Intrathecal chemotherapy for advanced retinoblastoma with CNS metastasis
Official title: Intrathecal Chemotherapy for Central Nervous System Metastasis in Retinoblastoma (A Multicenter Prospective Single Arm Trial)
Run by Eye & ENT Hospital of Fudan University · NCT04903678 · Data updated 2026-11-12
This study is testing if a special chemotherapy treatment given directly into the spinal fluid can help children with advanced retinoblastoma that has spread to the brain.
Quick facts
| Study type | Interventional |
|---|---|
| Sex | All |
| Enrollment | 18 (estimated) |
| Sponsor | Eye & ENT Hospital of Fudan University Academic / other |
| Locations | 1 site (Shanghai) |
| Status | Recruiting |
| Started | May 1, 2021 |
| Main results expected | May 1, 2028 |
| Study ends | May 1, 2030 |
| Registry record updated | February 25, 2025 |
| Trial ID | NCT04903678 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates include children diagnosed with stage IVB retinoblastoma who have evidence of CNS metastasis and meet specific health criteria.
✕Probably not a fit
Patients with other eye diseases, recent infections, or those with certain systemic conditions may not benefit from this treatment.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This clinical trial investigates the use of intrathecal chemotherapy with melphalan for treating advanced retinoblastoma that has metastasized to the central nervous system. Retinoblastoma is a common childhood eye cancer, and when it spreads to the CNS, it significantly worsens the prognosis. The study aims to evaluate the effectiveness of melphalan, a potent drug against retinoblastoma cells, in improving outcomes for patients with this severe condition. Participants will be closely monitored for treatment response and side effects.
Why it matters
Potential benefit: If successful, this treatment could significantly improve survival rates and quality of life for children with advanced retinoblastoma and CNS metastasis.
How similar studies have gone: While the use of melphalan has shown promise in treating retinoblastoma, this specific approach for CNS metastasis is novel and has not been extensively tested in prior studies.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: * The pathological diagnosis was retinoblastoma. According to the IRSs (International retinoblastoma staging system) staging system, the stage was IVB * CSF tumor cells on cytology, or radiographic evidence of NM on MRI scans and histologic diagnosis of systemic malignancy needed * KPS≥60% * Adequate bone marrow and organ function * The parents signed the informed consent and were willing to accept the treatment and follow-up. Exclusion Criteria: * 1.Eye diseases other than retinoblastoma * The eyes were infected within 30 days before screening * There was a history of surgery and / or unhealed wound within 1 month before enrollment . * Those who have allergic reaction or allergic history to chemotherapeutic drugs. * Infectious diseases requiring oral, intramuscular or intravenous administration. * Patients with systemic immune diseases. * Active disseminated intravascular coagulation. * Abnormal coagulation function. * Abnormal bone marrow and organ function. * Uncontrollable clinical problems. * The researchers believe that those who need to be excluded.
Where it's running
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Fudan Eye & ENT Hospital
Shanghai, China Recruiting
Study contacts
- Principal investigator: Jiang Qian, MD — Fudan Eye & ENT Hospital
- Study coordinator: Jiang Qian, MD
- Email: qianjiang@fudan.edu.cn
- Phone: +86 (021) 64377134
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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Linking RB1 genetic changes to eye tumors and metastasis
We will use targeted long-read genetic sequencing on people with retinoblastoma to see if specific RB1 mutations match tumor features and the risk of spread.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.