In short
A clinical trial is underway to assess the safety and effectiveness of a new eye drop treatment containing Nerve Growth Factor (NGF) for children and young adults with Optic Pathway Glioma (OPG). This study aims to explore how multiple doses of this innovative medication, known as CHF6467, may improve visual function in patients with this rare condition.
At a glance
- Study Title
- Safety and Efficacy of multiple doses of the PAINLESS Nerve Growth Factor CHF6467 in paediatric subjects with Optic Pathway Glioma (OPG)
- Medical Condition
- Optic Pathway Glioma (OPG)
- Drug Name
- Nerve Growth Factor, Recombinant (R100E)
- Drug Form
- Eye Drops
- Age Range
- 3 to 40 years old
- Primary Endpoint
- Largest radius of the field of view measured by kinetic perimetry
- Key Inclusion Criteria
- OPG-induced visual damage, stable disease for at least 6 months
- Key Exclusion Criteria
- Concomitant eye disorders, recent cancer treatments
What is NERVE GROWTH FACTOR, RECOMBINANT (R100E)?
NERVE GROWTH FACTOR, RECOMBINANT (R100E) is a specially engineered protein that mimics a natural substance in our body called nerve growth factor. This medication, also known by its code name CHF6467, is being studied as a potential treatment for certain eye conditions. It's important to note that this is a recombinant biological/biotechnological drug, which means it's created using advanced laboratory techniques to produce a protein similar to one found in nature.
What Medical Condition Does It Treat?
The primary focus of the current research is on treating a condition called Optic Pathway Glioma (OPG). OPG is a type of brain tumor that affects the optic nerves, which are responsible for carrying visual information from the eyes to the brain. This condition can cause vision problems and is often found in children and young adults. Some people with OPG also have a genetic condition called type 1 neurofibromatosis (NF-1), but not all OPG patients have NF-1.
Current Clinical Trial
A clinical trial is currently underway to study the effects of NERVE GROWTH FACTOR, RECOMBINANT (R100E) on patients with Optic Pathway Glioma. This trial is specifically looking at how safe and effective this treatment is for improving visual function in people with OPG.
How Is It Administered?
The medication is being tested in the form of eye drops. This means that if proven effective, patients would be able to apply the medicine directly to their eyes, making it a potentially convenient and non-invasive treatment option.
Who Is Eligible for the Trial?
The trial has specific criteria for who can participate:
- Age range: Participants must be between 3 and 40 years old. This wide age range allows the researchers to study the effects on both children and young adults.
- Diagnosis: Participants must have been diagnosed with visual damage caused by Optic Pathway Glioma. They may or may not also have type 1 neurofibromatosis.
- Stable condition: The OPG must be stable, as confirmed by two brain MRI scans performed at least 6 months before the screening for the trial.
There are also some conditions that would prevent someone from participating in the trial:
- Having any other eye disorders that could interfere with the evaluation of the treatment's effectiveness.
- Having received radiotherapy, chemotherapy, or any other specific cancer treatment within 9 months before entering the trial.
What Is the Trial Trying to Achieve?
The main goal of this randomized clinical trial is to assess two key aspects of the NERVE GROWTH FACTOR, RECOMBINANT (R100E) eye drops:
- Safety: The researchers want to ensure that the medication doesn't cause harmful side effects when used multiple times.
- Efficacy: They aim to determine if the eye drops can improve visual function in patients with Optic Pathway Glioma.
How Will Success Be Measured?
The primary way the researchers will measure the success of the treatment is by looking at changes in the patient's field of view. Specifically, they will use a test called kinetic perimetry according to Goldmann with the aim V/4e. This test measures the largest radius of the field of view in degrees of visual angle. In simpler terms, they're checking how wide an area the patient can see without moving their eyes.
This measurement is important because Optic Pathway Gliomas often affect peripheral vision (the edges of what we can see), so improvements in the field of view could indicate that the treatment is helping to preserve or restore vision affected by the tumor.