Status:
RECRUITING
High-dose Topotecan for Retinoblastoma With Recurrent or Refractory Vitreous Seed
Lead Sponsor:
Eye & ENT Hospital of Fudan University
Conditions:
Retinoblastoma
Retinoblastoma, Recurrent
Eligibility:
All Genders
1-12 years
Phase:
EARLY_PHASE1
Brief Summary
Retinoblastoma is the most common intraocular malignancy in infancy and childhood,with an estimated 8000 new cases globally each year.The major cause of failure in the management of retinoblastoma rem...
Eligibility Criteria
Inclusion
- Patients with retinoblastoma with a somatic mutation of the gene RB1 and active tumor in a single eye, or germinal mutation of RB1 with active tumor/s in an eye and the contralateral eye unaffected, enucleated or without tumor activity. In both cases, relapsed or refractory vitreous seeding with the use of systemic, intraarterial or intravitreal chemotherapy or radiotherapy, in accordance with the availability at his/her referral site, in whom enucleation is the only recommended treatment under opinion of the medical team treating the case at the originating referral site. But the patient has a strong desire to preserve the eye.
- Normal renal function: serum creatinine: \< 45 μmol/L (0-2 years); \< 57 μmol/L (3-6 years); \< 60 μmol/L (7-10 years); \< 80 μmol/L (11-13 years).
- Normal Hepatic function: serum ALT: \< 0,52 μkat/L (de 9 months -12 years); serum AST: 61-80 g/L (8 months-5 years); 63-83 g/L (5-9 years); 63-82 g/L (9-12 years).
- Adequate marrow reserve manifested in an absolute neutrophil count \> 1000 / mm3, platelets \> 100,000 / mm3 and hemoglobin\> 8 g / dl, without transfusional or cytokine support at least one month prior to study entry.
- Age greater than 1 year and less than 12 years at the time of inclusion in the study.
- Sign the informed consent form and be willing to follow up at the specified time.
Exclusion
- Presence of factors that require immediate enucleation of the affected eye such as glaucoma, rubeosis iridis, anterior chamber involvement.
- Comorbidities: Uncontrolled epilepsy with anticonvulsant treatment, cardiac disease not compensated by treatment.
- Active Infections.
- Other chronic or active acute diseases that under the criterion of the researcher were an exclusion criterion.
- History of having received attenuated or live vaccines in the 30 days prior to inclusion in the study.
- Any cause of Immunosuppression.
- Trilateral Retinoblastoma.
- Extraocular spread.
- History of having received treatment for retinoblastoma with chemotherapy or radiation therapy by any means within 30 days prior to inclusion in the study.
- Patients who can not complete the study procedures for reasons psychologically or socially
Key Trial Info
Start Date :
April 1 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 31 2027
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT06972602
Start Date
April 1 2025
End Date
December 31 2027
Last Update
May 15 2025
Active Locations (1)
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1
Fudan Eye & ENT Hospital
Shanghai, China, China