Chemo plaque could spare Kids' eyes from removal
NCT ID NCT06679634
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase 2 trial tests a chemotherapy eye implant (Episcleral Topotecan) in 30 children under 8 with advanced or recurrent retinoblastoma. The goal is to see if the implant can safely treat the eye tumor and avoid the need to remove the eye. Researchers are checking for side effects and how well the drug works at different doses.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Episcleral Topotecan (chemotherapy eye implant)
- What this could lead to
- If successful, this could offer a way to save eyes with advanced retinoblastoma, avoiding removal while controlling the cancer.
- What could go wrong
- This is a small, early-phase trial (30 children) focused on safety and dosing. It may not work for all, and side effects from the chemotherapy implant are possible.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 30 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Feb 2025
- Expected to finish
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Dec 2029
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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Up to 8 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * 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). If both eyes require treatment, there will receive independent and individual treatment. Non-study eye will be treated by standard of care, with only focal therapy during the Study Period, if required. * 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: 1. Adequate Bone Marrow Function defined as: * 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: Age Maximum Serum Creatinine (mg/dL) Male Female 1 month to \< 6 months 0.4 0.4 6 months to \< 1 year 0.5 0.5 1. to \< 2 years 0.6 0.6 2. to \< 6 years 0.8 0.8 6 to \< 7 years 1 1 The threshold creatinine values in this Table were derived from the Schwartz formula for estimating GFR utilizing child length and stature data published by the CDC. 3. Adequate Liver Function defined as: * 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 Criteria: * Disease status. Participants known to have any of the following are excluded: 1. tumor 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. * 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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
2 sites. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Children's Wisconsin
RECRUITINGMilwaukee, Wisconsin, 53226, United States
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NewYork Presbyterian Morgan Stanley Children's Hospital
RECRUITINGNew York, New York, 10032, United States
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