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Alkylating agents

Individualized Therapy for Retinoblastoma

Phase 3

Waitlist Available

Led By Ibrahim Qaddoumi, M.D.

Research Sponsored by St. Jude Children's Research Hospital

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up patients were assessed at 5 years of age

Awards & highlights

Pivotal Trial

No Placebo-Only Group

Summary

This trial is investigating how children with retinoblastoma respond to different individualized therapies. The main goal is to cure the cancer and save the child's life, while also saving their vision.

Who is the study for?

This trial is for children with newly diagnosed retinoblastoma, a type of eye cancer. They must have good liver and kidney function, be expected to live at least 8 weeks, and have a performance status showing they can do some daily activities. Kids who've had surgery or focal treatments on one eye but then get cancer in the other are also eligible. Children with spread of cancer outside the eye or an active infection cannot join.

What is being tested?

The study tests various treatments based on whether one or both eyes are affected and how advanced the disease is. It includes chemotherapy combinations like topotecan and vincristine (with G-CSF support), carboplatin around the eye, plus potential surgeries and focal therapies like laser treatment. The aim is to cure cancer, save vision, understand how kids adapt visually post-treatment, and learn more about retinoblastoma biology.

What are the potential side effects?

Treatments may cause side effects such as reactions from chemotherapy drugs including nausea, hair loss, increased risk of infections due to low blood cell counts (from G-CSF), liver issues indicated by abnormal blood tests results (bilirubin/SGOT/SGPT levels), fatigue from radiation therapy; specific side effects will depend on individual responses.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ patients were assessed at 5 years of age

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~patients were assessed at 5 years of age

This trial's timeline: 3 weeks for screening, Varies for treatment, and patients were assessed at 5 years of age for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Stratum B Response to Window Therapy

Secondary study objectives

Assessment of School Readiness

Change in Cognitive Functioning

Change in Distortion Product Otoacoustic Emissions (DPOAEs)

+26 more

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

3Treatment groups

Experimental Treatment

Group I: Stratum CExperimental Treatment5 Interventions

Patients with advanced unilateral advanced intraocular disease. First intervention is enucleation. If enucleated eye does not have disease outside the retina (low risk), no additional treatment is given. For patients whose enucleated eye shows tumor outside the retina (intermediate risk), they will receive 4 courses of vincristine, cyclophosphamide, and doxorubicin followed by G-CSF. For patients with high risk disease (involvement of the sclera, optic nerve at the level of the cut-end), treatment after enucleation is 6 courses of alternating chemotherapy with vincristine, carboplatin, etoposide (VCE) to alternate with vincristine, cyclophosphamide, and doxorubicin (VCD). High risk patients also receive external-beam radiation therapy.

Group II: Stratum BExperimental Treatment8 Interventions

Patients with bilateral disease (at least one advanced stage eye), candidate for conservative management. Treatment included window treatment with vincristine and topotecan, Followed by 3 more courses of vincristine-topotecan if they had a response to the window+ 6 courses of vincristine and carboplatin. If they do not respond to the window, they receive 6 courses of vincristine, carboplatin, and etoposide. Periocular carboplatin is also given three times, depending on whether they respond to window. External Beam Radiation 44-46 Gy administered using standard practices.

Group III: Stratum AExperimental Treatment5 Interventions

Patients with early bilateral or unilateral, or patients with bilateral that have already had the advanced eye enucleated. Treatment included vincristine and carboplatin for 8 courses, given at 3-4 week intervals. Focal therapies any time after second course can include cryotherapy, laser photocoagulation, thermotherapy, and plaque radiotherapy

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

External Beam Radiation

2014

Completed Phase 2

~70

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