RetinoblastomaRELRB1NCT00980551
Pilot Study of Topotecan/Vincristine With Subconjunctival Carboplatin for Patients With Bilateral Retinoblastoma
A study testing subtenon Carboplatin, Topotecan Hydrochloride and Vincristine in people with Retinoblastoma, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.
Trial summary
The investigators are testing subtenon carboplatin in combination with vincristine and topotecan given by vein in the hopes of finding a drug combination that may be effective against retinoblastoma that has come back (recurrent) or is resistant to other treatment (refractory). Read more about this study
- Who is it for?
- Ages Up to 10 years
- Conditions the study is for
- Retinoblastoma
- What is being tested?
- subtenon Carboplatin, Topotecan Hydrochloride and Vincristine
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- May 2010to Mar 2012
- Phase
- Interventional
- 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 Up to 10 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age: ≤ 10 years of age
- Diagnosis: Patients must have a history of bilateral Retinoblastoma AND Recurrent/Refractory Intra-Ocular Retinoblastoma considered not amenable to cure with local therapies alone (photocoagulation with argon laser, cryotherapy, transpupillary thermotherapy, radioactive plaque therapy).
- Therapeutic Options: Patient's disease status is one for which there are no known options proven to provide a high chance for ocular salvage or cure other than external beam radiation or enucleation.
- Remaining visual function in target eye (s) is required
- Life Expectancy of > 8 weeks
- Lansky ≥ 50
Show all 21Show fewer
- Prior Therapy: Patients must have local relapsed/refractory disease after receiving standard upfront therapy involving at least one chemotherapeutic regimen. There is no limit to prior chemotherapeutic regimens permitted. Patients may have received previous intravenous carboplatin, but may not have received prior subtenon carboplatin or intravenous topotecan.
- Patients must have recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study, as described below:
- Myelosuppressive chemotherapy: patients must not have received myelosuppressive chemotherapy within 3 weeks of study enrollment
- Biologic therapies: Patients must not have received biologic anti-cancer agents within one week of study enrollment
- Radiation therapy: Four weeks must have elapsed since external beam radiation therapy, if given.
- Adequate Bone Marrow Function Defined as:
- Peripheral absolute neutrophil count (ANC) ≥ 750/µL
- Platelet count ≥ 75,000/µL (transfusion independent, defined as not receiving platelet transfusions within a 7-day period prior to enrollment)
- Hemoglobin ≥ 8.0 gm/dL (may receive RBC transfusions)
- Adequate Renal Function Defined as:
- Creatinine clearance or radioisotope GFR ≥ 70ml/min/1.73 m2 OR threshold creatinine values based on age/gender derived from the Schwartz formula for estimating GFR
- Adequate Liver Function Defined As:
- Bilirubin (sum of conjugated + unconjugated) ≤ 1.5 x upper limit of normal (ULN) for age
- SGPT (ALT) ≤ 5 x upper limit of normal (ULN) for age
- Serum albumin ≥ 2 g/dL
Who cannot join
- Extra-ocular retinoblastoma
- Asynchronous involvement of the contralateral eye, previously untreated
- Uncontrolled infection at time of protocol entry
- Concomitant Medications:
- Growth factors that support platelet or white cell number or function must not have been administered within the past 3 days
- Patients who are currently receiving investigational drugs, or who have received an investigational drug within the last 7 days, are ineligible
- Patients who are currently receiving other anti-cancer agents are ineligible
- Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible
Trial locations and contacts
1 site in 1 country.
United States1 site
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
About this study
Official title: A Pilot Study of Intravenous Topotecan and Vincristine in Combination With Subconjunctival Carboplatin for Patients With a History of Bilateral Retinoblastoma and Refractory/Recurrent Intraocular Disease (IND# 104,942)
The investigators are testing subtenon carboplatin in combination with vincristine and topotecan given by vein in the hopes of finding a drug combination that may be effective against retinoblastoma that has come back (recurrent) or is resistant to other treatment (refractory).
