Unilateral RetinoblastomaPhase 3RbGALOP2NCT03475121
Treatment Protocol for Non-Metastatic Unilateral Retinoblastoma
A Phase 3 study testing Combination Chemotherapy plus Intrathecal Topotecan, Higher Dose Combination Chemotherapy plus Intrathecal Topotecan and Higher Dose Combination Chemotherapy plus Intrathecal Topotecan and Orbital Radiotherapy in people with Unilateral Retinoblastoma, run by Hospital JP Garrahan at 1 site in Argentina.
Trial summary
This protocol provides guidelines for the management of non-metastatic unilateral retinoblastoma and introduces an innovative adjuvant therapy for higher risk patients based upon the results of the Grupo de America Latina de Oncologia Pediatrica (GALOP) I study. Conservative therapy will be not protocolized. Read more about this study
- Who is it for?
- Ages 0 to 6 years
- Conditions the study is for
- Unilateral Retinoblastoma
- What is being tested?
- Combination Chemotherapy plus Intrathecal Topotecan, Higher Dose Combination Chemotherapy plus Intrathecal Topotecan and Higher Dose Combination Chemotherapy plus Intrathecal Topotecan and Orbital Radiotherapy
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 84 participants
- Where
- 1 siteArgentina
- Study dates
- 1 Jan 2018to 28 Mar 2025
- Phase
- Phase 3
- 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 0 to 6 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Histological diagnosis of retinoblastoma confirmed at participating institutions
- Ophthalmological diagnosis of unilateral retinoblastoma in patients undergoing conservative therapy.
- No prior therapy for retinoblastoma
- Lansky Performance Scale greater or equal to 50
- Normal organ function in those patients assigned for chemotherapy
- Signed informed consent
Who cannot join
- Patients with unilateral retinoblastoma and germline mutations of the Rb1 gene or family history for retinoblastoma
Trial locations and contacts
1 site in 1 country.
Argentina1 site
Hospital JP Garrahan
Buenos Aires, Buenos Aires F.D., C1245AAL
About this study
Official title: GALOP II Protocol for the Treatment of Unilateral Retinoblastoma
This protocol provides guidelines for the management of non-metastatic unilateral retinoblastoma and introduces an innovative adjuvant therapy for higher risk patients based upon the results of the Grupo de America Latina de Oncologia Pediatrica (GALOP) I study. Conservative therapy will be not protocolized.
Read the detailed description
Patients with non-metastatic retinoblastoma undergoing enucleation will be staged using the American Joint Committee on Cancer (AJCC), version 8, Tumor, Node, Metastasis (TNM)- H system and the International Retinoblastoma Staging System (IRSS). IRSS stage I patients recognized as higher risk will be assigned for adjuvant therapy. Those with standard risk will not receive adjuvant therapy after enucleation. Higher risk patients are defined as those with pathological retrolaminar optic nerve invasion and-or any degree of scleral invasion (pT3b, pT3c, pT3d). Based on the results of the GALOP I protocol, they will receive a reduced dose adjuvant chemotherapy regimen with 3 cycles of alternating cyclophosphamide, vincristine and idarubicin alternating with another 3 cycles of carboplatin and etoposide. Six doses of intrathecal topotecan will be given. Patients presenting with severe buphthalmia (cT3c-cT3e) will receive neo-adjuvant therapy with the same intensive regimen but including a higher dose of carboplatin as per GALOP I protocol plus intrathecal topotecan and secondary enucleation followed by adjuvant chemotherapy for a total of 8 cycles. Stage II patients (pT4) will receive the same adjuvant regimen plus orbital radiotherapy (45 cGy).
