RetinoblastomaPhase 1/2ChemosurgeryNCT00901238
Intra-arterial Chemotherapy(Chemosurgery) for Retinoblastoma
A Phase 1/2 study testing Intra-arterial Chemotherapy in people with Retinoblastoma, run by Weill Medical College of Cornell University at 2 sites in United States.
Trial summary
Conventional treatments of retinoblastoma involves laser photocoagulation, cryotherapy (freezing of the tumor), plaque radiotherapy,external beam radiotherapy, and intravenous chemotherapy. Enucleation (removing of the eye)is the last option when the tumor cannot be controlled otherwise. Read more about this study
- Who is it for?
- Ages 1 month and older
- Conditions the study is for
- Retinoblastoma
- What is being tested?
- Intra-arterial Chemotherapy
- Type of treatment
- Injection or infusion, Surgery or procedure
- How many people
- 100 participants
- Where
- 2 sitesUnited States
- Study dates
- May 2006to Jul 2009
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
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 1 month and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- advanced retinoblastoma in one or both eyes
- recurrent retinoblastoma after failure of conventional methods
Who cannot join
- retinoblastoma judged curable by conventional methods
- patient judged unable to undergo the procedure
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
Weill Medical College of Cornell University
New York, New York, 10065
About this study
Conventional treatments of retinoblastoma involves laser photocoagulation, cryotherapy (freezing of the tumor), plaque radiotherapy,external beam radiotherapy, and intravenous chemotherapy. Enucleation (removing of the eye)is the last option when the tumor cannot be controlled otherwise. However,many children with retinoblastoma present with advanced intraocular disease for which enucleation is the only option. Intra-arterial chemotherapy (Chemosurgery)delivers anti-tumor drug directly into the ophthalmic artery (the artery feeding the eye) in order to increase the dose of drug reaching the tumor while minimizing toxicity to the rest of the body.
Read the detailed description
Present treatments for intraocular retinoblastoma cure 99% of children but have significant toxicity. Enucleation of the eye is effective but blinds the eye and leaves a lifelong cosmetic deformity. Radiation is associated with the subsequent development of fatal cancers. Systemic (intravenous)chemotherapy is used worldwide but experience with it has shown that the majority of eyes initially treated with chemotherapy still require additional treatments, such as radiation, laser, cryotherapy or even enucleation. In addition blood transfusions, secondary infections, insertion of ports and permanent hearing loss are now well reported. Three years ago we developed this technique of Chemosurgery for significantly increasing the dose of drug to the cancer while decreasing the dose of drug administered to children. This approach has decreased the need for enucleation in advanced eyes scheduled for enucleation with minimal systemic toxicity. We now offer treatment of both eyes simultaneously (in bilateral cases) and to eyes with less advanced disease and normal vision as an alternative to toxic systemic chemotherapy. In cases of very advanced ocular disease we will be using multiple drugs infused at the same session to increase tumor kill.
Chemosurgery interventions are performed under general anesthesia. The femoral artery (artery at the groin) is punctured and a catheter (a small plastic tube)is advanced into the opthalmic artery (the artery of the eye)using fluoroscopic (X-ray) guidance. The drugs are injected directly into the opthalmic artery over a period of 30-45 min.The catheter is then removed, manual compression exerted to the femoral artery, the child is awaken and goes to recovery for 6 hours. The procedure is repeated every 3-4 weeks for a total of 2 to 6 sessions according to tumor response. Since April 2006, our center has treated by chemosurgery 60 eyes in 52 patients with advanced intra-ocular retinoblastoma for which enucleation was considered.
- Conditions
- Retinoblastoma
- Being tested
- Intra-arterial Chemotherapy
- Keywords
- retinoblastoma, intra-arterial chemotherapy, chemosurgery, melphalan, topotecan
- Medical subject headings
- Retinoblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Intra-arterial ChemotherapyProcedure or surgery
Also called chemosurgery
selective catheterization of the ophthalmic artery and injection of melphalan and topotecan
What is being measured
The primary outcome is the main question the study is designed to answer.
tumor control
Measured: 6 month
Secondary outcomes (1)
tumor control with vision
Measured: 6 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 May 2009 (estimated)
- Study startMay 2006
- Primary completionJul 2009
- Study completionJul 2009
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for RetinoblastomaStudies of the same condition, open and under way.
- More trials run by Weill Medical College of Cornell 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Weill Medical College of Cornell University
- Sponsor type
- University, hospital or other
- Collaborators
- Memorial Sloan Kettering Cancer Center
- Investigators
- David H Abramson, MD, Principal investigator, Weill Cornell Medical College and Memorial Sloan-Kettering Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00901238
- Sponsor's protocol ID
- RTB1
- Acronym
- Chemosurgery
- Status last verified
- Dec 2009
- First submitted
- 12 May 2009
- Last updated
- 17 Dec 2009
- Results posted
- No
- Data monitoring committee
- No
Publications
Brodie SE, Pierre Gobin Y, Dunkel IJ, Kim JW, Abramson DH. Persistence of retinal function after selective ophthalmic artery chemotherapy infusion for retinoblastoma. Doc Ophthalmol. 2009 Aug;119(1):13-22. doi: 10.1007/s10633-008-9164-3. Epub 2009 Jan 25.
