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Alkylating agents

Carboplatin + Topotecan for Retinoblastoma

Phase 2

Waitlist Available

Led By Carlos Rodriguez-Galindo, MD

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

Participants must have an adequate liver function, as defined by bilirubin ≤ to 3X upper limit of normal (ULN), and SGOT and SGPT ≤ to 3X ULN.

Participants must have adequate renal function as defined by serum creatinine ≤ to 3X ULN for age.

Must not have

Presence of metastatic disease or gross (residual) orbital involvement

Participants must not have an invasive infection at time of protocol entry.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up after two upfront courses of chemotherapy (approximately two months after patient enrollment)

Awards & highlights

No Placebo-Only Group

Summary

This trial is studying how well carboplatin and topotecan work when given together in treating patients with retinoblastoma.

Who is the study for?

This trial is for children with newly diagnosed, untreated intraocular retinoblastoma. It includes those who had unilateral retinoblastoma and now have it in the other eye. They must be able to perform daily activities (ECOG ≤ 2) and have normal kidney and liver function. Those with previous treatments, metastatic disease, active infections or unable to consent are excluded.

What is being tested?

The study tests how well advanced intraocular retinoblastoma responds to direct-eye chemotherapy (carboplatin) plus systemic topotecan. It also uses various therapies like cryotherapy, thermotherapy, radiation therapy, surgery or enucleation if necessary while trying to preserve vision.

What are the potential side effects?

Possible side effects include reactions from chemotherapy drugs like nausea, hair loss, low blood counts leading to infection risk; damage from radiation; pain or complications from surgical procedures; and potential vision loss depending on treatment type.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My liver functions within normal limits.

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My kidney function is within the normal range for my age.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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My cancer has spread to other parts of my body or affects my eye socket.

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I do not have a serious infection right now.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ after two upfront courses of chemotherapy (approximately two months after patient enrollment)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~after two upfront courses of chemotherapy (approximately two months after patient enrollment)

This trial's timeline: 3 weeks for screening, Varies for treatment, and after two upfront courses of chemotherapy (approximately two months after patient enrollment) for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Response rate (complete or partial response)

Secondary study objectives

Event-free survival

The mechanism (or frequencies) for each RB1 biallelic inactivation

ocular survival

Side effects data

From 2014 Phase 3 trial • 361 Patients • NCT00976911

31%

Neutropenia

27%

Fatigue

20%

Anaemia

18%

Peripheral sensory neuropathy

17%

Hypertension

13%

Leukopenia

13%

Mucosal inflammation

12%

Proteinuria

11%

Infection

11%

Palmar-plantar erythrodysaesthesia syndrome

Abdominal pain

Diarrhoea

Nausea

Urinary tract infection

Vomiting

Alopecia

Constipation

Thrombocytopenia

Dyspnoea

Weight decreased

Decreased appetite

Abdominal pain upper

Epistaxis

General physical health deterioration

Subileus

Ileus

Pyrexia

Catheter site necrosis

Hypertransaminasaemia

Bacteraemia

Posterior reversible encephalopathy syndrome

Hypertensive crisis

Bone pain

Embolism venous

Gastroenteritis

Breast cancer

Ischaemic stroke

Myocardial ischaemia

Shock

Venous thrombosis

Influenza like illness

Intestinal obstruction

Small intestinal obstruction

Intestinal perforation

Syncope

Arterial occlusive disease

Deep vein thrombosis

Embolism arterial

Sepsis

Wrong drug administered

Tooth abscess

Bone disorder

Nephrotic syndrome

Skin ulcer

Device related infection

Dehydration

Vesical fistula

Female genital tract fistula

Pleural effusion

Pneumonia aspiration

Gastrointestinal haemorrhage

Haemorrhagic ascites

Ileal perforation

Ileal stenosis

Infectious peritonitis

Postoperative wound infection

Pulmonary embolism

Cardiac arrest

Coronary artery disease

Coronary artery stenosis

100%

80%

60%

40%

20%

Study treatment Arm

Chemotherapy + Bevacizumab

Chemotherapy

Awards & Highlights

No Placebo-Only Group

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

Trial Design

4Treatment groups

Experimental Treatment

Group I: Stratum DExperimental Treatment9 Interventions

Participants with bilateral retinoblastoma who may require upfront enucleation for one eye due to advanced disease (R-E IV-V and IC E). Interventions (see Detailed Description): vincristine, carboplatin, topotecan, etoposide, enucleation, filgrastim or PEG-filgrastim, focal therapy, including cryotherapy, laser photocoagulation, thermotherapy (and thermo-chemotherapy) and episcleral plaque brachytherapy, and external beam radiation or proton beam radiation in select cases.

Group II: Stratum CExperimental Treatment9 Interventions

Participants with advanced (R-E IV-V and IC D-E) unilateral retinoblastoma who require upfront enucleation. Participants will be assessed and treated by low, intermediate or high risk. Interventions (see Detailed Description): vincristine, cyclophosphamide, MESNA, doxorubicin, etoposide, carboplatin, filgrastim or PEG-filgrastim, enucleation

Group III: Stratum BExperimental Treatment7 Interventions

Participants considered candidates for conservative management including those: 1. Participants with bilateral retinoblastoma who have R-E IV-V and IC D in one eye 2. Participants with advanced unilateral (unifocal or multifocal) retinoblastoma (R-E IV-V and IC D-E) who demonstrate foveal sparing by the tumor during EUA. Due to foveal sparing, these patients have potential for vision preservation. Interventions (see Detailed Description): vincristine, topotecan, carboplatin, etoposide, filgrastim or PEG-filgrastim and focal therapy, including cryotherapy, laser photocoagulation, thermo-therapy, plaque radiotherapy.

Group IV: Stratum AExperimental Treatment6 Interventions

Participants with early bilateral or unilateral (unifocal or multifocal) retinoblastoma (R-E I-III, IC A-B; R-E IV with IC A or B; or IC C with limited sub-retinal seeding), and participants with bilateral disease in whom the advanced eye has been enucleated upfront (without any high risk histopathology) and the remaining eye has early stage disease (as defined above). Interventions (see detailed description): vincristine, carboplatin, topotecan, filgrastim or PEG-filgrastim, and focal therapy, including cryotherapy, laser photocoagulation, thermo-therapy, plaque radiotherapy.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

carboplatin

2010

Completed Phase 3

~4790

doxorubicin

2005

Completed Phase 3

~9130