Retinoblastoma Phase II Expanded Access Clinical Trial

NCT06679634 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: 3TRB01-2
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 2

Brief 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. 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.

Conditions

Retinoblastoma

Keywords

Retinoblastoma

Outcome Measures

Primary Outcomes (1)

• 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

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.

  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.

  12 weeks

• To describe the anti-tumor efficacy of Episcleral Topotecan

  12 weeks and follow up

Study Arms (1)

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.

Drug: Chemoplaque also referred to as Episcleral Topotecan

Interventions

Chemoplaque also referred to as Episcleral Topotecan DRUG

Sustained Release Episcleral Topotecan

Also known as: Episcleral Topotecan

Chemoplaque

Eligibility Criteria

• Age: Up to 8 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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:
• 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

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Targeted Therapy Technologies, LLC: lead
• Morgan Stanley Children's Hospital: collaborator

Study Sites (2)

NewYork Presbyterian Morgan Stanley Children's Hospital

New York, New York, 10032, United States

RECRUITING

Children's Wisconsin

Milwaukee, Wisconsin, 53226, United States

RECRUITING

MeSH Terms

Conditions

Retinoblastoma

Condition Hierarchy (Ancestors)

Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue; Retinal Neoplasms; Eye Neoplasms; Neoplasms by Site; Eye Diseases, Hereditary; Eye Diseases; Retinal Diseases

Central Study Contacts

Brian Marr, MD

CONTACT

212-305-0011; bpm2133@cumc.columbia.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Single Arm Open Label Expanded Access

Study Record Dates

• First Submitted: November 5, 2024
• First Posted: November 7, 2024
• Study Start: February 21, 2025
• Primary Completion (Estimated): November 1, 2028
• Study Completion (Estimated): November 1, 2028
• Last Updated: April 14, 2026

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)