Retinoblastoma, ExtraocularNCT04903678
Intrathecal Chemotherapy for Central Nervous System Metastasis in Retinoblastoma
A study testing Melphalan in people with Retinoblastoma, Extraocular, run by Eye & ENT Hospital of Fudan University at 1 site in China.
Trial summary
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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Retinoblastoma, Extraocular
- What is being tested?
- Melphalan
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteChina
- Study dates
- 1 May 2021to 1 May 2030
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Fudan Eye & ENT Hospital
Shanghai
Jiang Qian, MD
About this study
Official title: Intrathecal Chemotherapy for Central Nervous System Metastasis in Retinoblastoma (A Multicenter Prospective Single Arm Trial)
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.
- Conditions
- Retinoblastoma, Extraocular
- Being tested
- Melphalan
- Keywords
- retinoblastoma, central metastases, intrathecal Chemotherapy
- Medical subject headings
- Retinoblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MelphalanDrug
Melphalan is performed for intrathecal chemotherapy
| Study group | Role | What they receive |
|---|---|---|
| intrathecal chemotherapy in patients with central metastases | Experimental | Systemic chemotherapy and intrathecal chemotherapy are performed every 3 weeks. After three treatment cycles, the treatment response is comprehensively evaluated including cerebrospinal fluid, intracranial and orbital tumors. If necessary, local radiotherapy and arterial interventional chemotherapy are performed for local solid tumors. Cerebrospinal fluid is detected in each treatment cycle. If RB tumor cells still exist in cerebrospinal fluid, chemotherapy and intrathecal chemotherapy are continued until the end event. If cerebrospinal fluid was negative, intrathecal chemotherapy is supplemented for another 2 cycles with a total of 6 cycles of systemic chemotherapy. The patients are followed up after treatment. Receives: Drug: Melphalan |
What is being measured
The primary outcome is the main question the study is designed to answer.
progression-free survival
living subjects
Measured: 1 year
overall survival at 2-year interval
living subjects
Measured: 2 years
overall survival at 5-year interval
living subjects
Measured: 5 years
response rate
tumor-free subjects
Measured: 1 year
disease-free survival at 2-year interval
tumor-free subjects
Measured: 2 years
disease-free survival at 5-year interval
tumor-free subjects
Measured: 5 years
Secondary outcomes (6)
Number of Participants with Arachnoiditis
A disorder characterized by inflammation of the arachnoid membrane and adjacent subarachnoid space.
Measured: 2 years
Number of Participants with Encephalopathy
A disorder characterized by a pathologic process involving the brain.
Measured: 5 years
Number of Participants with Headache
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.
Measured: 2 years
Number of Participants with Encephalomyelitis infection
A disorder characterized by an infectious process involving the brain and spinal cord tissues.
Measured: 1 year
Health-Related Quality of life
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)
Measured: 10 years
Ophthalmic Cancer-Related Quality of life
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).
Measured: 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 May 2021
- Study start1 May 2021
- Primary completion1 May 2028 (estimated)
- Study completion1 May 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Retinoblastoma, ExtraocularStudies of the same condition, open and under way.
- More trials run by Eye & ENT Hospital of Fudan UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Eye & ENT Hospital of Fudan University
- Sponsor type
- University, hospital or other
- Collaborators
- Zhongshan Ophthalmic Center, Sun Yat-sen University, Children's Hospital of Fudan University
- Responsible party
- Principal investigator, Jiang Qian, Eye & ENT Hospital of Fudan University
- Investigators
- Jiang Qian, MD, Principal investigator, Fudan Eye & ENT Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04903678
- Sponsor's protocol ID
- FD-EENT-2021056
- Status last verified
- Feb 2025
- First submitted
- 22 May 2021
- Last updated
- 25 Feb 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 25 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Intrathecal Chemotherapy for Central Nervous System Metastasis in Retinoblastoma (A Multicenter Prospective Single Arm Trial)
- ClinicalTrials.gov ID
- NCT04903678
- Acronym / sponsor ID
- FD-EENT-2021056
- Conditions
- Retinoblastoma, Extraocular
- Keywords
- retinoblastoma, central metastases, intrathecal Chemotherapy
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- Eye & ENT Hospital of Fudan UniversityUniversity, hospital or other
- Collaborators
- Zhongshan Ophthalmic Center, Sun Yat-sen University, Children's Hospital of Fudan University
- Study start
- 1 May 2021 (actual)
- Primary completion
- 1 May 2028 (estimated)
- Study completion
- 1 May 2030 (estimated)
- First posted
- 26 May 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| intrathecal chemotherapy in patients with central metastases | Experimental | Drug: MelphalanSystemic chemotherapy and intrathecal chemotherapy are performed every 3 weeks. After three treatment cycles, the treatment response is comprehensively evaluated including cerebrospinal fluid, intracranial and orbital tumors. If necessary, local radiotherapy and arterial interventional chemotherapy are performed for local solid tumors. Cerebrospinal fluid is detected in each treatment cycle. If RB tumor cells still exist in cerebrospinal fluid, chemotherapy and intrathecal chemotherapy are continued until the end event. If cerebrospinal fluid was negative, intrathecal chemotherapy is supplemented for another 2 cycles with a total of 6 cycles of systemic chemotherapy. The patients are followed up after treatment. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (11 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MelphalanMelphalan is performed for intrathecal chemotherapy | Drug | intrathecal chemotherapy in patients with central metastases |
Primary outcome
progression-free survival
Time frame: 1 year
living subjects
overall survival at 2-year interval
Time frame: 2 years
living subjects
overall survival at 5-year interval
Time frame: 5 years
living subjects
response rate
Time frame: 1 year
tumor-free subjects
disease-free survival at 2-year interval
Time frame: 2 years
tumor-free subjects
disease-free survival at 5-year interval
Time frame: 5 years
tumor-free subjects
6 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Feb 2025.
- Individual participant dataSharing planned: Yes
Related trials for Retinoblastoma, Extraocular
RecruitingObservational study
Detailed Phenotypic and Genotype Study to Correlate RB1 Mutations Relating to Primary Ocular Tumors and Secondary Extra-ocular Metastasis.
Retinoblastoma Bilateral, Retinoblastoma Unilateral
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Jiang Qian, MD principal investigator
Fudan Eye & ENT Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Melphalan in people with Retinoblastoma, Extraocular, run by Eye & ENT Hospital of Fudan University at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 25 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jiang Qian, MD (+86 (021) 64377134, qianjiang@fudan.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
progression-free survival (time frame: 1 year). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Feb 2025.