The goals of this study are:
Read more
- To decide if the drug combination is a useful treatment for recurrent or refractory retinoblastoma
- To test the safety of the drug combination and to see what kind of effects (good and bad) can be expected from the drug combination
- To measure visual changes before and after the study therapy
- To use a special MRI scan to measure brain function involved in vision processing, both before and after the study therapy
In this study, the investigators are also testing a new experimental way of giving carboplatin "subtenon carboplatin". The carboplatin will be given directly in the eye through a needle placed under the covering of the eye. This is to try to get more carboplatin to the retinoblastoma inside the eye.
- Conditions
- Retinoblastoma
- Being tested
- subtenon Carboplatin, Topotecan Hydrochloride, Vincristine, Filgrastim
- Medical subject headings
- Retinoblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
subtenon CarboplatinDrug
Also called Paraplatin
Available in 50mg, 150mg, 450 mg, 600mg vials. It is provided as a premixed aqueous solution or lyophilized powder for injection given on day 1/week 10 (cycles 1-3).
Topotecan HydrochlorideDrug
Also called Hycamtin
Each vial contains topotecan hydrochloride equivalent to 4 mg of topotecan as free base. Given intravenously on days 1-5 of each week (cycles 1-6).
VincristineDrug
Also called Oncovin
Each vial contains vincristine sulfate, 1 mg; mannitol, 100 mg; sterile water for injection; Acetic acid and sodium acetate are added for pH control. Given intravenously on day 1/week 1 (cycles 1-6).
FilgrastimDrug
Also called Neupogen
Supplied as a clear solution in 300 mcg/ml 1 ml or 1.6 ml vials and prefilled syringes containing 300mcg/0.5mL or 480mcg/0.8mL. Given intravenously on day 6 of each week (cycles 1-6).
| Study group | Role | What they receive |
|---|---|---|
| Topotecan/Vincristine with subtenon Carboplatin | Experimental | Not described Receives: Drug: subtenon Carboplatin; Drug: Topotecan Hydrochloride; Drug: Vincristine; Drug: Filgrastim |
What is being measured
The primary outcome is the main question the study is designed to answer.
To estimate the event-free survival, where an event is defined at the ocular level as the need for non-protocol therapy defined as additional non-protocol chemotherapy, external beam radiation, or enucleation.
Measured: at 1 year
To estimate the patterns of response, and response rate of locally refractory retinoblastoma to combination intravenous and peri-ocular chemotherapy.
Measured: at 1 yr
Secondary outcomes (4)
To describe the toxicities associated with the proposed regimen.
Measured: at 1 yr
To describe the histologic findings in the eyes ultimately requiring enucleation.
Measured: at 1 yr
To describe the visual acuity of patients with advanced intraocular retinoblastoma before and after the proposed therapy.
Measured: at 1 yr
To describe primary visual cortex function in patients with retinoblastoma via functional Magnetic Resonance Imaging (fMRI) technique both before and after the proposed therapy.
Measured: at 1 yr
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Sep 2009 (estimated)
- Study startMay 2010
- Primary completionMar 2012
- Study completionMar 2012
Why it stopped: No enrollment and competing studies
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.
- More trials run by Children's Hospital Medical Center, CincinnatiThe 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
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- James Geller, MD, Principal investigator, Children's Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00980551
- Sponsor's protocol ID
- RELRB1
- Acronym
- RELRB1
- Status last verified
- Feb 2017
- First submitted
- 18 Sep 2009
- Last updated
- 3 Mar 2017
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 3 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study of Intravenous Topotecan and Vincristine in Combination With Subconjunctival Carboplatin for Patients With a History of Bilateral Retinoblastoma and Refractory/Recurrent Intraocular Disease (IND# 104,942)
- ClinicalTrials.gov ID
- NCT00980551
- Acronym / sponsor ID
- RELRB1 / RELRB1
- Conditions
- Retinoblastoma
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: No enrollment and competing studies
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Study start
- May 2010 (actual)
- Primary completion
- Mar 2012 (actual)
- Study completion
- Mar 2012 (actual)
- First posted
- 21 Sep 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.
The investigators are testing subtenon carboplatin in combination with vincristine and topotecan given by vein in the hopes of finding a drug combination that may be effective against retinoblastoma that has come back (recurrent) or is resistant to other treatment (refractory).
