Adjuvant Chemotherapy for High-risk Retinoblastoma After Enucleation

Three Cycles Versus Six Cycles of Adjuvant Chemotherapy for the Patients With High-risk Retinoblastoma After Enucleation: Prospective Randomized Control Study

The purpose of this study is to determine whether 3 cycles of chemotherapy(CEV) is not inferior to 6 cycles of chemotherapy(CEV) in the treatment of Stage I enucleated retinoblastoma.

Study Overview

• Status: Active, not recruiting
• Conditions: Retinoblastoma
• Intervention / Treatment: Drug: 3 cycles chemotherapy; Drug: 6 cycles chemotherapy

Detailed Description

This study will be a phase Ⅲ open label interventional case series. Patients with unilateral Stage I enucleated retinoblastoma will be randomized to receive chemotherapy(CEV) of 3 cycles or 6 cycles every 21 days. Patients will be followed for 60 months.

• Study Type: Interventional
• Enrollment (Actual): 179
• Phase: Phase 3

Contacts and Locations

Study Locations

• China
  • Guangdong
    • Guangzhou, Guangdong, China, 510000
      • Zhongshan Ophthalmic Center, Sun Yat-sen University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Ability to provide written informed consent and comply with study assessments for the full duration of the study.
• Definite pathology signs of retinoblastoma, Stage I base on International Retinoblastoma Staging System.
• Received enucleation in the study eye.
• Monocular retinoblastoma.

Exclusion Criteria:

• Any previous disease in the study eye.
• Previous participation in any studies of investigational drugs within 1 month preceding Day 0 (excluding vitamins and minerals).
• History of chemical intervention for retinoblastoma in the study eye.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Single

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 3 cycles chemotherapy; Chemotherapy: vincristine 0.05 mg/kg (or 1.5 mg/m2 for children ≥ 3 years of age) on Day 1, carboplatin 18.6 mg/kg (or 560 mg/m2 for children ≥ 3 years of age) on Day 1, etoposide 5.0 mg/kg/day (or 150 mg/ m2 for children ≥ 3 years of age) on Days 1 and 2 and 2. Cycles were repeated every 21 days for three cycles.	Drug: 3 cycles chemotherapy; Chemotherapy: vincristine 0.05 mg/kg (or 1.5 mg/m2 for children ≥ 3 years of age) on Day 1, carboplatin 18.6 mg/kg (or 560 mg/m2 for children ≥ 3 years of age) on Day 1, etoposide 5.0 mg/kg/day (or 150 mg/ m2 for children ≥ 3 years of age) on Days 1 and 2 and 2. Cycles were repeated every 21 days for three cycles.; Other Names: vincristine, carboplatin, etoposide.
Active Comparator: 6 cycles chemotherapy; Chemotherapy: vincristine 0.05 mg/kg (or 1.5 mg/m2 for children ≥ 3 years of age) on Day 1, carboplatin 18.6 mg/kg (or 560 mg/m2 for children ≥ 3 years of age) on Day 1, etoposide 5.0 mg/kg/day (or 150 mg/ m2 for children ≥ 3 years of age) on Days 1 and 2 and 2. Cycles were repeated every 21 days for six cycles.	Drug: 6 cycles chemotherapy; Chemotherapy: vincristine 0.05 mg/kg (or 1.5 mg/m2 for children ≥ 3 years of age) on Day 1, carboplatin 18.6 mg/kg (or 560 mg/m2 for children ≥ 3 years of age) on Day 1, etoposide 5.0 mg/kg/day (or 150 mg/ m2 for children ≥ 3 years of age) on Days 1 and 2 and 2. Cycles were repeated every 21 days for six cycles.; Other Names: vincristine, carboplatin, etoposide.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
disease-free survival	measure the disease-free survival rate for the two groups at five years	five years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
overall survival	time from randomization to death as a result of any cause at five years	five years
safety: side effects of chemotherapy in the Treatment of Retinoblastoma	Number of participants with side effects (systemic check-up, audio acuity, ect)before each treatment, at 1,3,6,9,12,18,24,30,36,42,48,54,60 months after the treatment.	five years
quality of life (PedsQL4.0 scale) in the Treatment of Retinoblastoma	quality of life (PedsQL4.0 scale) before each treatment, at 1,3,6,9,12,18,24,30,36,42,48,54,60 months after the treatment. PedsQL 4.0 questionnaire has two modules (reported by parents and children) with 23 items encompassing the physical domains (8 items), emotional (5 items), social (5 items) and school (5 items). A five scores scale is used, after being asked, ''How much, in the last month, your child has had problems with'' (0 = never a problem; 1 = almost never a problem; 2 = sometimes a problem; 3 = is often a problem and 4 = almost always a problem). The items are reverse-score and linearly transformed to a scale from 0 to 100 (0=100, 1=75, 2=50, 3=25, 4 = 0). So high scores indicate better QoL.	five years
economic burden due to chemotherapy	All costs, including direct cost and indirect cost, are expressed in Chinese YUAN. Direct costs include the average cost of systemic chemotherapy, operation, supportive treatment, and examination. Indirect costs include accommodation, transportation, diet, and lost income of parents.	during chemotherapy (9 weeks for 3-cycle group, and 18 weeks for 6-cycle group)

Collaborators and Investigators

• Sponsor: Sun Yat-sen University
• Collaborators: Fudan University
• Investigators: Study Chair: Huasheng Yang, Doctor, Zhongshan Ophthalmic Center, Sun Yat-sen University

Study record dates

Study Major Dates

• Study Start (Actual): August 1, 2013
• Primary Completion (Estimated): April 1, 2024
• Study Completion (Estimated): April 1, 2024

Study Registration Dates

• First Submitted: July 20, 2013
• First Submitted That Met QC Criteria: July 23, 2013
• First Posted (Estimated): July 24, 2013

Study Record Updates

• Last Update Posted (Actual): April 9, 2024
• Last Update Submitted That Met QC Criteria: April 6, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Neoplasms by Site; Neoplasms, Glandular and Epithelial; Eye Diseases; Retinal Diseases; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms, Nerve Tissue; Eye Diseases, Hereditary; Eye Neoplasms; Retinal Neoplasms; Retinoblastoma; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Antineoplastic Agents; Tubulin Modulators; Antimitotic Agents; Mitosis Modulators; Antineoplastic Agents, Phytogenic; Topoisomerase II Inhibitors; Topoisomerase Inhibitors; Carboplatin; Etoposide; Etoposide phosphate; Vincristine
• Other Study ID Numbers: yanghs20130507

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No