PubMed 19169884Abramson DH, Dunkel IJ, Brodie SE, Kim JW, Gobin YP. A phase I/II study of direct intraarterial (ophthalmic artery) chemotherapy with melphalan for intraocular retinoblastoma initial results. Ophthalmology. 2008 Aug;115(8):1398-404, 1404.e1. doi: 10.1016/j.ophtha.2007.12.014. Epub 2008 Mar 14.
PubMed 18342944
Study at a glance
From the registry record, last updated by the sponsor on 17 Dec 2009. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Intra-arterial Chemotherapy(Chemosurgery) for Retinoblastoma
- ClinicalTrials.gov ID
- NCT00901238
- Acronym / sponsor ID
- Chemosurgery / RTB1
- Conditions
- Retinoblastoma
- Keywords
- retinoblastoma, intra-arterial chemotherapy, chemosurgery, melphalan, topotecan
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Weill Medical College of Cornell UniversityUniversity, hospital or other
- Collaborators
- Memorial Sloan Kettering Cancer Center
- Study start
- May 2006
- Primary completion
- Jul 2009 (actual)
- Study completion
- Jul 2009 (actual)
- First posted
- 13 May 2009 (estimated)
- 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.
Conventional treatments of retinoblastoma involves laser photocoagulation, cryotherapy (freezing of the tumor), plaque radiotherapy,external beam radiotherapy, and intravenous chemotherapy. Enucleation (removing of the eye)is the last option when the tumor cannot be controlled otherwise. However,many children with retinoblastoma present with advanced intraocular disease for which enucleation is the only option. Intra-arterial chemotherapy (Chemosurgery)delivers anti-tumor drug directly into the ophthalmic artery (the artery feeding the eye) in order to increase the dose of drug reaching the tumor while minimizing toxicity to the rest of the body.
Detailed description
Present treatments for intraocular retinoblastoma cure 99% of children but have significant toxicity. Enucleation of the eye is effective but blinds the eye and leaves a lifelong cosmetic deformity. Radiation is associated with the subsequent development of fatal cancers. Systemic (intravenous)chemotherapy is used worldwide but experience with it has shown that the majority of eyes initially treated with chemotherapy still require additional treatments, such as radiation, laser, cryotherapy or even enucleation. In addition blood transfusions, secondary infections, insertion of ports and permanent hearing loss are now well reported. Three years ago we developed this technique of Chemosurgery for significantly increasing the dose of drug to the cancer while decreasing the dose of drug administered to children. This approach has decreased the need for enucleation in advanced eyes scheduled for enucleation with minimal systemic toxicity. We now offer treatment of both eyes simultaneously (in bilateral cases) and to eyes with less advanced disease and normal vision as an alternative to toxic systemic chemotherapy. In cases of very advanced ocular disease we will be using multiple drugs infused at the same session to increase tumor kill.
Chemosurgery interventions are performed under general anesthesia. The femoral artery (artery at the groin) is punctured and a catheter (a small plastic tube)is advanced into the opthalmic artery (the artery of the eye)using fluoroscopic (X-ray) guidance. The drugs are injected directly into the opthalmic artery over a period of 30-45 min.The catheter is then removed, manual compression exerted to the femoral artery, the child is awaken and goes to recovery for 6 hours. The procedure is repeated every 3-4 weeks for a total of 2 to 6 sessions according to tumor response. Since April 2006, our center has treated by chemosurgery 60 eyes in 52 patients with advanced intra-ocular retinoblastoma for which enucleation was considered.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 1 month and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- advanced retinoblastoma in one or both eyes
- recurrent retinoblastoma after failure of conventional methods
Key exclusion criteria (2 listed)
- retinoblastoma judged curable by conventional methods
- patient judged unable to undergo the procedure
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 |
|---|---|---|
| Intra-arterial ChemotherapyAlso: chemosurgeryselective catheterization of the ophthalmic artery and injection of melphalan and topotecan | Procedure or surgery | – |
Primary outcome
tumor control
Time frame: 6 month
1 secondary outcome
tumor control with vision
Time frame: 6 month
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Dec 2009.
- Background: PubMed 19169884Brodie SE, Pierre Gobin Y, Dunkel IJ, Kim JW, Abramson DH. Persistence of retinal function after selective ophthalmic artery chemotherapy infusion for retinoblastoma. Doc Ophthalmol. 2009 Aug;119(1):13-22. doi: 10.1007/s10633-008-9164-3. Epub 2009 Jan 25.
- Results publication: PubMed 18342944Abramson DH, Dunkel IJ, Brodie SE, Kim JW, Gobin YP. A phase I/II study of direct intraarterial (ophthalmic artery) chemotherapy with melphalan for intraocular retinoblastoma initial results. Ophthalmology. 2008 Aug;115(8):1398-404, 1404.e1. doi: 10.1016/j.ophtha.2007.12.014. Epub 2008 Mar 14.
- RetinoblastomaLink provided by the sponsor.
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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. This study is not recruiting at the moment.
David H Abramson, MD principal investigator
Weill Cornell Medical College and Memorial Sloan-Kettering Cancer Center
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Intra-arterial Chemotherapy in people with Retinoblastoma, run by Weill Medical College of Cornell University at 2 sites in United States.
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 2 inclusion and 2 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: finished, as of the sponsor's last update on 17 Dec 2009. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. 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.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
tumor control (time frame: 6 month). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00901238, which also lists 2 linked publications.
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 17 Dec 2009.