Retinoblastoma Phase II Expanded Access Clinical Trial
ConditionRetinoblastoma
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
AgeUp to 8
SponsorTargeted Therapy Technologies, LLC
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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.
Trial design
Design model
Single group
Treatments tested in this trial
Chemoplaque also referred to as Episcleral Topotecan
DrugSustained Release Episcleral Topotecan
Treatment groups
30 Participants
are divided into 1 treatment groupGroup A: ChemoplaqueExperimental treatment 1 intervention
Trial outcomes
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.
Time frame
12 weeks and follow upSecondary outcomes
1
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 up2
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 weeks3
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.
Time frame
12 weeks and follow upSponsors and contacts
Click on the lead sponsor to view all of their trials.
Targeted Therapy Technologies, LLC
Lead sponsor
Morgan Stanley Children's Hospital
Collaborator
Source ClinicalTrials.gov