Combination Chemotherapy + Stem Cell Transplant/Radiation for Retinoblastoma

Recruiting at 77 trial locations

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Children's Oncology Group

Disqualifiers: Prior chemotherapy, Prior radiotherapy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 7

60 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This phase III trial is studying the side effects and how well giving combination chemotherapy together with autologous stem cell transplant and/or radiation therapy works in treating young patients with extraocular retinoblastoma. Giving chemotherapy before an autologous stem cell transplant stops the growth of tumor cells by stopping them from dividing or killing them. After treatment, stem cells are collected from the patient's blood and/or bone marrow and stored. More chemotherapy is given to prepare the bone marrow for the stem cell transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy. Radiation therapy uses high energy x-rays to kill tumor cells. Giving radiation therapy after combination chemotherapy and/or autologous stem cell transplant may kill any remaining tumor cells.

Who Is on the Research Team?

Ira J Dunkel

Principal Investigator

Children's Oncology Group

Are You a Good Fit for This Trial?

This trial is for young patients with extraocular retinoblastoma, a type of eye cancer that has spread outside the eye. They should be in good physical condition (ECOG score 0-2), have not received prior treatment for this condition, and meet specific blood count and organ function criteria. Consent from patients or guardians is required.

Inclusion Criteria

All patients and/or their parents or legal guardians must sign a written informed consent

All institutional, FDA, and NCI requirements for human studies must be met

Serum glutamic oxaloacetic transaminase (SGOT) (AST) or serum glutamate pyruvate transaminase (SGPT) (ALT) < 2.5 x ULN

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Induction Chemotherapy

Patients receive vincristine, cisplatin, cyclophosphamide, etoposide, and filgrastim. Treatment repeats every 21 days for up to 4 courses.

12 weeks

4 visits (in-person)

Stem Cell Harvesting

Peripheral blood stem cells or bone marrow cells are collected after at least 1 course of induction chemotherapy.

1 week

High-Dose Consolidation Chemotherapy

Patients receive carboplatin, thiotepa, and etoposide.

1 week

Autologous Stem Cell Infusion

Patients undergo autologous stem cell infusion and receive filgrastim until blood counts recover.

1 week

Radiotherapy

Patients undergo radiotherapy to sites initially involved based on response.

6 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment.

1 year

Every 3 months for 1 year, then annually for 9 years

What Are the Treatments Tested in This Trial?

Interventions

• Autologous Bone Marrow Transplantation
• Autologous Hematopoietic Stem Cell Transplantation
• Carboplatin
• Cisplatin
• Etoposide
• Filgrastim
• In Vitro-Treated Peripheral Blood Stem Cell Transplantation
• Radiation Therapy
• Thiotepa
• Vincristine Sulfate

Trial Overview The study tests how well combination chemotherapy works with autologous stem cell transplant and/or radiation therapy to treat these patients. The process involves using drugs to stop tumor growth, collecting the patient's own stem cells, giving more chemo to prepare for transplanting these cells back into the patient, followed by possible radiation therapy.

How Is the Trial Designed?

Group I: Treatment (chemotherapy, radiotherapy, autologous SCI) Experimental Treatment 11 Interventions

INDUCTION: Patients receive vincristine IV; cisplatin IV; cyclophosphamide IV; and G-CSF SC beginning on day 3 and continuing until blood counts recover. CONSOLIDATION (stage 4a or 4b disease only): Patients receive carboplatin IV; thiotepa IV; and etoposide IV. AUTOLOGOUS STEM CELL INFUSION (stage 4a or 4b disease only): Patients undergo autologous stem cell infusion on day 0 and receive G-CSF SC beginning on day 1 and continuing until blood counts recover. RADIOTHERAPY: Patients with stage 2 or 3 disease (orbital and/or regional involvement) undergo radiotherapy to sites that were initially involved beginning within 42 days after the start of course 4 of induction chemotherapy. Patients with stage 4a or 4b disease undergo radiotherapy to sites initially involved based on response beginning approximately 42 days after autologous stem cell infusion.

Carboplatin is already approved in United States, European Union, Canada for the following indications:

Approved in United States as Paraplatin for:

• Ovarian cancer
• Testicular cancer
• Lung cancer
• Head and neck cancer
• Brain cancer

Approved in European Union as Carboplatin for:

• Ovarian cancer
• Small cell lung cancer

Approved in Canada as Carboplatin for:

• Ovarian cancer
• Small cell lung cancer
• Testicular cancer

Who Is Running the Clinical Trial?

Children's Oncology Group

Lead Sponsor

Trials

467

Recruited

241,000+

National Cancer Institute (NCI)

Collaborator

Trials

14,080

Recruited

41,180,000+