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NCT Number: NCT04903678

Intrathecal Chemotherapy for Central Nervous System Metastasis in Retinoblastoma

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.

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Key information

Conditions

Retinoblastoma, Extraocular Eye Diseases Eye Diseases, Hereditary Eye Neoplasms Neoplasms Neoplasms by Histologic Type Neoplasms by Site Neoplasms, Germ Cell and Embryonal Neoplasms, Glandular and Epithelial Neoplasms, Nerve Tissue Neoplasms, Neuroepithelial Neuroectodermal Tumors Retinal Diseases Retinal Neoplasms Retinoblastoma

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Fudan Eye & ENT Hospital

Shanghai, China

Location status: Recruiting

Location contact

Jiang Qian, MD

CONTACT

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

melphalan

Drug

Melphalan is performed for intrathecal chemotherapy

Primary outcomes

  1. progression-free survival

    Time frame: 1 year

    living subjects

  2. overall survival at 2-year interval

    Time frame: 2 years

    living subjects

  3. overall survival at 5-year interval

    Time frame: 5 years

    living subjects

  4. response rate

    Time frame: 1 year

    tumor-free subjects

  5. disease-free survival at 2-year interval

    Time frame: 2 years

    tumor-free subjects

  6. disease-free survival at 5-year interval

    Time frame: 5 years

    tumor-free subjects

Secondary outcomes

  1. Number of Participants with Arachnoiditis

    Time frame: 2 years

    A disorder characterized by inflammation of the arachnoid membrane and adjacent subarachnoid space.

  2. Number of Participants with Encephalopathy

    Time frame: 5 years

    A disorder characterized by a pathologic process involving the brain.

  3. Number of Participants with Headache

    Time frame: 2 years

    A disorder characterized by a sensation of marked discomfort in various parts of the head, not confined to the area of distribution of any nerve.

  4. Number of Participants with Encephalomyelitis infection

    Time frame: 1 year

    A disorder characterized by an infectious process involving the brain and spinal cord tissues.

  5. Health-Related Quality of life

    Time frame: 10 years

    The health-related quality of life will be assessed based on the EORTC (European Organisation for Research and Treatment of Cancer) Quality of Life Questionnaire Core 30 (QLQ-C30) downloaded from https://www.eortc.org. This 30-item questionnaire includes five functional scales, three symptom scales, a global health status / QoL scale, and six single items. The multi-item scales include a different set of items. Each item includes four subscales, namely "Not at all" (1 point), "A little" (2 points), "Quite a bit" (3 points) and "Very much" (4 points)

  6. Ophthalmic Cancer-Related Quality of life

    Time frame: 10 years

    The ophthalmic cancer-related quality of life will be assessed based on the EORTC (European Organisation for Research and Treatment of Cancer) Quality of Life Questionnaire Ophthalmic Cancer Module 30 (QLQ-OPT30)

    • Page 4 of 6 [DRAFT] - downloaded from https://www.eortc.org. The QLQ-OPT30 comprises of 30 questions assessing vision impairment (9 items), functional problems due to vision impairment (8 items), eye symptoms (6 items), worry about recurrent disease (2 items), problems with appearance (1 items), problems driving (2 items), headache (1 item), problems reading (1 item). Each item includes four subscales, namely "Not at all" (1 point), "A little" (2 points), "Quite a bit" (3 points) and "Very much" (4 points).

Study contacts

Contact information is provided by the study sponsor or research team.

Jiang Qian, MD

CONTACT

[email protected]

+86 (021) 64377134

Sponsors and collaborators

Lead sponsor

Eye & ENT Hospital of Fudan University

Other

Collaborators

  • Children's Hospital of Fudan University
  • Zhongshan Ophthalmic Center, Sun Yat-sen University

Registry information

Official study title

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

Important dates

Study start
2021
Primary completion
2028
Study completion
2030
First posted
May 26, 2021
Registry last updated
Feb 25, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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