RetinoblastomaPhase 2NCT01857752
Phase II Study Temozolomide for Retinoblastoma Metastatic to the Central Nervous System for Patients From Guatemala
A Phase 2 study testing Temozolomide in people with Retinoblastoma, run by Stanford University at 1 site in Guatemala.
Trial summary
Primary Objectives: 1. To investigate the response rate (complete response plus partial response) of temozolomide for 8 weeks (2 cycles), in patients with retinoblastoma metastatic to the central nervous system (mass only) in two strata: A. Initial diagnosis (mass) B. At relapse (mass) 2. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Retinoblastoma
- What is being tested?
- Temozolomide
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteGuatemala
- Study dates
- Mar 2012to Jun 2015
- 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 Up to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- This study must be evaluated and confirmed by the local ethics committees and institutional review board of the participating institution, in accordance to the declaration of Helsinki. Informed consent must be administered and the parent or guardian must sign the document, authorized by the Ethics Committee and human subjects (therapy can not start if the documents are not signed).
- Patients less than 21 years of age with diagnosis of retinoblastoma with metastasis to the CNS (central nervous system) must be confirmed by an ophthalmologist and/or a pathologist; in conjunction with the pediatric oncologist.
- Metastasis to the CNS is defined as a mass in the chiasm or other site in the CNS, confirmed by MRI and/or
- CT:
- At diagnosis
- At relapse after conventional therapy.
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- It is acceptable to have other sites of metastatic disease: lymphatic, bone, bone marrow or others.
- All sites of metastasis must be measured in 3 planes, in millimeters and described in POND (Pediatric Oncology Network Database*)
- Quality of life must be 3 by Lansky and 50% by Karnofksy
- Hemogram: Hemoglobin of 7, ANC of 750, platelets of 75K.
- Chemistry: direct bilirubin less than 3.0, indirect bilirubin less than 4.0, AST less 5x normal and ALT 5x normal, creatinine less than 1.5.
- Treatment must start not more than 15 days from diagnosis of metastatic retinoblastoma.
- Every patient with relapse or progression into the CNS must be documented with CT scan or MRI of the brain. Other sites of relapse may be evaluated, including bone marrow.
- Patients who are newly diagnosed should have not received any other chemotherapeutic therapy (with the exception of dexamethasone) 1 week before starting, or radiotherapy, 4 weeks before starting.
- All patients to be included in this study must be presented to the principal investigator using Horizon Live Web-conferencing through the Cure4Kids website. Eligibility and target CNS sites will be determined, as well as non-target sites.
Who cannot join
- Patients with metastatic to CNS with only CSF involvement are NOT ELIGIBLE.
- Relapse patients should have NOT received chemotherapy for 4 weeks, and no patient should have received nitroureas (melphalan, CCNU or mustard). No patient should have received radiation therapy in the previous 42 days. These patients are NOT eligible.
- Diagnosis of AIDS or HIV positive.
- Patients with disease NOT in the CNS are NOT eligible
Trial locations and contacts
1 site in 1 country.
About this study
Primary Objectives:
- To investigate the response rate (complete response plus partial response) of temozolomide for 8 weeks (2 cycles), in patients with retinoblastoma metastatic to the central nervous system (mass only) in two strata:
- A. Initial diagnosis (mass)
- B. At relapse (mass)
- To determine hematologic toxicity: absolute neutrophil count (ANC), platelets and hemoglobin count.
Secondary Objectives:
- To determine the response in other metastatic sites (non target) (orbit, bone marrow, bone, lung, liver and others)
- To determine the remission rate and time to relapse on temozolomide.
