Retinoblastoma
MONDO:0008380A malignant tumor that originates in the nuclear layer of the retina. As the most common primary tumor of the eye in children, retinoblastoma is still relatively uncommon, accounting for only 1% of all malignant tumors in pediatric patients. Approximately 95% of cases are diagnosed before age 5. These tumors may be multifocal, bilateral, congenital, inherited, or acquired. Seventy-five percent of retinoblastomas are unilateral; 60% occur sporadically. A predisposition to retinoblastoma has been associated with 13q14 cytogenetic abnormalities. Patients with the inherited form also appear to be at increased risk for secondary nonocular malignancies such as osteosarcoma, malignant fibrous histiocytoma, and fibrosarcoma.
Also known as: RB, retinoblastoma, retinoblastoma, malignant, RB1, Rb, eye cancer, retinoblastoma
2875 clinical trials for this condition and its sub-types, 32 tagged with Retinoblastoma itself.
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Sub-types of Retinoblastoma
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Unilateral retinoblastoma 6 trials
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Intraocular retinoblastoma 4 trials · 5 incl. sub-types
1 sub-type
- Pediatric intraocular retinoblastoma 2 trials
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Bilateral retinoblastoma 3 trials
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Extraocular retinoblastoma 2 trials
1 sub-type
- Pediatric extraocular retinoblastoma 0 trials
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Hereditary retinoblastoma 2 trials
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Non-hereditary retinoblastoma 0 trials
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Trilateral retinoblastoma 0 trials
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Engineered immune cells take on childhood cancers in first human trial
Disease control By invitation onlyThis early-stage trial is testing a new treatment called GPC2 CAR T cells for children with neuroblastoma or retinoblastoma that has come back or not responded to standard treatments. The therapy uses a patient's own immune cells, which are modified in a lab to better recognize a…
Phase 1 • Sponsor: Stephan Grupp MD PhD • Aim: Disease control
Last updated Aug 16, 2026 00:00 UTC
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Eye-Directed chemotherapy aims to save sight in children with retinoblastoma
Disease control OngoingThis phase 2 trial tests whether giving chemotherapy directly into the eye (subconjunctival carboplatin) along with systemic topotecan can shrink advanced retinoblastoma tumors in children with disease in both eyes. The goal is to preserve the eye and vision, avoiding removal. Pa…
Phase 2 • Sponsor: St. Jude Children's Research Hospital • Aim: Disease control
Last updated Jul 01, 2026 00:00 UTC
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Engineered immune cells take aim at childhood cancers in early trial
Disease control OngoingThis early-phase study tests a personalized cell therapy for children and young adults (up to age 26) with solid tumors that have come back or not responded to standard treatment. The therapy uses the patient's own immune cells, modified to recognize and attack a protein called B…
Phase 1 • Sponsor: Seattle Children's Hospital • Aim: Disease control
Last updated Jun 27, 2026 12:25 UTC
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New hope for kids with eye cancer: extra chemo may stop spread
Disease control OngoingThis study looks at whether giving chemotherapy (and sometimes radiation) after removing the eye can prevent retinoblastoma from coming back in children. About 195 children with advanced retinoblastoma in one eye took part. The goal is to see if this extra treatment reduces the c…
Phase 2 • Sponsor: Institut Curie • Aim: Disease control
Last updated Jun 27, 2026 08:09 UTC
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New chemo cocktails aim to save eyes in kids with retinoblastoma
Disease control OngoingThis phase 2 trial tests different chemotherapy regimens in 133 children with retinoblastoma, a rare eye cancer. The goal is to shrink tumors and avoid removing the eye or using radiation. Treatments include intravenous and intra-arterial chemotherapy, sometimes combined with las…
Phase 2 • Sponsor: Institut Curie • Aim: Disease control
Last updated Jun 27, 2026 07:59 UTC
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Engineered immune cells take on childhood cancers in first human test
Disease control OngoingThis early-stage trial tests a new type of immunotherapy for children and young adults with solid tumors that have not responded to standard treatments. The therapy uses the patient's own immune cells (T cells) that are genetically modified to recognize and attack cancer cells ca…
Phase 1 • Sponsor: Seattle Children's Hospital • Aim: Disease control
Last updated Jun 27, 2026 07:59 UTC
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Newborn screening study aims to catch rare diseases at birth
Diagnosis OngoingThis study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…
Sponsor: RTI International • Aim: Diagnosis
Last updated Jul 03, 2026 00:00 UTC
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Can your phone spot a white pupil? app aims to catch childhood eye cancer early
Diagnosis CancelledThis study planned to test a smartphone app called GoCheckKids as a screening tool for leukocoria (a white pupil reflex) in children from birth to 10 years old. Leukocoria can be an early sign of retinoblastoma, the most common eye cancer in children. The goal was to see if the a…
Sponsor: Phoenix Children's Hospital • Aim: Diagnosis
Last updated Jun 27, 2026 07:51 UTC
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Childhood eye cancer survivors share life lessons in landmark study
Knowledge-focused OngoingThis study looks at the long-term health and quality of life of adults who had retinoblastoma, a rare eye cancer, as children. Researchers want to understand how survivors are doing physically and emotionally, and what challenges they face. The goal is to improve care and follow-…
Sponsor: Memorial Sloan Kettering Cancer Center • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:01 UTC
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Eye cancer survivors: does anesthesia affect the brain?
Knowledge-focused By invitation onlyThis study looks at how cancer treatment and multiple rounds of anesthesia affect thinking skills, emotions, and quality of life in people who survived retinoblastoma (a rare eye cancer) or were at risk for it. Researchers will test memory, attention, and other brain functions in…
Sponsor: Amsterdam University Medical Center • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:01 UTC
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Can yearly MRIs catch second cancers in retinoblastoma survivors?
Knowledge-focused OngoingThis study looks at whether yearly MRI scans can find new head and neck or brain cancers early in people who had hereditary retinoblastoma and were treated with radiation. Researchers will follow 88 participants for 10 years to see if early detection leads to better surgery outco…
Sponsor: Institut Curie • Aim: Knowledge-focused
Last updated Jun 26, 2026 16:56 UTC