Intra-arterial Chemotherapy for Retinoblastoma

Phase 1

Completed

• Conditions: Retinoblastoma
• Interventions: Drug: Melphalan

• Registration Number: NCT04342572
• Lead Sponsor: Washington University School of Medicine

Brief Summary

Children with retinoblastoma who may benefit from intra-arterial chemotherapy will receive up to 3 doses of melphalan and will be assessed for feasibility, toxicity, and response.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2020-04-08
• Study First Submitted QC: 2020-04-08
• Study First Posted: 2020-04-13
• Study Start: 2020-08-11
• Primary Completion: 2023-01-04
• Study Completion: 2025-03-21
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-09
• Last Update Posted: 2025-05-11

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 5

Inclusion Criteria

• Patients diagnosed with retinoblastoma >4 months of age16

• Patients whose other treatment options would require systemic chemotherapy, radiotherapy, or enucleation

• Patient or parent/legal guardian must sign a written informed consent

• One of a, b, or c:

  • Patients who have bilateral COG stage B, C, D, or E retinoblastoma (refer to Appendix A) who have undergone systemic chemotherapy without resolution (meaning either has not had CR or has progressed despite systemic chemotherapy) and would have the following treatment options remaining:

    • IAC
    • enucleation of one eye
    • local radiation

  • Patients with non-germline retinoblastoma with unilateral disease who have COG A, B, C, or D tumors (refer to Appendix A).

  • Other patients may be considered on a case by case basis after discussion with pediatric ophthalmology, hematology/oncology, and interventional neuroradiology.

Exclusion Criteria

• Opaque or hazy media which precluded visualization of the fundus.
• New or recurrent retinoblastoma that can be controlled with other conservative measures such as cryotherapy, thermotherapy, or plaque radiotherapy.
• Unilateral COG group E retinoblastoma (refer to Appendix A) with very poor visual prognosis as defined by pediatric ophthalmologist.
• Patients who would benefit from systemic chemotherapy.
• Patients with clinical or radiological evidence suggestive of retinoblastoma invasion of the optic nerve, choroid, sclera, orbit or metastatic sites.
• Currently receiving any other investigational agents.
• A history of allergic reactions attributed to compounds of similar chemical or biologic composition to melphalan or other agents used in the study.
• Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, or cardiac arrhythmia.
• Pregnant and/or breastfeeding. Participants of childbearing potential must have a negative pregnancy test within 14 days of study entry.
• Patients with HIV are eligible unless their CD4+ T-cell counts are < 350 cells/mcL or they have a history of AIDS-defining opportunistic infection within the 12 months prior to registration. Concurrent treatment with effective ART according to DHHS treatment guidelines is recommended.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Intra-arterial injections of melphalan	Melphalan	-Participants will receive intra-arterial injections of melphalan Q4W for 3 cycles.

Primary Outcome Measures

Name	Time	Method
Feasibility of intra-arterial chemotherapy as measured by the number of participants who receive 3 intra-arterial injections of melphalan	Completion of enrollment and treatment of all patients (estimated to be 32 months)	-Feasibility is defined as the ability to receive 3 intra-arterial injections of melphalan per patient.

Secondary Outcome Measures

Name	Time	Method
Visual acuity as measured by Cardiff testing	6 months post-treatment
Rate of ocular salvage	2 years post-treatment	A patient will be considered a successful ocular salvage if the child does not experience enucleation because of disease progression or toxicity during 2 years of follow-up.
Rate of procedure-related complications	Through 30 days following completion of treatment (estimated to be 4 months)
Rate of metastatic disease	Completion of treatment (estimated to be 3 months)	-Patients will be determined to have metastatic disease if they have received 1 or more administrations of IAC and they are found to have progression of disease or appearance of tumor outside of the eye after protocol therapy. Pineal tumors will not be considered metastatic disease.

Trial Locations

Locations (1)

Washington University School of Medicine (Saint Louis Children's Hospital); 🇺🇸 Saint Louis, Missouri, United States