Treatment Protocol for Non-Metastatic Unilateral Retinoblastoma

Study Purpose

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.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Interventional
Eligible Ages N/A - 6 Years
Gender All
More Inclusion & Exclusion Criteria

Inclusion Criteria:

  • - 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.

Exclusion Criteria:

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

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT03475121
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Phase 3
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Hospital JP Garrahan
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

N/A
Principal Investigator Affiliation N/A
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Other
Overall Status Recruiting
Countries Argentina
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Unilateral Retinoblastoma
Additional Details

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).

Arms & Interventions

Arms

Experimental: Low Risk Patients

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

Experimental: Higher Risk Patients

Patients with IRSS stage I, pT3b, pT3c, pT3d will receive 6 cycles of adjuvant chemotherapy plus 6 doses of intrathecal topotecan

Experimental: Stage II Patients

Patients with Stage II (pT4) will receive 6 cycles of adjuvant chemotherapy plus 6 doses of intrathecal topotecan and orbital radiotherapy

Experimental: Patients with buphthalmus

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.

Interventions

Combination Product: - Combination Chemotherapy plus Intrathecal Topotecan

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

Combination Product: - Higher Dose Combination Chemotherapy plus Intrathecal Topotecan

GALOP I-based systemic adjuvant therapy plus intrathecal topotecan

Combination Product: - Higher Dose Combination Chemotherapy plus Intrathecal Topotecan and Orbital Radiotherapy

GALOP I-based systemic adjuvant therapy plus intrathecal topotecan and orbital radiotherapy (45 Gy) up to chiasm

Other: - No Adjuvant Therapy

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

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

International Sites

Hospital JP Garrahan, Buenos Aires, CF, Argentina

Status

Recruiting

Address

Hospital JP Garrahan

Buenos Aires, CF, C1245AAL

Site Contact

Carolina Cernadas, MD

coordinacioninvestigacion@garrahan.gov.ar

541141226335

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