- Conditions
- Unilateral Retinoblastoma
- Being tested
- Combination Chemotherapy plus Intrathecal Topotecan, Higher Dose Combination Chemotherapy plus Intrathecal Topotecan, Higher Dose Combination Chemotherapy plus Intrathecal Topotecan and Orbital Radiotherapy, No Adjuvant Therapy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Combination Chemotherapy plus Intrathecal TopotecanCombination product
Adjuvant chemotherapy with a reduced dose (compared to GALOP I protocol) of carboplatin
Higher Dose Combination Chemotherapy plus Intrathecal TopotecanCombination product
GALOP I-based systemic adjuvant therapy plus intrathecal topotecan
Higher Dose Combination Chemotherapy plus Intrathecal Topotecan and Orbital RadiotherapyCombination product
GALOP I-based systemic adjuvant therapy plus intrathecal topotecan and orbital radiotherapy (45 Gy) up to chiasm
No Adjuvant TherapyOther
Patients will not receive any adjuvant therapy after enucleation of the affected eye.
| Study group | Role | What they receive |
|---|---|---|
| Low Risk Patients | Experimental | Patients with IRSS stage I, pT1, pT2 and pT3 stage will not receive adjuvant therapy Receives: Other: No Adjuvant Therapy |
| Higher Risk Patients | Experimental | Patients with IRSS stage I, pT3b, pT3c, pT3d will receive 6 cycles of adjuvant chemotherapy plus 6 doses of intrathecal topotecan Receives: Combination Product: Combination Chemotherapy plus Intrathecal Topotecan |
| Stage II Patients | Experimental | Patients with Stage II (pT4) will receive 6 cycles of adjuvant chemotherapy plus 6 doses of intrathecal topotecan and orbital radiotherapy Receives: Combination Product: Higher Dose Combination Chemotherapy plus Intrathecal Topotecan and Orbital Radiotherapy |
| Patients with buphthalmus | Experimental | Patients with buphthalmus (cT3c, cT3e) will receive 2 cycles of neo-adjuvant chemotherapy plus 6 doses of intrathecal topotecan followed by secondary enucleation and 6 cycles of adjuvant chemotherapy. Receives: Combination Product: Higher Dose Combination Chemotherapy plus Intrathecal Topotecan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of extraocular relapses
Number of participants experiencing extraocular relapse
Measured: 3 years
Evaluation of number of patients experiencing acute, chronic and fatal toxicities
Number of participants experiencing treatment-related adverse effects and mortality as measured by CTCAE v4.0
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Mar 2018
- Study start1 Jan 2018
- Primary completion31 Jan 2023
- Study completion28 Mar 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Unilateral 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 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 84 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hospital JP Garrahan
- Sponsor type
- Other government
- Responsible party
- Principal investigator, Pedro Zubizarreta, Hospital JP Garrahan
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03475121
- Sponsor's protocol ID
- 1031. Retinoblastoma GALOP 2
- Acronym
- RbGALOP2
- Status last verified
- Apr 2025
- First submitted
- 12 Mar 2018
- Last updated
- 13 Apr 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- GALOP II Protocol for the Treatment of Unilateral Retinoblastoma
- ClinicalTrials.gov ID
- NCT03475121
- Acronym / sponsor ID
- RbGALOP2 / 1031. Retinoblastoma GALOP 2
- Conditions
- Unilateral Retinoblastoma
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 84 participants (actual)
- Lead sponsor
- Hospital JP GarrahanOther government
- Study start
- 1 Jan 2018 (actual)
- Primary completion
- 31 Jan 2023 (actual)
- Study completion
- 28 Mar 2025 (actual)
- First posted
- 23 Mar 2018 (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.
This protocol provides guidelines for the management of non-metastatic unilateral retinoblastoma and introduces an innovative adjuvant therapy for higher risk patients based upon the results of the Grupo de America Latina de Oncologia Pediatrica (GALOP) I study. Conservative therapy will be not protocolized.
