Condition

Retinoblastoma clinical trials and research

• 15Clinical trials
• 13NIH research projects
• 11Countries

Newest Retinoblastoma clinical trials

All recruiting trials (15)

• High-dose Topotecan treatment for recurrent retinoblastoma with vitreous seeds

  This study is testing whether high-dose Topotecan can help patients with recurrent retinoblastoma and vitreous seeds keep their eye while fighting the tumor.

  Recruiting Early Phase 1 Eye & ENT Hospital of Fudan University · Shanghai, China · NCT06972602

• Linking RB1 genetic changes to eye tumors and metastasis

  We will use targeted long-read genetic sequencing on people with retinoblastoma to see if specific RB1 mutations match tumor features and the risk of spread.

  Recruiting University of Washington · Seattle, Washington · NCT06725173

• Treatment for advanced retinoblastoma using a chemoplaque

  This study is testing a new treatment using a special chemotherapy patch for young children with advanced retinoblastoma to see if it helps fight the cancer while keeping their vision safe.

  Recruiting at 2 sites Phase 2 Targeted Therapy Technologies, LLC · New York, New York and 1 other locations · NCT06679634

• New treatment for children with hard-to-treat retinoblastoma

  This study is testing a new drug for children with hard-to-treat eye cancer to see if it works better and has fewer side effects than standard chemotherapy.

  Recruiting Early Phase 1 Eye & ENT Hospital of Fudan University · Shanghai · NCT06424301

• Understanding the incidence and outcomes of trilateral retinoblastoma

  This study looks at how common trilateral retinoblastoma is and how well patients survive, comparing data from different countries to see if there are differences based on income levels.

  Recruiting Amsterdam UMC, location VUmc · Amsterdam, North Holland · NCT06367530

• Chemotherapy for high-risk retinoblastoma after eye removal

  This study is testing if giving chemotherapy after removing the eye can help prevent high-risk retinoblastoma from coming back in children with Stage I disease.

  Recruiting Phase 3 Sun Yat-sen University · Guangzhou, Guangdong · NCT05080010

• Collecting and analyzing eye fluid from children with retinoblastoma

  This study is trying to see if collecting eye fluid from children with retinoblastoma can help find important markers for better treatment and understanding of the disease.

  Recruiting at 9 sites Children's Hospital Los Angeles · Phoenix, Arizona and 8 other locations · NCT04959097

• Using topotecan and melphalan to treat retinoblastoma

  This study is testing if giving the chemotherapy drugs topotecan and melphalan directly into the eye can effectively treat retinoblastoma while saving the eye from removal.

  Recruiting Phase 3 Sun Yat-sen University · Guangzhou, Guangdong · NCT04799002

• New treatments for retinoblastoma to improve vision outcomes

  This study is testing new treatment options for children with retinoblastoma to see if they can improve vision and control the disease better than current methods.

  Recruiting at 28 sites Phase 2/3 Institut Curie · Saint-Denis, La Réunion and 27 other locations · NCT04681417

• Improving treatment recommendations for children with retinoblastoma in Africa

  This study is trying to see if better treatment recommendations and early diagnosis can help improve survival rates for children with retinoblastoma in Africa.

  Recruiting at 7 sites French Africa Pediatric Oncology Group · Ouagadougou and 6 other locations · NCT04425434

• Evaluating health outcomes in retinoblastoma patients after treatment

  This study looks at the health and vision outcomes of people who have been treated for retinoblastoma to see how they are doing over time.

  Recruiting at 11 sites Vanderbilt-Ingram Cancer Center · Chicago, Illinois and 10 other locations · NCT03932786

• Examining the impact of anesthesia on brain development in children with retinoblastoma

  This study is trying to see if having anesthesia for retinoblastoma treatment affects how well children think and develop compared to those who haven't had anesthesia.

  Recruiting Memorial Sloan Kettering Cancer Center · New York, New York · NCT03546387

• Using MRI to predict chemotherapy response in children with retinoblastoma

  This study is testing whether special MRI scans can help doctors predict how well children with retinoblastoma will respond to chemotherapy.

  Recruiting Fondation Ophtalmologique Adolphe de Rothschild · Paris · NCT03267459

• Investigating genetic factors in childhood cancer

  This study is trying to find out if certain genes passed down in families can increase the risk of childhood cancer by looking at samples from kids who have had cancer and their relatives.

  Recruiting St. Jude Children's Research Hospital · Memphis, Tennessee · NCT03050268

• Collecting and storing samples from children and adults with common childhood cancers

  This study is collecting and storing samples from children and adults with common childhood cancers to help researchers learn more about these diseases and find better treatments.

