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RecruitingPHASE2INTERVENTIONAL

A Study to Give Treatment Inside the Eye to Treat Retinoblastoma

This study is investigating a new treatment approach for retinoblastoma, a type of eye cancer that affects children. It focuses on cases where the cancer is considered harder to treat, especially when little cancer cells (vitreous seeds) are floating in the eye's jelly-like fluid. The trial combines existing chemotherapy medicines given through the body (like carboplatin, vincristine, and etoposide) with a new method: injecting an additional medicine called melphalan directly into the eye. Researchers want to find out if adding melphalan directly into the eye is safe, what side effects it might have, and if it helps stop the cancer from growing or coming back. The aim is to improve treatment for this specific type of retinoblastoma.

At a glance

Status
Recruiting
Phase
PHASE2
Sponsor
Children's Oncology Group
Enrolment target
26
Start
04 Nov 2022
Estimated completion
31 Mar 2028

What is this study about?

This study is looking into a new way to treat retinoblastoma, which is a type of cancer that develops in the back of the eye, often affecting young children. The retina is like the film in a camera, sensing light and sending messages to the brain, so it's a very important part of the eye.

Sometimes, retinoblastoma can be more challenging to treat, especially when tiny cancer cells, called 'vitreous seeds', are floating in the jelly-like fluid inside the eye. These seeds can make the cancer harder to get rid of completely. Current treatments often involve chemotherapy, medicines given through the body to kill cancer cells. This study is exploring if adding a specific medicine called melphalan, given directly into the eye through an injection, can make these treatments even better, particularly for those tricky vitreous seeds.

The main goal is to check if this combination of standard body-wide chemotherapy and direct eye injections of melphalan is safe for children and what side effects it might cause. They also want to see how well it works in stopping the cancer. Researchers hope that by targeting the cancer directly in the eye, they can reduce the risk of the cancer not responding to treatment or coming back later, ultimately helping children keep their eyesight and live healthy lives.

Key takeaways

  • This study explores a new way to treat retinoblastoma, a type of childhood eye cancer.
  • It combines standard chemotherapy with a direct injection of medicine (melphalan) into the eye.
  • The focus is on retinoblastoma with 'vitreous seeds', which can be harder to treat.
  • The main goals are to check the safety, side effects, and effectiveness of this new treatment.
  • This is a Phase 2 study, meaning it's assessing safety and early effectiveness.

Who may be eligible?

This study is for children under 18 years old who have been newly diagnosed with retinoblastoma that is only in the eye (not spread elsewhere). Your child's doctor would need to confirm that their specific type of retinoblastoma fits the study criteria, usually meaning it's a more advanced type within the eye, often with those 'vitreous seeds' present.

Your child also needs to be generally well enough to participate, with certain blood counts (like those that fight infection and help blood clot) and kidney function within a healthy range. These checks help ensure they can safely receive the treatments. If your child has other serious health problems, they might not be able to join.

It's important to know that the study is not for children whose cancer has spread outside the eye. The doctors will review all your child's medical information carefully to see if this study is a good match for them.

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.

  1. Is your child under 18 years old?
  2. Has your child been diagnosed with retinoblastoma only in the eye, not spread elsewhere?
  3. Does their retinoblastoma include 'vitreous seeds'?
  4. Is your child generally well enough, with healthy blood counts and kidney function?
  5. Are you able to commit to the required hospital visits and treatments?
Answer every question to see your result.

What does participation involve?

If your child participates, they will receive a combination of standard chemotherapy medicines given through a drip into their vein. These will be given over two days in cycles, with breaks in between. Additionally, a medicine called melphalan will be carefully injected directly into the affected eye. This injection happens during planned procedures where your child will be under anaesthesia, so they won't feel anything.

Throughout the study, your child will have regular check-ups, which include detailed examinations of their eyes while they are under anaesthesia, along with ultrasound scans and MRI scans to monitor the cancer and how they are responding to treatment. Blood samples will also be taken to check their health and how the medicines are affecting them. The total duration of treatment and follow-up will vary depending on how your child responds, but a treatment cycle typically lasts around two weeks initially.

Potential risks and benefits

Participating in this study might offer the potential benefit of a new, possibly more effective, treatment approach for retinoblastoma, especially for more challenging types with vitreous seeds. However, all medical treatments carry risks. Your child might experience side effects from the chemotherapy medicines, such as being tired, feeling sick, or having a higher risk of infections. The eye injections also carry specific risks, such as eye irritation, or, rarely, damage to the eye. The doctors will monitor your child closely for any side effects and provide supportive care. You have the right to withdraw your child from the study at any time, for any reason, without affecting their future medical care.

Locations (20)

  • Children's Hospital of Alabama
    Verified postcode
    Birmingham, United States· Recruiting
  • Children's Hospital Los Angeles
    Verified postcode
    Los Angeles, United States· Recruiting
  • Lucile Packard Children's Hospital Stanford University
    Verified postcode
    Palo Alto, United States· Recruiting
  • Children's Hospital Colorado
    Verified postcode
    Aurora, United States· Recruiting
  • Children's Healthcare of Atlanta - Arthur M Blank Hospital
    Verified postcode
    Atlanta, United States· Recruiting
  • C S Mott Children's Hospital
    Verified postcode
    Ann Arbor, United States· Recruiting
  • Washington University School of Medicine
    Verified postcode
    St Louis, United States· Recruiting
  • Duke University Medical Center
    Verified postcode
    Durham, United States· Recruiting
  • Children's Hospital Medical Center of Akron
    Verified postcode
    Akron, United States· Recruiting
  • Cleveland Clinic Foundation
    Verified postcode
    Cleveland, United States· Recruiting
  • Saint Jude Children's Research Hospital
    Verified postcode
    Memphis, United States· Recruiting
  • Dell Children's Medical Center of Central Texas
    Verified postcode
    Austin, United States· Recruiting

Common questions

What is retinoblastoma?

Retinoblastoma is a rare type of cancer that starts in the retina, the light-sensing part at the back of a child's eye.

What are 'vitreous seeds'?

These are tiny clusters of cancer cells that break away from the main tumour and float in the jelly-like fluid inside the eye, making the cancer harder to treat.

Why inject medicine directly into the eye?

Injecting medicine directly into the eye helps target the cancer cells more precisely, especially the vitreous seeds, potentially making the treatment more effective where it's needed most.

What is 'standard chemotherapy'?

Standard chemotherapy involves medicines given through a drip into a vein that travel through the body to kill cancer cells wherever they are.

How often will my child need hospital visits?

Your child will need regular hospital visits for their chemotherapy cycles, eye examinations, and scans. The exact schedule will be explained by the study team.

How to find out more

Always speak to your GP or specialist before deciding to take part in a study.

Interested in taking part?

Register your interest

Share your details and the research team for "A Study to Give Treatment Inside the Eye to Treat Retinoblas…" will contact you if you may be eligible. Always speak to your GP before agreeing to take part.

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