The goals of this study are:
- To decide if the drug combination is a useful treatment for recurrent or refractory retinoblastoma
- To test the safety of the drug combination and to see what kind of effects (good and bad) can be expected from the drug combination
- To measure visual changes before and after the study therapy
- To use a special MRI scan to measure brain function involved in vision processing, both before and after the study therapy
In this study, the investigators are also testing a new experimental way of giving carboplatin "subtenon carboplatin". The carboplatin will be given directly in the eye through a needle placed under the covering of the eye. This is to try to get more carboplatin to the retinoblastoma inside the eye.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Topotecan/Vincristine with subtenon Carboplatin | Experimental | Drug: subtenon Carboplatin, Drug: Topotecan Hydrochloride, Drug: Vincristine, Drug: Filgrastim |
Who may qualify
- Ages
- Up to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (21 listed)
- Age: ≤ 10 years of age
- Diagnosis: Patients must have a history of bilateral Retinoblastoma AND Recurrent/Refractory Intra-Ocular Retinoblastoma considered not amenable to cure with local therapies alone (photocoagulation with argon laser, cryotherapy, transpupillary thermotherapy, radioactive plaque therapy).
- Therapeutic Options: Patient's disease status is one for which there are no known options proven to provide a high chance for ocular salvage or cure other than external beam radiation or enucleation.
- Remaining visual function in target eye (s) is required
- Life Expectancy of > 8 weeks
Key exclusion criteria (8 listed)
- Extra-ocular retinoblastoma
- Asynchronous involvement of the contralateral eye, previously untreated
- Uncontrolled infection at time of protocol entry
- Concomitant Medications:
- Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible
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 |
|---|---|---|
| subtenon CarboplatinAlso: ParaplatinAvailable in 50mg, 150mg, 450 mg, 600mg vials. It is provided as a premixed aqueous solution or lyophilized powder for injection given on day 1/week 10 (cycles 1-3). | Drug | Topotecan/Vincristine with subtenon Carboplatin |
| Topotecan HydrochlorideAlso: HycamtinEach vial contains topotecan hydrochloride equivalent to 4 mg of topotecan as free base. Given intravenously on days 1-5 of each week (cycles 1-6). | Drug | Topotecan/Vincristine with subtenon Carboplatin |
| VincristineAlso: OncovinEach vial contains vincristine sulfate, 1 mg; mannitol, 100 mg; sterile water for injection; Acetic acid and sodium acetate are added for pH control. Given intravenously on day 1/week 1 (cycles 1-6). | Drug | Topotecan/Vincristine with subtenon Carboplatin |
| FilgrastimAlso: NeupogenSupplied as a clear solution in 300 mcg/ml 1 ml or 1.6 ml vials and prefilled syringes containing 300mcg/0.5mL or 480mcg/0.8mL. Given intravenously on day 6 of each week (cycles 1-6). | Drug | Topotecan/Vincristine with subtenon Carboplatin |
Primary outcome
To estimate the event-free survival, where an event is defined at the ocular level as the need for non-protocol therapy defined as additional non-protocol chemotherapy, external beam radiation, or enucleation.
Time frame: at 1 year
To estimate the patterns of response, and response rate of locally refractory retinoblastoma to combination intravenous and peri-ocular chemotherapy.
Time frame: at 1 yr
4 secondary outcomes
To describe the toxicities associated with the proposed regimen.
Time frame: at 1 yr
To describe the histologic findings in the eyes ultimately requiring enucleation.
Time frame: at 1 yr
To describe the visual acuity of patients with advanced intraocular retinoblastoma before and after the proposed therapy.
Time frame: at 1 yr
To describe primary visual cortex function in patients with retinoblastoma via functional Magnetic Resonance Imaging (fMRI) technique both before and after the proposed therapy.
Time frame: at 1 yr
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Mar 2017.
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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.
James Geller, MD principal investigator
Children's Hospital Medical Center, Cincinnati
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing subtenon Carboplatin, Topotecan Hydrochloride and Vincristine in people with Retinoblastoma, run by Children's Hospital Medical Center, Cincinnati at 1 site 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 21 inclusion and 8 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: withdrawn, as of the sponsor's last update on 3 Mar 2017. 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.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
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 estimate the event-free survival, where an event is defined at the ocular level as the need for non-protocol therapy defined as additional non-protocol chemotherapy, external beam radiation, or enucleation. (time frame: at 1 year). The study lists 2 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 3 Mar 2017.