Intrathecal chemotherapy for advanced retinoblastoma with CNS metastasis

Intrathecal Chemotherapy for Central Nervous System Metastasis in Retinoblastoma (A Multicenter Prospective Single Arm Trial)

NA · Eye & ENT Hospital of Fudan University · NCT04903678

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

PhaseNA
Study typeInterventional
Enrollment18 (estimated)
SexAll
SponsorEye & ENT Hospital of Fudan University (other)
Locations1 site (Shanghai)
Trial IDNCT04903678 on ClinicalTrials.gov

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.

Who should consider this trial

Good fit: Ideal candidates include children diagnosed with stage IVB retinoblastoma who have evidence of CNS metastasis and meet specific health criteria.

Not a fit: Patients with other eye diseases, recent infections, or those with certain systemic conditions may not benefit from this treatment.

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 performed: 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.

Eligibility criteria

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 this trial is running

Shanghai

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

Conditions: Retinoblastoma, Extraocular, retinoblastoma, central metastases, intrathecal Chemotherapy

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.