Retinoblastoma Phase II Expanded Access Clinical Trial
This study is looking into a new way to treat advanced or recurring retinoblastoma, a type of eye cancer, using a treatment called Episcleral Topotecan. For children whose retinoblastoma is advanced or hasn't responded to other treatments, eye removal is often suggested. This study aims to see if a "chemoplaque" (a small device that delivers medicine) can help save the eye. You might be able to join if you are under 8 years old and have retinoblastoma that is advanced, or if it has come back after initial treatments. The main goal is to check the safety and how well Episcleral Topotecan is tolerated, by looking at any side effects over 12 weeks and during follow-up. The current status of this study is unclear.
- Study design
- This interventional study plans to enroll 30 participants to investigate the safety and effectiveness of Episcleral Topotecan.
- What's involved
- You would have a chemoplaque placed during surgery, which stays on your eye for 56 days. You would have eye exams under anesthesia on Day 0, Day 28, Day 56 (before removal), and Day 84, with periodic blood draws.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed until Day 84, when the study treatment is completed.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
NCT06679634
Retinoblastoma Phase II Expanded Access Clinical Trial
Recruiting
PHASE2Up to 8InterventionalTreatmentTargeted Therapy Technologies, LLC
~30 participants
Updated 2026-05-22 on ClinicalTrials.gov
What's tested:Chemoplaque also referred to as Episcleral Topotecan
At a glance
Recruiting sites
2 of 2 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
To determine the safety and tolerability of Episcleral Topotecan in participants with retinoblastoma by measuring the rate and severity of treatment related adverse events in study participants as assessed by CTCAE version 5.0 criteria.
Measured over 12 weeks and follow up
Conditions
Where it's being run
2 sites across 2 statesNew York1
Wisconsin1
Who to contact
Opens a ready-to-send draft in your own email app — review before sending.
Do you actually qualify for this trial?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Inclusion
Age: Participants must be \< 8 years of age.
Diagnosis and Treatment. Participants must have: Group D or earlier-stage intraocular retinoblastoma in which enucleation is a recommended therapy, OR Active residual or recurrent intraocular retinoblastoma in at least one eye following completion of first- line therapy (focal therapy for IIRC Group A eyes, or systemic or intra-arterial chemotherapy).
Must have demonstrated intraocular calcium in the tumor-containing eye by ophthalmic ultrasound or by neuroimaging as part of standard retinoblastoma diagnosis.
Study eye must have vision potential, at least light perception vision in the tumor-bearing eye either with pupil response testing or demonstration of avoidance behavior to light presentation in the affected eye, and no clinical features suggestive of high risk of extraocular extension.
Performance Level: Lansky ≥ 50 (\<16 years of age); Karnofsky performance scale of ≥50 (≥16 years of age).
Organ Function Requirements:
Peripheral absolute neutrophil count (ANC) ≥ 1000/mm3
Platelet count ≥ 100,000/mm3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 Days prior to enrollment)
Hemoglobin ≥ 8.0 g/dL at baseline (may receive RBC transfusions) 2. Adequate Renal Function defined as:
Creatinine clearance or radioisotope GFR ≥70ml/min/1.73 m2 or
A serum creatinine based on age/gender as follows:
Bilirubin (sum of conjugated + unconjugated) ≤ 1.5 x upper limit of normal (ULN) for age.
SGPT (ALT) ≤ 110 U/L. For the purpose of this study, the ULN for SGPT is 45 U/L.
Serum albumin ≥ 2 g/dL. 4. 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 Episcleral Topotecan administration. 5. Informed consent. All participants 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.
Exclusion
Disease status. Participants known to have any of the following are excluded:
Allergy. Participants with reported allergy to topotecan, camptothecin or derivatives thereof are excluded.
Concomitant treatment. Participants who have received chemotherapy, other focal retinoblastoma therapy or any other investigational agent within 3 weeks of Episcleral Topotecan placement are not eligible.
Uncontrolled intercurrent illness. Participants with known uncontrolled intercurrent illness that, in the investigator's opinion, would put the participant at undue risk or limit compliance with the study requirements, are not eligible.
Febrile illness. Participants with clinically significant febrile illness (as determined by the investigator) within one week prior to initiation of protocol therapy are excluded.
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.
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.
What this trial measures
- To determine the safety and tolerability of Episcleral Topotecan in participants with retinoblastoma by measuring the rate and severity of treatment related adverse events in study participants as assessed by CTCAE version 5.0 criteria.12 weeks and follow up