RetinoblastomaPhase 2NCT06679634
Retinoblastoma Phase II Expanded Access Clinical Trial
A Phase 2 study testing Chemoplaque also referred to as Episcleral Topotecan in people with Retinoblastoma, run by Targeted Therapy Technologies, LLC at 2 sites in United States.
Trial summary
In this research study investigators want to learn more about treatment of advanced or recurrent retinoblastoma. For children with retinoblastoma that have an advanced stage of presentation in one eye or if they have failed all conventional treatment, eye removal is considered. Read more about this study
- Who is it for?
- Ages Up to 8 years
- Conditions the study is for
- Retinoblastoma
- What is being tested?
- Chemoplaque also referred to as Episcleral Topotecan
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 2 sitesUnited States
- Study dates
- 21 Feb 2025to Dec 2029
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 8 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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).
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- Organ Function Requirements:
- 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)
- 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
- to < 2 years 0.6 0.6
- 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.
- 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.
- 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.
- 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.
Who cannot join
- Disease status. Participants known to have any of the following are excluded:
- tumor involving the optic nerve rim
- clinical or EUA evidence of extraocular extension
- evidence of metastatic retinoblastoma
- 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.
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- 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.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States2 sites, 2 recruiting
- Recruiting
Children's Wisconsin
Milwaukee, Wisconsin, 53226
Aparna Ramasubramanian, MD, 414-266-2020, aramasubramanian@mcw.edu
Kimberly Mayer, RN, 414-477-5336, kmayer@childrenswi.org
- Recruiting
NewYork Presbyterian Morgan Stanley Children's Hospital
New York, New York, 10032
Brian Marr, MD, 610-6594664
Lauren Yeager, MD, 847-840-1082
About this study
In this research study investigators want to learn more about treatment of advanced or recurrent retinoblastoma. For children with retinoblastoma that have an advanced stage of presentation in one eye or if they have failed all conventional treatment, eye removal is considered. This study will investigate the utility of a chemoplaque(s) to salvage eyes involved with retinoblastoma. The goal of the study is to further determine/assess the safety and efficacy and optimal chemotherapy dose for retinoblastoma.
Read the detailed description
The chemoplaque will be placed during the first exam under anesthesia (EUA) and this involves a surgery wherein the reservoir that will release the chemotherapy medication will be placed under the conjunctiva (surface lining of the eye) and will be applied with tissue adhesive to the sclera (coating of the eye). The chemoplaque will remain on the eye until day 56 when the chemoplaque will be removed. Eye exam under anesthesia (EUAs) will be performed on Day 0, Day 28, Day 56 (prior to chemoplaque removal), and Day 84 (final visit). Blood will be drawn periodically for testing the blood parameters (CBC) and to evaluate toxicity of the medication. Study subjects will be followed until Day 84 when protocol therapy will be completed.
- Conditions
- Retinoblastoma
- Being tested
- Chemoplaque also referred to as Episcleral Topotecan
- Keywords
- Retinoblastoma
- Medical subject headings
- Retinoblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Chemoplaque also referred to as Episcleral TopotecanDrug
Also called Episcleral Topotecan
Sustained Release Episcleral Topotecan
| Study group | Role | What they receive |
|---|---|---|
| Chemoplaque | Experimental | Chemoplaque (also referred to as Episcleral Topotecan) treatment will involve single application of the Episcleral Topotecan 1.2 mg dose that delivers sustained delivery for 8 weeks. Receives: Drug: Chemoplaque also referred to as Episcleral Topotecan |
What is being measured
The primary outcome is the main question the study is designed to answer.
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: 12 weeks and follow up
Secondary outcomes (3)
To determine the safety and tolerability of Episcleral Topotecan in participants with retinoblastoma by measuring the rate and severity of treatment related ocular adverse events in study participants as assessed by CTCAE version 5.0 criteria.
Measured: 12 weeks and follow up
To determine the safety and tolerability of Episcleral Topotecan in participants with retinoblastoma by measuring the rate and severity of treatment related systemic adverse events in study participants as assessed by CTCAE version 5.0 criteria.
