Topotecan Episcleral Plaque for Treatment of Retinoblastoma
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ClinicalTrials.gov Identifier: NCT04156347 |
Recruitment Status :
Active, not recruiting
First Posted : November 7, 2019
Last Update Posted : October 31, 2023
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Tracking Information | |||||||
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First Submitted Date ICMJE | November 4, 2019 | ||||||
First Posted Date ICMJE | November 7, 2019 | ||||||
Last Update Posted Date | October 31, 2023 | ||||||
Actual Study Start Date ICMJE | June 16, 2021 | ||||||
Estimated Primary Completion Date | October 1, 2024 (Final data collection date for primary outcome measure) | ||||||
Current Primary Outcome Measures ICMJE |
To determine the safety and tolerability of Episcleral Topotecan in participants with retinoblastoma-Maximum Tolerated Dose (MTD) and/or Recommended Phase II Dose (RP2D). [ Time Frame: 9 Weeks ] To determine the safety and tolerability of Episcleral Topotecan in participants with retinoblastoma.
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Original Primary Outcome Measures ICMJE |
The primary outcome measure of the study is safety assessment as determined by comprehensive ophthalmic exam that enables determination of maximum tolerated dose. [ Time Frame: 29 Days ] The primary outcome measure of the study is safety assessment as determined by comprehensive ophthalmic exam that enables determination of maximum tolerated dose.
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Change History | |||||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
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Current Other Pre-specified Outcome Measures | Not Provided | ||||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||||
Descriptive Information | |||||||
Brief Title ICMJE | Topotecan Episcleral Plaque for Treatment of Retinoblastoma | ||||||
Official Title ICMJE | A Phase I Study of Sequestered Transscleral, Controlled-Release Topotecan Delivered From an Episcleral Reservoir in Retinoblastoma Eyes | ||||||
Brief Summary | This single-arm, non-randomized, dose escalation phase I clinical trial will assess primarily the safety and secondarily the efficacy of episcleral topotecan in patients with active de novo or recurrent intraocular retinoblastoma in at least one eye following completion of first-line therapy. | ||||||
Detailed Description | Retinoblastoma is the most common pediatric malignant intraocular tumor and originates from the retina. Treatment of eyes with advanced intraocular retinoblastoma remains a challenge. The historic standard of care for patients with unilateral disease is enucleation and for those with bilateral disease, a variety of modalities have been tried. These include radiation therapy, systemic chemotherapy, periocular administration of chemotherapy, selective intra-arterial chemotherapy, and intravitreal chemotherapy. Unfortunately, all of these modalities are associated with significant morbidity and investigators are looking for new ways to treat these patients either with novel directed drug delivery methods or with new less toxic agents. This study will evaluate the safety and efficacy of topotecan delivered directly to the eye using a novel sustained-release topotecan episcleral delivery system (also referred to as a Chemoplaque) in patients with active de novo or recurrent intraocular retinoblastoma in at least one eye following completion of first-line therapy. The study intervention involves the insertion and removal of the Chemoplaque, examinations under anaesthesia (EUAs), visits to clinic to monitor for adverse events throughout, and post plaque removal toxicity evaluation. EUAs, clinic visits and laboratory tests are standard of care for retinoblastoma patients. | ||||||
Study Type ICMJE | Interventional | ||||||
Study Phase ICMJE | Phase 1 | ||||||
Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment |
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Condition ICMJE | Retinoblastoma | ||||||
Intervention ICMJE | Drug: Episcleral Topotecan
Transscleral Topotecan
Other Name: Episcleral Topotecan, Transscleral Topotecan, Sustained Release Episcleral Topotecan, Chemoplaque
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Study Arms ICMJE | Experimental: Phase I Open Label Study
Phase I Single Arm
Intervention: Drug: Episcleral Topotecan
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Publications * | Not Provided | ||||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||||
Recruitment Status ICMJE | Active, not recruiting | ||||||
Estimated Enrollment ICMJE |
42 | ||||||
Original Estimated Enrollment ICMJE |
12 | ||||||
Estimated Study Completion Date ICMJE | October 14, 2024 | ||||||
Estimated Primary Completion Date | October 1, 2024 (Final data collection date for primary outcome measure) | ||||||
Eligibility Criteria ICMJE | Inclusion Criteria: Age: Participants must be < 21 years of age. Diagnosis and Treatment. Participants must have: Unilateral Group D or earlier-stage intraocular retinoblastoma in which enucleation is a recommended therapy, with no previous local or systemic therapy for retinoblastoma with intraocular calcium in the tumor-containing eye by ophthalmic ultrasound or neuroimaging. 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). One eye will be the Study Eye. When participants have two eyes with retinoblastoma, the eye with worst disease or best vision potential will be designated the Study Eye. There will only be one eye per child treated in this Phase I study, since treatment of two eyes would double the systemic dose of drug. The 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 greater than or equal 50 (<16 years of age); Karnofsky performance scale of >50 (>16 years of age). Organ Function Requirements:
Exclusion Criteria 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. |
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Sex/Gender ICMJE |
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Ages ICMJE | up to 18 Years (Child, Adult) | ||||||
Accepts Healthy Volunteers ICMJE | No | ||||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||||
Listed Location Countries ICMJE | United States | ||||||
Removed Location Countries | Canada | ||||||
Administrative Information | |||||||
NCT Number ICMJE | NCT04156347 | ||||||
Other Study ID Numbers ICMJE | 3TRB01 | ||||||
Has Data Monitoring Committee | Yes | ||||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
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Current Responsible Party | Targeted Therapy Technologies, LLC | ||||||
Original Responsible Party | Same as current | ||||||
Current Study Sponsor ICMJE | Targeted Therapy Technologies, LLC | ||||||
Original Study Sponsor ICMJE | Same as current | ||||||
Collaborators ICMJE | Not Provided | ||||||
Investigators ICMJE | Not Provided | ||||||
PRS Account | Targeted Therapy Technologies, LLC | ||||||
Verification Date | April 2023 | ||||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |