RetinoblastomaPhase 2NCT00179920
Chemotherapy Treatment for Children With Intraocular Germ-Line Retinoblastoma
A Phase 2 study testing Carboplatin, VP-16 and Local Surgery in people with Retinoblastoma and Retinal Neoplasms, run by Ann & Robert H Lurie Children's Hospital of Chicago at 1 site in United States.
Trial summary
Retinoblastoma is an unusual cancer of early childhood involving tumor is both eyes or, in certain circumstances, one eye only. This condition is the result of an abnormal gene which makes both retinas (the back of the eye) vulnerable to develop multiple tumors. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Retinoblastoma, Retinal Neoplasms
- What is being tested?
- Carboplatin, VP-16 and Local Surgery
- Type of treatment
- Surgery or procedure
- How many people
- 30 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 1996to Sep 2006
- Phase
- Phase 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Eligibility Criteria:
- Initial clinical diagnosis of Retinoblastoma, with Bilateral tumors, OR Unilateral tumor with hope for salvage of vision AND Multiple primary tumors, or positive family history for retinoblastoma, or age < 12 months
- No prior antitumor therapy (except enucleation of one eye)
- At least one tumor exceeds 6 mm in diameter
- No evidence of dissemination outside the globe
- Signed IRB-approved informed consent
- Patient/family available for follow-up
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Memorial Hospital
Chicago, Illinois, 60614
About this study
Official title: Chemotherapy Plus Local Surgical Treatment in Children With Intraocular Germ-Line Retinoblastoma
Retinoblastoma is an unusual cancer of early childhood involving tumor is both eyes or, in certain circumstances, one eye only. This condition is the result of an abnormal gene which makes both retinas (the back of the eye) vulnerable to develop multiple tumors. Growths in the eye impair vision temporarily or permanently. These tumors are malignant, which means that they can grow within the eye, spread outside of the eye, and be fatal if untreated.
Standard therapy for bilateral retinoblastoma includes removal of one eye if vision cannot be save and radiation treatment of either eye in which vision might be saved. Radiation controls tumor growth in the majority of cases. Another standard method is cryotherapy (freezing a tumor to kill it). Chemotherapy (medicines used to kill tumor cells) has been used in the past for tumor in or outside the eye, but is not standard. Hyperthermia, increasing the temperature of a tumor to kill it, is widely performed, and can be done to a retinoblastoma tumor by a laser; this method is not standard.
Read more
The problem with removal of an eye is that any hope of vision is lost. The problems with radiation include incomplete control of tumor, injury to the eye or surrounding tissue with decreased growth, and that (due to the abnormal retinoblastoma gene) children are very susceptible to develop other tumors, especially in the tissue which was given radiation.
The doctors at Children's Memorial Hospital are using a newer form of treatment, including laser hyperthermia, chemotherapy and cryotherapy to decrease retinoblastoma tumors. Some may be controlled indefinitely, reducing the number of eyes that need radiation or removal.
OBJECTIVES
- To find out how well chemotherapy plus cryotherapy and laser hyperthermia work on retinoblastoma tumors.
- To find out whether vision can be saved and tumors controlled without radiation or removal of the eye.
- Conditions
- Retinoblastoma, Retinal Neoplasms
- Being tested
- Carboplatin, VP-16, Local Surgery, Cryotherapy, Laser hyperthermia
- Medical subject headings
- Retinoblastoma, Retinal Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CarboplatinDrug
VP-16Drug
Local SurgeryProcedure or surgery
CryotherapyProcedure or surgery
Laser hyperthermiaProcedure or surgery
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine if a multimodal approach can provide durable control of retinoblastoma and specifically:
Estimate the proportion of eyes in which radiation therapy can be omitted
Estimate the proportion of eyes in which vision can be retained
Secondary outcomes (5)
To estimate the response rate and duration for primary retinoblastoma tumors to combination therapy with carboplatin and VP-16
To estimate the response rate of qualifying in situ retinoblastoma tumors to local surgical therapies, specifically estimate tumor response and duration after laser hyperthermia concurrent with carboplatin
Estimate tumor response and duration after cryotherapy
To estimate the control rate of vitreous involvement by retinoblastoma to carboplatin and VP-16
To detect any toxicities resulting from multimodal therapy plus local surgical options
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Sep 2005 (estimated)
- Study startApr 1996
- Study completionSep 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for RetinoblastomaStudies of the same condition, open and under way.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants
Sponsor and investigators
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Stewart Goldman, Ann & Robert H Lurie Children's Hospital of Chicago
- Investigators
- Stewart Goldman, MD, Principal investigator, Ann & Robert H Lurie Children's Hospital of Chicago
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00179920
- Sponsor's protocol ID
- CNS 0294
- Status last verified
- Aug 2020
- First submitted
- 12 Sep 2005
- Last updated
- 5 Aug 2020
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Aug 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Chemotherapy Plus Local Surgical Treatment in Children With Intraocular Germ-Line Retinoblastoma
- ClinicalTrials.gov ID
- NCT00179920
- Acronym / sponsor ID
- CNS 0294
- Conditions
- Retinoblastoma, Retinal Neoplasms
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of ChicagoUniversity, hospital or other
- Study start
- Apr 1996
- Study completion
- Sep 2006
- First posted
- 16 Sep 2005 (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.
Retinoblastoma is an unusual cancer of early childhood involving tumor is both eyes or, in certain circumstances, one eye only. This condition is the result of an abnormal gene which makes both retinas (the back of the eye) vulnerable to develop multiple tumors. Growths in the eye impair vision temporarily or permanently. These tumors are malignant, which means that they can grow within the eye, spread outside of the eye, and be fatal if untreated.
Standard therapy for bilateral retinoblastoma includes removal of one eye if vision cannot be save and radiation treatment of either eye in which vision might be saved. Radiation controls tumor growth in the majority of cases. Another standard method is cryotherapy (freezing a tumor to kill it). Chemotherapy (medicines used to kill tumor cells) has been used in the past for tumor in or outside the eye, but is not standard. Hyperthermia, increasing the temperature of a tumor to kill it, is widely performed, and can be done to a retinoblastoma tumor by a laser; this method is not standard.
The problem with removal of an eye is that any hope of vision is lost. The problems with radiation include incomplete control of tumor, injury to the eye or surrounding tissue with decreased growth, and that (due to the abnormal retinoblastoma gene) children are very susceptible to develop other tumors, especially in the tissue which was given radiation.
The doctors at Children's Memorial Hospital are using a newer form of treatment, including laser hyperthermia, chemotherapy and cryotherapy to decrease retinoblastoma tumors. Some may be controlled indefinitely, reducing the number of eyes that need radiation or removal.
OBJECTIVES
- To find out how well chemotherapy plus cryotherapy and laser hyperthermia work on retinoblastoma tumors.
- To find out whether vision can be saved and tumors controlled without radiation or removal of the eye.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
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 |
|---|---|---|
| Carboplatin | Drug | – |
| VP-16 | Drug | – |
| Local Surgery | Procedure or surgery | – |
| Cryotherapy | Procedure or surgery | – |
| Laser hyperthermia | Procedure or surgery | – |
Primary outcome
To determine if a multimodal approach can provide durable control of retinoblastoma and specifically:
Estimate the proportion of eyes in which radiation therapy can be omitted
Estimate the proportion of eyes in which vision can be retained
5 secondary outcomes
To estimate the response rate and duration for primary retinoblastoma tumors to combination therapy with carboplatin and VP-16
To estimate the response rate of qualifying in situ retinoblastoma tumors to local surgical therapies, specifically estimate tumor response and duration after laser hyperthermia concurrent with carboplatin
Estimate tumor response and duration after cryotherapy
To estimate the control rate of vitreous involvement by retinoblastoma to carboplatin and VP-16
To detect any toxicities resulting from multimodal therapy plus local surgical options
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 5 Aug 2020.
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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.
Stewart Goldman, MD principal investigator
Ann & Robert H Lurie Children's Hospital of Chicago
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Carboplatin, VP-16 and Local Surgery in people with Retinoblastoma and Retinal Neoplasms, run by Ann & Robert H Lurie Children's Hospital of Chicago at 1 site in United States.
The full registered description is in the About this study section and the official record.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Aug 2020. 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 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 phase mean?
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?
To determine if a multimodal approach can provide durable control of retinoblastoma and specifically:. The study lists 3 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 5 Aug 2020.