- To document the response of leptomeningeal metastasis (cerebrospinal fluid) to temozolomide
- Conditions
- Retinoblastoma
- Being tested
- Temozolomide
- Keywords
- Retinoblastoma
- Medical subject headings
- Retinoblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TemozolomideDrug
| Study group | Role | What they receive |
|---|---|---|
| temozolomide | Experimental | Not described Receives: Drug: Temozolomide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Response rate
Response rate (complete response plus partial response) of temozolomide for 8 weeks (2 cycles), in patients with retinoblastoma metastatic to the central nervous system (mass only) in two strata:
- Initial diagnosis (mass)
- At relapse (mass)
Measured: 8 weeks
Evaluate the toxicity suffered during each cycle of temozolamide will be evaluated using the CTCAE v3.0
Measured: 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 May 2013 (estimated)
- Study startMar 2012
- Primary completionJun 2015
- Study completionJun 2015
Why it stopped: accrual
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 Stanford 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stanford University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Sandra Luna-Fineman, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01857752
- Sponsor's protocol ID
- PEDSEYE0003
- Other IDs
- 22753
- Status last verified
- Jan 2017
- First submitted
- 25 Mar 2013
- Last updated
- 30 Jan 2017
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Jan 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study Temozolomide for Retinoblastoma Metastatic to the Central Nervous System for Patients From Guatemala
- ClinicalTrials.gov ID
- NCT01857752
- Acronym / sponsor ID
- PEDSEYE0003
- Conditions
- Retinoblastoma
- Keywords
- Retinoblastoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: accrual
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Stanford UniversityUniversity, hospital or other
- Study start
- Mar 2012 (actual)
- Primary completion
- Jun 2015 (actual)
- Study completion
- Jun 2015 (actual)
- First posted
- 20 May 2013 (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.
Primary Objectives:
- To investigate the response rate (complete response plus partial response) of temozolomide for 8 weeks (2 cycles), in patients with retinoblastoma metastatic to the central nervous system (mass only) in two strata:
- A. Initial diagnosis (mass)
- B. At relapse (mass)
- To determine hematologic toxicity: absolute neutrophil count (ANC), platelets and hemoglobin count.
Secondary Objectives:
- To determine the response in other metastatic sites (non target) (orbit, bone marrow, bone, lung, liver and others)
- To determine the remission rate and time to relapse on temozolomide.
- To document the response of leptomeningeal metastasis (cerebrospinal fluid) to temozolomide
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| temozolomide | Experimental | Drug: Temozolomide |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (15 listed)
- This study must be evaluated and confirmed by the local ethics committees and institutional review board of the participating institution, in accordance to the declaration of Helsinki. Informed consent must be administered and the parent or guardian must sign the document, authorized by the Ethics Committee and human subjects (therapy can not start if the documents are not signed).
- Patients less than 21 years of age with diagnosis of retinoblastoma with metastasis to the CNS (central nervous system) must be confirmed by an ophthalmologist and/or a pathologist; in conjunction with the pediatric oncologist.
- Metastasis to the CNS is defined as a mass in the chiasm or other site in the CNS, confirmed by MRI and/or
- It is acceptable to have other sites of metastatic disease: lymphatic, bone, bone marrow or others.
- All sites of metastasis must be measured in 3 planes, in millimeters and described in POND (Pediatric Oncology Network Database*)
Key exclusion criteria (4 listed)
- Patients with metastatic to CNS with only CSF involvement are NOT ELIGIBLE.
- Relapse patients should have NOT received chemotherapy for 4 weeks, and no patient should have received nitroureas (melphalan, CCNU or mustard). No patient should have received radiation therapy in the previous 42 days. These patients are NOT eligible.
- Diagnosis of AIDS or HIV positive.
- Patients with disease NOT in the CNS 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 |
|---|---|---|
| Temozolomide | Drug | temozolomide |
Primary outcome
Response rate
Time frame: 8 weeks
Response rate (complete response plus partial response) of temozolomide for 8 weeks (2 cycles), in patients with retinoblastoma metastatic to the central nervous system (mass only) in two strata: 1. Initial diagnosis (mass) 2. At relapse (mass)
Evaluate the toxicity suffered during each cycle of temozolamide will be evaluated using the CTCAE v3.0
Time frame: 8 weeks
Results and safety
The study was stopped early (reason given: accrual). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Jan 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.
Sandra Luna-Fineman principal investigator
Stanford University
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 Temozolomide in people with Retinoblastoma, run by Stanford University at 1 site in Guatemala.
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 15 inclusion and 4 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: stopped early, as of the sponsor's last update on 30 Jan 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 Guatemala. 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 was stopped early (reason given: accrual). Results are not available in the source record.
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?
Response rate (time frame: 8 weeks). 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 30 Jan 2017.