glioma is a type of brain tumor that affects the optic nerves, which are responsible for vision. It is most commonly found in children and can be associated with a genetic condition known as neurofibromatosis type 1. The tumor can cause vision problems, including loss of vision, due to its location along the visual pathways. As the tumor grows, it may lead to further visual impairment and other neurological symptoms. The progression of the disease can vary, with some tumors growing slowly and others more rapidly. Regular monitoring is essential to understand how the disease is affecting the individual.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase ITrial ID2024-515753-16-00Protocol codeCHF6467-OPGEstimated enrolment36 patientsSponsorFondazione Policlinico Universitario Agostino Gemelli IRCCS
sourced from the EU Clinical Trials Register and site verification
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