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Recruiting NCT04903678

Intrathecal Chemotherapy for Central Nervous System Metastasis in Retinoblastoma

No phase Interventional Retinoblastoma, Extraocular

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Melphalan.
Who it may be relevant to
Registry conditions: Retinoblastoma, Extraocular. Basic parameters: No limits · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
China
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

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Official title

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

Overview

Retinoblastoma is the most common intraocular malignancy in infancy and childhood. The overall survival rate of retinoblastoma was reported to exceed 95% when children were early diagnosed with localized intraocular phase. The advanced retinoblastoma cases with central nervous system metastasis (CNS)is associated with exceedingly poor prognosis. CNS metastasis is the main cause of death in retinoblastoma. Meningeal involvement combined with spinal cord membrane involvement might be due to the meninges invasion and/or the spreading of CSF. As the most sensitive drug to retinoblastoma tumor cells in vitro, melphalan is the most important drug in the local treatment of retinoblastoma, and it is also an irreplaceable drug in the current eye preservation treatment, which greatly improves the success rate of eye preservation for advanced retinoblastoma. According to the research and the practice of vitreous injection (melphalan), the drug concentration in vitreous can effectively kill retinoblastoma tumor cells. It is reasonable to speculate that the tumor cells in CSF can be effectively killed by melphalan. Therefore, based on the above background, this study will explore the effectiveness and feasibility of intrathecal injection of melphalan in patients with CNS metastasis of retinoblastoma through a multicenter prospective study.

Interventions

  • Drug Melphalan
    Melphalan is performed for intrathecal chemotherapy

Primary outcome measures

  • progression-free survival [Time frame: 1 year]
  • overall survival at 2-year interval [Time frame: 2 years]
  • overall survival at 5-year interval [Time frame: 5 years]
  • response rate [Time frame: 1 year]
  • disease-free survival at 2-year interval [Time frame: 2 years]
  • disease-free survival at 5-year interval [Time frame: 5 years]
Secondary outcome measures (6)
  • Number of Participants with Arachnoiditis [Time frame: 2 years]
  • Number of Participants with Encephalopathy [Time frame: 5 years]
  • Number of Participants with Headache [Time frame: 2 years]
  • Number of Participants with Encephalomyelitis infection [Time frame: 1 year]
  • Health-Related Quality of life [Time frame: 10 years]
  • Ophthalmic Cancer-Related Quality of life [Time frame: 10 years]

Eligibility 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

China · 1 center
  • Fudan Eye & ENT Hospital — Shanghai

Identifiers

NCT: NCT04903678 · FD-EENT-2021056

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