NCT03475121

Brief 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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
84

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Jan 2018

Longer than P75 for phase_3

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 1, 2018

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

March 12, 2018

Completed
11 days until next milestone

First Posted

Study publicly available on registry

March 23, 2018

Completed
4.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 31, 2023

Completed
2.2 years until next milestone

Study Completion

Last participant's last visit for all outcomes

March 28, 2025

Completed
Last Updated

April 13, 2025

Status Verified

April 1, 2025

Enrollment Period

5.1 years

First QC Date

March 12, 2018

Last Update Submit

April 10, 2025

Conditions

Unilateral Retinoblastoma

Outcome Measures

Primary Outcomes (2)

  • Rate of extraocular relapses

    Number of participants experiencing extraocular relapse

    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

    5 years

Study Arms (4)

Low Risk Patients

EXPERIMENTAL

Patients with IRSS stage I, pT1, pT2 and pT3 stage will not receive adjuvant therapy

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

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

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.

Combination Product: Higher Dose Combination Chemotherapy plus Intrathecal Topotecan

Interventions

Combination Chemotherapy plus Intrathecal TopotecanCOMBINATION_PRODUCT

Adjuvant chemotherapy with a reduced dose (compared to GALOP I protocol) of carboplatin

Higher Risk Patients
Higher Dose Combination Chemotherapy plus Intrathecal TopotecanCOMBINATION_PRODUCT

GALOP I-based systemic adjuvant therapy plus intrathecal topotecan

Patients with buphthalmus
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

Stage II Patients
No Adjuvant TherapyOTHER

Patients will not receive any adjuvant therapy after enucleation of the affected eye.

Low Risk Patients

Eligibility Criteria

Age0 Years - 6 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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

You may not qualify if:

  • Patients with unilateral retinoblastoma and germline mutations of the Rb1 gene or family history for retinoblastoma

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Hospital JP Garrahan

Buenos Aires, Buenos Aires F.D., C1245AAL, Argentina

Location

MeSH Terms

Interventions

Drug Therapy, Combination

Intervention Hierarchy (Ancestors)

Drug TherapyTherapeutics

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER GOV
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
MD, Head Hematology and Oncology Department

Study Record Dates

First Submitted

March 12, 2018

First Posted

March 23, 2018

Study Start

January 1, 2018

Primary Completion

January 31, 2023

Study Completion

March 28, 2025

Last Updated

April 13, 2025

Record last verified: 2025-04

Data Sharing

IPD Sharing
Will not share

Locations

AR(1)