Topotecan Episcleral Plaque for Treatment of Retinoblastoma

  • End date
    Oct 14, 2023
  • participants needed
  • sponsor
    Targeted Therapy Technologies, LLC
Updated on 9 October 2021
platelet count
renal function
neutrophil count
intraocular retinoblastoma


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.


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.

Condition Retinoblastoma
Treatment Episcleral Topotecan
Clinical Study IdentifierNCT04156347
SponsorTargeted Therapy Technologies, LLC
Last Modified on9 October 2021


Yes No Not Sure

Inclusion Criteria

Age: Participants must be < 18 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
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
Adequate Bone Marrow Function defined as
Peripheral absolute neutrophil count (ANC) greater than or equal 1000/mm3
Platelet count greater than or equal 100,000/mm3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 Days prior to enrollment)
Hemoglobin greater than or equal 8.0 g/dL at baseline (may receive RBC transfusions)
Adequate Renal Function defined as
Creatinine clearance or radioisotope GFR greater than or equal 70ml/min/1.73 m2 or
A serum creatinine based on age/gender as follows
Age Maximum Serum Creatinine (mg/dL) Male Female
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
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) less than or equal 1.5 x upper limit of normal (ULN) for age
SGPT (ALT) less than or equal 110 U/L. For the purpose of this study, the ULN for SGPT is 45 U/L
Serum albumin greater than or requal 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
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
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