Actively Recruiting
Intrathecal Chemotherapy for Central Nervous System Metastasis in Retinoblastoma A Multicenter Prospective Single Arm Trial
Led by Eye & ENT Hospital of Fudan University · Updated on 2025-02-25
18
Participants Needed
1
Research Sites
104 weeks
Total Duration
On this page
Sponsors
E
Eye & ENT Hospital of Fudan University
Lead Sponsor
Z
Zhongshan Ophthalmic Center, Sun Yat-sen University
Collaborating Sponsor
AI-Summary
What this Trial Is About
Retinoblastoma is the most common eye cancer in infants and children. While early diagnosis of localized retinoblastoma has a survival rate exceeding 95%, cases with central nervous system (CNS) metastasis have a very poor prognosis, with CNS involvement being the main cause of death. This study investigates the use of intrathecal injection of melphalan, a drug sensitive to retinoblastoma tumor cells, to treat CNS metastasis in retinoblastoma through a multicenter prospective approach. Participants will receive systemic chemotherapy combined with intrathecal chemotherapy every three weeks. After three cycles, treatment response is evaluated by examining cerebrospinal fluid, brain, and eye tumors. If needed, local radiotherapy and arterial interventional chemotherapy may be applied for solid tumors. Chemotherapy continues until no tumor cells are found in cerebrospinal fluid, with a total of six cycles planned including additional intrathecal chemotherapy. Patients will be followed up after completing treatment. During the study, researchers monitor cerebrospinal fluid samples each cycle and assess tumor response. They will measure progression-free survival, overall survival, response rates, and disease-free survival at various time points up to five years. Secondary outcomes include monitoring side effects such as arachnoiditis, encephalopathy, headaches, infections, and quality of life related to health and eye cancer over up to ten years. The total duration of follow-up varies with long-term assessments included.
CONDITIONS
Brief Title
Intrathecal Chemotherapy for Central Nervous System Metastasis in Retinoblastoma
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosed with retinoblastoma stage IVB according to the International Retinoblastoma Staging System
- Presence of tumor cells in cerebrospinal fluid or radiographic evidence of nervous system metastasis confirmed by MRI and histology
- Karnofsky Performance Status of 60% or higher
- Adequate bone marrow and organ function
- Parents have signed informed consent and agree to treatment and follow-up
You will not qualify if you...
- Eye diseases other than retinoblastoma
- Eye infection within 30 days before screening
- History of surgery or unhealed wound within 1 month before enrollment
- Allergic reaction or history of allergy to chemotherapy drugs
- Infectious diseases requiring oral, intramuscular or intravenous treatment
- Presence of systemic immune diseases
- Active disseminated intravascular coagulation
- Abnormal coagulation function
- Abnormal bone marrow or organ function
- Uncontrollable clinical problems
- Any other conditions deemed exclusionary by the researchers
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)
Duration - Approximately 18 weeks (6 cycles every 3 weeks)
Participants receive systemic chemotherapy and intrathecal chemotherapy every 3 weeks. After three treatment cycles, response is evaluated including cerebrospinal fluid, intracranial and orbital tumors. Additional local radiotherapy and arterial interventional chemotherapy may be performed if needed. Chemotherapy continues depending on cerebrospinal fluid tumor cell presence, up to a total of 6 cycles of systemic chemotherapy.
Every 3 weeks for up to 6 cycles
Duration - Up to 10 years
Participants are followed up after completing treatment to monitor health outcomes and quality of life.
Visits at intervals over several years as per protocol
Trial Site Locations
Total: 1 location
1
Fudan Eye & ENT Hospital
Shanghai, China
Actively Recruiting
Research Team
J
Jiang Qian, MD
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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