Treatment Protocol for Non-Metastatic Unilateral Retinoblastoma (RbGALOP2)

Hospital JP Garrahan

Status and phase: Completed; Phase 3

Conditions: Unilateral Retinoblastoma

Treatments: Combination Product: Higher Dose Combination Chemotherapy plus Intrathecal Topotecan and Orbital Radiotherapy; Combination Product: Combination Chemotherapy plus Intrathecal Topotecan; Other: No Adjuvant Therapy; Combination Product: Higher Dose Combination Chemotherapy plus Intrathecal Topotecan

Study type: Interventional

Funder types: Other

Identifiers

NCT03475121

1031. Retinoblastoma GALOP 2

Details and patient eligibility

About

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.

Full 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).

Enrollment: 84 patients

Sex: All

Ages: Under 6 years old

Volunteers: No Healthy Volunteers

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 design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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84 participants in 4 patient groups

Low Risk Patients

Experimental group

Description:

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

Treatment:

Other: No Adjuvant Therapy

Higher Risk Patients

Experimental group

Description:

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

Treatment:

Combination Product: Combination Chemotherapy plus Intrathecal Topotecan

Stage II Patients

Experimental group

Description:

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

Treatment:

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

Patients with buphthalmus

Experimental group

Description:

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.

Treatment:

Combination Product: Higher Dose Combination Chemotherapy plus Intrathecal Topotecan