1. Age. Patients must be <18 years of age.
2. Diagnosis and Treatment. Patients must have active residual or recurrent intraocular
retinoblastoma in at least one eye following completion of first-line therapy
(chemotherapy, systemic or intra-arterial, or brachytherapy).
3. One eye will be the Study Eye. When patients have two eyes with retinoblastoma, the
eye with worst disease or best vision potential will be designated the Study Eye.
There will only be one eye per child treated in this Phase I study, since treatment of
two eyes would double the systemic dose of drug. The Non-study eye will be treated by
standard of care, with only focal therapy during the Study Period, if required.
4. Disease status. Study eye must have vision potential and no clinical features
suggestive of high risk of extraocular extension.
5. Performance status. Lansky play score ≥ 50 if <16 years of age; Karnofsky performance
scale of ≥ 50 if ≥16 years of age (Appendix I)
6. Organ function:
1. Adequate bone marrow function and platelet count
2. Adequate renal function
3. Adequate liver function
7. Pregnancy prevention. Females of reproductive potential must agree to the use of
highly effective contraception during study participation and for an additional 40
days after the end of the Chemoplaque administration
8. Informed consent. All patients and/or their parents or legally authorized
representatives must have the ability to understand and the willingness to sign a
written informed consent. Assent, where appropriate, will also be obtained.
1. Disease status. Any of the following:
1. tumour involving the optic nerve rim
2. clinical or EUA evidence of extraocular extension
3. evidence of metastatic retinoblastoma
4. existing neuroimaging showing suspicion of, or definitive, optic nerve invasion,
trilateral retinoblastoma or extra-ocular extension.
2. Allergy. Reported allergy to topotecan, camptothecin or derivatives thereof.
3. Concomitant treatment. Patients may not receive chemotherapy or other focal
retinoblastoma therapy or any other investigational agent within 3 weeks of the
placement and removal of the Chemoplaque, nor while the Chemoplaque is in situ.
4. Uncontrolled intercurrent illness. Patients with uncontrolled intercurrent illness
that, in the investigator's opinion, would put the participant at undue risk or limit
compliance with the study requirements.
5. Febrile illness. Patients with clinically significant febrile illness (as determined
by the investigator) within one week prior to initiation of protocol therapy.
6. Pregnancy and lactation. Females of reproductive potential must have a negative serum
pregnancy test within 72 hours prior to initiation of protocol therapy. Due to the
unknown but potential risk for adverse events (AEs) in nursing infants secondary to
treatment of the mother with the study agents, breastfeeding must be discontinued if
the mother is treated on study.
7. Compliance. Any condition of diagnosis that could in the opinion of the Principal
Investigator or delegate interfere with the participant's ability to comply with the
study instruction, might confound the interpretation of the study results, or put the
participant at risk.