Measured: 12 weeks
To describe the anti-tumor efficacy of Episcleral Topotecan
Tumor response will be assessed via image analysis with following criteria: Complete Response (CR): Decrease in tumor activity 100% from baseline. Very good partial response (VGPR): Decrease in tumor activity greater than or equal to 80% and less than 100% from baseline. Partial response (PR): Decrease in tumor activity greater than or equal to 20% and less than 80% from baseline. Stable disease (SD): Less than 20% decrease in tumor activity from baseline. Progressive disease (PD): Increase in tumor activity greater than or equal to 20% from baseline or development of new tumours requiring more than minimal focal therapy.
Measured: 12 weeks and follow up
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Nov 2024
- Study start21 Feb 2025
- Primary completionNov 2029 (estimated)
- Study completionDec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for RetinoblastomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Single Arm Open Label Expanded Access
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Targeted Therapy Technologies, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Morgan Stanley Children's Hospital
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06679634
- Sponsor's protocol ID
- 3TRB01-2
- Status last verified
- May 2026
- First submitted
- 5 Nov 2024
- Last updated
- 22 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Retinoblastoma Phase II Expanded Access Clinical Trial
- ClinicalTrials.gov ID
- NCT06679634
- Acronym / sponsor ID
- 3TRB01-2
- Conditions
- Retinoblastoma
- Keywords
- Retinoblastoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Targeted Therapy Technologies, LLCIndustry (pharma, biotech, devices)
- Collaborators
- Morgan Stanley Children's Hospital
- Study start
- 21 Feb 2025 (actual)
- Primary completion
- Nov 2029 (estimated)
- Study completion
- Dec 2029 (estimated)
- First posted
- 7 Nov 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
In this research study investigators want to learn more about treatment of advanced or recurrent retinoblastoma. For children with retinoblastoma that have an advanced stage of presentation in one eye or if they have failed all conventional treatment, eye removal is considered. This study will investigate the utility of a chemoplaque(s) to salvage eyes involved with retinoblastoma. The goal of the study is to further determine/assess the safety and efficacy and optimal chemotherapy dose for retinoblastoma.
Detailed description
The chemoplaque will be placed during the first exam under anesthesia (EUA) and this involves a surgery wherein the reservoir that will release the chemotherapy medication will be placed under the conjunctiva (surface lining of the eye) and will be applied with tissue adhesive to the sclera (coating of the eye). The chemoplaque will remain on the eye until day 56 when the chemoplaque will be removed. Eye exam under anesthesia (EUAs) will be performed on Day 0, Day 28, Day 56 (prior to chemoplaque removal), and Day 84 (final visit). Blood will be drawn periodically for testing the blood parameters (CBC) and to evaluate toxicity of the medication. Study subjects will be followed until Day 84 when protocol therapy will be completed.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Single Arm Open Label Expanded Access
| Arm | Type | Receives |
|---|---|---|
| Chemoplaque | Experimental | Drug: Chemoplaque also referred to as Episcleral TopotecanChemoplaque (also referred to as Episcleral Topotecan) treatment will involve single application of the Episcleral Topotecan 1.2 mg dose that delivers sustained delivery for 8 weeks. |
Who may qualify
- Ages
- Up to 8 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (26 listed)
- 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.
Key exclusion criteria (11 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Chemoplaque also referred to as Episcleral TopotecanAlso: Episcleral TopotecanSustained Release Episcleral Topotecan | Drug | Chemoplaque |
Primary outcome
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.
Time frame: 12 weeks and follow up
3 secondary outcomes
To determine the safety and tolerability of Episcleral Topotecan in participants with retinoblastoma by measuring the rate and severity of treatment related ocular adverse events in study participants as assessed by CTCAE version 5.0 criteria.
Time frame: 12 weeks and follow up
To determine the safety and tolerability of Episcleral Topotecan in participants with retinoblastoma by measuring the rate and severity of treatment related systemic adverse events in study participants as assessed by CTCAE version 5.0 criteria.
Time frame: 12 weeks
To describe the anti-tumor efficacy of Episcleral Topotecan
Time frame: 12 weeks and follow up
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 May 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Chemoplaque also referred to as Episcleral Topotecan in people with Retinoblastoma, run by Targeted Therapy Technologies, LLC at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 26 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 22 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Brian Marr, MD (212-305-0011, bpm2133@cumc.columbia.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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. (time frame: 12 weeks and follow up). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 May 2026; AzTrials last refreshed its copy on 6 Nov 2026.