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.
Em resumo
Sobre o que é este estudo?
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.
Pontos principais
- 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.
Quem pode ser elegível?
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.
Este estudo pode ser adequado para você?
Responda a estas perguntas rápidas para ver se você pode ser elegível. Este é apenas um guia — a decisão final é da equipe de pesquisa.
- Is your child under 18 years old?
- Has your child been diagnosed with retinoblastoma only in the eye, not spread elsewhere?
- Does their retinoblastoma include 'vitreous seeds'?
- Is your child generally well enough, with healthy blood counts and kidney function?
- Are you able to commit to the required hospital visits and treatments?
O que envolve a participação?
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.
Riscos e benefícios potenciais
Locations (20)
- Hospital Infantil do AlabamaCódigo postal verificadoBirmingham, Estados Unidos· Recrutamento
- Hospital Infantil de Los AngelesCódigo postal verificadoLos Angeles, Estados Unidos· Recrutamento
- Lucile Packard Children's Hospital Stanford UniversityCódigo postal verificadoPalo Alto, Estados Unidos· Recrutamento
- Children's Hospital ColoradoCódigo postal verificadoAurora, Estados Unidos· Recrutamento
- Children's Healthcare of Atlanta - Arthur M Blank HospitalCódigo postal verificadoAtlanta, Estados Unidos· Recrutamento
- C S Mott Children's HospitalCódigo postal verificadoAnn Arbor, Estados Unidos· Recrutamento
- Faculdade de Medicina da Universidade de WashingtonCódigo postal verificadoSt Louis, Estados Unidos· Recrutamento
- Duke University Medical CenterCódigo postal verificadoDurham, Estados Unidos· Recrutamento
- Children's Hospital Medical Center of AkronCódigo postal verificadoAkron, United States· Recrutamento
- Fundação Cleveland ClinicCódigo postal verificadoCleveland, Estados Unidos· Recrutamento
- Saint Jude Children's Research HospitalCódigo postal verificadoMemphis, United States· Recrutamento
- Dell Children's Medical Center of Central TexasCódigo postal verificadoAustin, Estados Unidos· Recrutamento
Perguntas frequentes
O que é 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.
Como saber mais
Sempre consulte seu médico ou especialista antes de decidir participar de um estudo.
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