  Recruiting National Institutes of Health Clinical Center (CC) · Washington D.C., District of Columbia and 4 other locations · NCT01109394

NIH-funded research (13)

Early-stage science funded by the US National Institutes of Health. Most of these projects don't enroll patients, but they show what is being worked on.

• Understanding how premalignant retinoblastoma lesions form and progress in children

  Researchers are studying how early retinal lesions in kids with RB1 loss become malignant, with the goal of finding ways to prevent retinoblastoma progression.

  NIH research Children's Hospital of Los Angeles · Los Angeles, United States · NIH-11385023

• Aqueous humor liquid biopsy to guide prognosis and monitoring in children with retinoblastoma

  A liquid test using a small amount of eye fluid could reveal important tumor details to help predict how retinoblastoma in young children will behave and how well treatment is working.

  NIH research Children's Hospital of Los Angeles · Los Angeles, United States · NIH-11384505

• Parent-of-Origin Effects in Retinoblastoma: Epigenetic Drivers of Tumor Risk and Progression

  A study using long-read DNA sequencing to understand how the parent of origin of RB1 gene variants affects retinoblastoma risk and progression, with potential to guide personalized monitoring and treatment.

  NIH research University of Washington · Seattle, United States · NIH-11337130

• Retinoblastoma eye-sparing treatment for young children

  A new, targeted eye treatment for young children with retinoblastoma aims to save eyes and reduce radiation exposure by delivering medicine directly into the eye.

  NIH research Targeted Therapy Technologies, LLC · Somerset, United States · NIH-11193975

• How early retinal changes lead to retinoblastoma

  This project looks at how early abnormal cells in the retina form and later turn into retinoblastoma in children, using lab-grown retinal tissue.

  NIH research Children's Hospital of Los Angeles · Los Angeles, United States · NIH-11170656

• A tiny eye-fluid test to predict and track retinoblastoma in young children

  This project uses a very small sample of eye fluid to look for tumor DNA that could help predict how retinoblastoma will behave and to follow treatment in children.

  NIH research Children's Hospital of Los Angeles · Los Angeles, United States · NIH-11159608

• Handheld wide-view 3D eye scanner to find tiny retinoblastoma tumors

  This project is building a new handheld 3D eye scanner intended to help find very small retinoblastoma tumors early in young children who need screening.

  NIH research Oregon Health & Science University · Portland, United States · NIH-11116888

• New treatment for retinoblastoma in young children

  This study is testing a new way to treat retinoblastoma, a rare eye cancer in babies and young kids, by using a special device to deliver a chemotherapy drug right to the eye, which could help keep the cancer away without needing more intense treatments like surgery or radiation.

  NIH research Targeted Therapy Technologies, LLC · Somerset, United States · NIH-11105727

• Using eye fluid to improve diagnosis and monitoring of childhood eye cancer.

  This study is exploring a new way to check for eye cancer in kids by looking at a special fluid in their eyes, which could help doctors find important genetic clues without needing to do risky surgeries.

  NIH research Children's Hospital of Los Angeles · Los Angeles, United States · NIH-10889264

• Creating a 3D model to study childhood eye cancer

  This study is working on a new way to grow tiny eye models to learn more about retinoblastoma, the most common eye cancer in kids, so that we can find better and safer treatments for young patients.

  NIH research University of Miami School of Medicine · Coral Gables, United States · NIH-10857171

• Understanding how retinoblastoma, a childhood eye cancer, develops using 3D models of retinal cells.

  This study is looking at how retinoblastoma, a rare eye cancer in kids, starts and grows by using special lab-created eye tissues from children with genetic changes, which could help find better ways to treat the disease.

  NIH research St. Jude Children's Research Hospital · Memphis, United States · NIH-10834132

• Understanding how the retinoblastoma protein is controlled in cancer cells

  This study is looking at how a protein that helps prevent cancer, called Rb, is controlled in breast cancer, especially for patients who are being treated with specific medications, and it hopes to find better ways to make these treatments work more effectively for those with hormone-receptor positive, HER2-negative me

  NIH research Univ of North Carolina Chapel Hill · Chapel Hill, United States · NIH-10825894

• Improving early detection of retinoblastoma in children using advanced imaging technology

  This study is testing a new handheld device that takes detailed pictures of the eye to help doctors find early signs of retinoblastoma, a type of eye cancer in kids, so they can catch it sooner and provide better care.

  NIH research Oregon Health & Science University · Portland, United States · NIH-10789750

Related topics

Quick answers

How many Retinoblastoma clinical trials are recruiting right now?

15, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 11 countries. The most sites are in United States (7 trials), China (4 trials), Canada (2 trials), France (2 trials), Netherlands (1 trial). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 3: 2, Early Phase 1: 2, Phase 2/3: 1, Phase 2: 1. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

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