Actively Recruiting
Intravitreal Melphalan Combined with Chemotherapy for Treating Intraocular Retinoblastoma with Vitreous Seeding
Led by Children's Oncology Group · Updated on 2026-02-17
26
Participants Needed
20
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying retinoblastoma (RB), a cancer of the retina, focusing on cases with vitreous seeds, which are tumors in the eye's jelly-like fluid and increase treatment difficulty and risk. This phase II trial aims to evaluate the safety and side effects of adding melphalan, injected directly into the eye, to standard chemotherapy. The trial also explores the effectiveness and long-term visual outcomes of this combined treatment approach for Group D retinoblastoma with vitreous seeding. Participants receive systemic chemotherapy with carboplatin, vincristine, and etoposide over cycles lasting 28 days each. During the first two cycles, patients undergo imaging and urine sample collection without melphalan injections. From cycle 3 onwards, eligibility for melphalan injections is assessed before each cycle, and eligible patients receive one injection of melphalan into the eye per cycle, for up to six injections. Imaging, tissue sampling, and magnetic resonance imaging are conducted throughout the trial. Participants are monitored with eye imaging under anesthesia, ultrasound biomicroscopy, and blood and urine tests at regular intervals during treatment. After treatment, follow-ups occur at 4 weeks, then every 3 months for one year, and every 3 to 6 months for up to five years. Researchers measure the success of melphalan administration combined with chemotherapy, track serious side effects, and evaluate long-term event-free survival and visual outcomes.
CONDITIONS
Brief Title
A Study to Give Treatment Inside the Eye to Treat Retinoblastoma
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patient is younger than 18 years at enrollment
- Newly diagnosed intraocular retinoblastoma meeting one of these: unilateral Group D with vitreous seeding; bilateral retinoblastoma with worst eye Group D and vitreous seeding and other eye Group A-C; bilateral Group D with at least one eye with vitreous seeding; bilateral with one Group D eye with vitreous seeding and one Group E eye enucleated before therapy
- Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2
- Peripheral absolute neutrophil count (ANC) of at least 750/uL within 7 days before enrollment
- Platelet count of at least 75,000/uL without transfusion within 7 days before enrollment
- Serum creatinine within specified age and sex limits or adequate creatinine clearance or glomerular filtration rate (GFR) within 7 days before enrollment
- Total bilirubin less than or equal to 1.5 times upper limit of normal for age within 7 days before enrollment
- Serum glutamate pyruvate transaminase (SGPT/ALT) less than or equal to 135 U/L within 7 days before enrollment
- Written informed consent signed by patient and/or parents or guardians
- Meets all institutional, FDA, and NCI human study requirements
You will not qualify if you...
- Evidence of metastatic or extra-orbital spread of disease
- Presence of invasive infection at time of study entry
- Previous anti-cancer therapy other than cryotherapy or laser therapy to study or non-study eyes
- High-risk histopathology features in enucleated Group E eye prior to therapy including large choroid involvement, optic nerve involvement, or scleral invasion
- Female patients who are pregnant
- Lactating females planning to breastfeed
- Sexually active patients of reproductive potential not using effective contraception during study
- Failure to meet all institutional, FDA, and NCI human study requirements
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person) for eligibility assessments including blood tests and eye examinations
Duration - Up to 6 cycles of 28 days each
Participants receive systemic chemotherapy with carboplatin, vincristine, and etoposide every 28 days for up to 6 cycles. Starting with cycle 3, participants may receive an additional intravitreal injection of melphalan if eligible. Eye examinations under anesthesia and imaging occur prior to each cycle to assess eligibility for melphalan injection.
1 visit per cycle including intravenous chemotherapy and eye examinations; melphalan injection given once per cycle if eligible starting cycle 3
Duration - Up to 5 years
After completing study treatment, participants are followed up to monitor safety and disease status with periodic visits.
1 visit at 4 weeks post-treatment, then visits every 3 months for 1 year, and every 3-6 months for years 2 to 5
Trial Site Locations
Total: 20 locations
1
Children's Hospital of Alabama
Birmingham, Alabama, United States, 35233
Actively Recruiting
2
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
Actively Recruiting
3
Lucile Packard Children's Hospital Stanford University
Palo Alto, California, United States, 94304
Actively Recruiting
4
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
5
Children's Healthcare of Atlanta - Arthur M Blank Hospital
Atlanta, Georgia, United States, 30329
Actively Recruiting
6
C S Mott Children's Hospital
Ann Arbor, Michigan, United States, 48109
Actively Recruiting
7
Washington University School of Medicine
St Louis, Missouri, United States, 63110
Actively Recruiting
8
Duke University Medical Center
Durham, North Carolina, United States, 27710
Actively Recruiting
9
Children's Hospital Medical Center of Akron
Akron, Ohio, United States, 44308
Actively Recruiting
10
Cleveland Clinic Foundation
Cleveland, Ohio, United States, 44195
Actively Recruiting
11
Saint Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Actively Recruiting
12
Dell Children's Medical Center of Central Texas
Austin, Texas, United States, 78723
Actively Recruiting
13
UT Southwestern/Simmons Cancer Center-Dallas
Dallas, Texas, United States, 75390
Actively Recruiting
14
Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center
Houston, Texas, United States, 77030
Actively Recruiting
15
M D Anderson Cancer Center
Houston, Texas, United States, 77030
Actively Recruiting
16
Primary Children's Hospital
Salt Lake City, Utah, United States, 84113
Active, Not Recruiting
17
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, United States, 53226
Actively Recruiting
18
Royal Children's Hospital
Parkville, Victoria, Australia, 3052
Actively Recruiting
19
Perth Children's Hospital
Perth, Western Australia, Australia, 6009
Actively Recruiting
20
Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec, Canada, H3T 1C5
Actively Recruiting
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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