Actively Recruiting
High-dose Topotecan for Retinoblastoma With Recurrent or Refractory Vitreous Seed A Prospective Non-controlled Single Arm Trial
Led by Eye & ENT Hospital of Fudan University · Updated on 2025-05-15
10
Participants Needed
1
Research Sites
104 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Retinoblastoma is the most common eye cancer in infants and children worldwide, often complicated by persistent or returning vitreous seeding which challenges treatment success. This trial investigates a new approach using high-dose Topotecan, a drug that damages cancer cell DNA and promotes tumor cell death, to treat resistant or returning retinoblastoma. The study aims to assess how well this targeted treatment works and its safety, including effects on the immune system and vision preservation. Participants will receive an intravitreal injection of Topotecan at a dose of 100 micrograms, given on the first day of the first week. Treatment frequency will be personalized by eye doctors based on the tumor stage and response. Follow-up visits occur every four weeks where eye fluid samples are collected and electroretinograms are performed to monitor treatment effects and eye health. Treatment continues until the vitreous seeding disappears or stabilizes. Throughout the study, patients will be regularly checked with eye exams, laboratory tests, and vision assessments. Researchers will measure tumor response after 28 days and monitor for side effects, immune reactions, and visual function over 13 weeks. This prospective trial lasts up to three years with ongoing evaluations to understand the drug’s impact on eye tumors and safety.
CONDITIONS
Brief Title
High-dose Topotecan for Retinoblastoma With Recurrent or Refractory Vitreous Seed
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients with retinoblastoma having RB1 gene mutation and active tumor in one eye, or germinal RB1 mutation with tumor in one eye and no tumor activity in the other eye
- Relapsed or refractory vitreous seeding after prior chemotherapy or radiotherapy where enucleation is recommended but patient desires eye preservation
- Normal kidney function within age-specific serum creatinine limits
- Normal liver function within specified ALT and AST ranges for age
- Adequate bone marrow function with specific neutrophil, platelet, and hemoglobin counts without recent support
- Age greater than 1 year and less than 12 years at study entry
- Signed informed consent and willingness to follow up as scheduled
You will not qualify if you...
- Conditions requiring immediate removal of the affected eye, such as glaucoma or anterior chamber involvement
- Uncontrolled epilepsy or heart disease not managed by treatment
- Active infections
- Other chronic or acute diseases deemed exclusionary by the researcher
- Receipt of live or attenuated vaccines within 30 days before study entry
- Any immunosuppression cause
- Presence of trilateral retinoblastoma
- Spread of cancer outside the eye
- Retinoblastoma treatment with chemotherapy or radiation within 30 days prior to study
- Inability to complete study procedures due to psychological or social reasons
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Varies until complete disappearance of vitreous seeding or stable calcification
Participants receive intravitreal injections of topotecan. The initial dose is given on day 1 of week 1, with further injections personalized by ophthalmologists based on tumor response and vitreous seeding status.
Visits every 4 weeks for treatment evaluation and procedures
Duration - Up to 13 weeks
Participants are followed up with collection of aqueous humor samples and electroretinogram examinations every 4 weeks to monitor treatment response and safety.
Visits every 4 weeks
Trial Site Locations
Total: 1 location
1
Fudan Eye & ENT Hospital
Shanghai, China, China
Actively Recruiting
Research Team
K
Kang Xue, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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