Detailed description
Patients with non-metastatic retinoblastoma undergoing enucleation will be staged using the American Joint Committee on Cancer (AJCC), version 8, Tumor, Node, Metastasis (TNM)- H system and the International Retinoblastoma Staging System (IRSS). IRSS stage I patients recognized as higher risk will be assigned for adjuvant therapy. Those with standard risk will not receive adjuvant therapy after enucleation. Higher risk patients are defined as those with pathological retrolaminar optic nerve invasion and-or any degree of scleral invasion (pT3b, pT3c, pT3d). Based on the results of the GALOP I protocol, they will receive a reduced dose adjuvant chemotherapy regimen with 3 cycles of alternating cyclophosphamide, vincristine and idarubicin alternating with another 3 cycles of carboplatin and etoposide. Six doses of intrathecal topotecan will be given. Patients presenting with severe buphthalmia (cT3c-cT3e) will receive neo-adjuvant therapy with the same intensive regimen but including a higher dose of carboplatin as per GALOP I protocol plus intrathecal topotecan and secondary enucleation followed by adjuvant chemotherapy for a total of 8 cycles. Stage II patients (pT4) will receive the same adjuvant regimen plus orbital radiotherapy (45 cGy).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Low Risk Patients | Experimental | Other: No Adjuvant TherapyPatients with IRSS stage I, pT1, pT2 and pT3 stage will not receive adjuvant therapy |
| Higher Risk Patients | Experimental | Combination Product: Combination Chemotherapy plus Intrathecal TopotecanPatients with IRSS stage I, pT3b, pT3c, pT3d will receive 6 cycles of adjuvant chemotherapy plus 6 doses of intrathecal topotecan |
| Stage II Patients | Experimental | Combination Product: Higher Dose Combination Chemotherapy plus Intrathecal Topotecan and Orbital RadiotherapyPatients with Stage II (pT4) will receive 6 cycles of adjuvant chemotherapy plus 6 doses of intrathecal topotecan and orbital radiotherapy |
| Patients with buphthalmus | Experimental | Combination Product: Higher Dose Combination Chemotherapy plus Intrathecal TopotecanPatients with buphthalmus (cT3c, cT3e) will receive 2 cycles of neo-adjuvant chemotherapy plus 6 doses of intrathecal topotecan followed by secondary enucleation and 6 cycles of adjuvant chemotherapy. |
Who may qualify
- Ages
- 0 to 6 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Histological diagnosis of retinoblastoma confirmed at participating institutions
- Ophthalmological diagnosis of unilateral retinoblastoma in patients undergoing conservative therapy.
- No prior therapy for retinoblastoma
- Lansky Performance Scale greater or equal to 50
- Normal organ function in those patients assigned for chemotherapy
Key exclusion criteria (1 listed)
- Patients with unilateral retinoblastoma and germline mutations of the Rb1 gene or family history for retinoblastoma
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 |
|---|---|---|
| Combination Chemotherapy plus Intrathecal TopotecanAdjuvant chemotherapy with a reduced dose (compared to GALOP I protocol) of carboplatin | Combination product | Higher Risk Patients |
| Higher Dose Combination Chemotherapy plus Intrathecal TopotecanGALOP I-based systemic adjuvant therapy plus intrathecal topotecan | Combination product | Patients with buphthalmus |
| Higher Dose Combination Chemotherapy plus Intrathecal Topotecan and Orbital RadiotherapyGALOP I-based systemic adjuvant therapy plus intrathecal topotecan and orbital radiotherapy (45 Gy) up to chiasm | Combination product | Stage II Patients |
| No Adjuvant TherapyPatients will not receive any adjuvant therapy after enucleation of the affected eye. | Other | Low Risk Patients |
Primary outcome
Rate of extraocular relapses
Time frame: 3 years
Number of participants experiencing extraocular relapse
Evaluation of number of patients experiencing acute, chronic and fatal toxicities
Time frame: 5 years
Number of participants experiencing treatment-related adverse effects and mortality as measured by CTCAE v4.0
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 13 Apr 2025.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Combination Chemotherapy plus Intrathecal Topotecan, Higher Dose Combination Chemotherapy plus Intrathecal Topotecan and Higher Dose Combination Chemotherapy plus Intrathecal Topotecan and Orbital Radiotherapy in people with Unilateral Retinoblastoma, run by Hospital JP Garrahan at 1 site in Argentina.
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 6 inclusion and 1 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 13 Apr 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 Argentina. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
Rate of extraocular relapses (time frame: 3 years). 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 13 Apr 2025.