RECRUITING

Retinoblastoma Phase II Expanded Access Clinical Trial

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

Retinoblastoma Phase II Expanded Access Clinical Trial

Quick Facts

Study Start:2025-02-21

Study Completion:2028-11

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06679634

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:Not specified to 8 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD

Inclusion Criteria	Exclusion 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). * 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: 1. to \< 2 years 0.6 0.6 2. to \< 6 years 0.8 0.8 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.	* 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.

Inclusion Criteria

Exclusion 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).
* 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:
1. to \< 2 years 0.6 0.6
2. to \< 6 years 0.8 0.8
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.

* 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.

Contacts and Locations

Study Contact

David Carpi, MSc

CONTACT

949-892-5363

info@3tophthalmics.com

Study Locations (Sites)

NewYork Presbyterian Morgan Stanley Children's Hospital

New York, New York, 10032

United States

Collaborators and Investigators

Sponsor: Targeted Therapy Technologies, LLC

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-02-21

Study Completion Date2028-11

Study Record Updates

Study Start Date2025-02-21

Study Completion Date2028-11

Terms related to this study

Keywords Provided by Researchers

Retinoblastoma

Additional Relevant MeSH Terms

Retinoblastoma