Intra-arterial Chemotherapy for Retinoblastoma

NCT04342572 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: 202006160
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

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.

Conditions

Retinoblastoma

Outcome Measures

Primary Outcomes (1)

• Feasibility of intra-arterial chemotherapy as measured by the number of participants who receive 3 intra-arterial injections of melphalan

  -Feasibility is defined as the ability to receive 3 intra-arterial injections of melphalan per patient.

  Completion of enrollment and treatment of all patients (estimated to be 32 months)

Secondary Outcomes (4)

• Rate of procedure-related complications

  Through 30 days following completion of treatment (estimated to be 4 months)

• Rate of ocular salvage

  2 years post-treatment

• Visual acuity as measured by Cardiff testing

  6 months post-treatment

• Rate of metastatic disease

  Completion of treatment (estimated to be 3 months)

Study Arms (1)

Intra-arterial injections of melphalan

EXPERIMENTAL

-Participants will receive intra-arterial injections of melphalan Q4W for 3 cycles.

Drug: Melphalan

Interventions

Melphalan DRUG

-The drug is commerically available

Also known as: Evomela

Intra-arterial injections of melphalan

Eligibility Criteria

• Age: 4 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Washington University School of Medicine: lead

Study Sites (1)

Washington University School of Medicine (Saint Louis Children's Hospital)

St Louis, Missouri, 63110, United States

Location

Related Links

• Alvin J. Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of Medicine

MeSH Terms

Conditions

Retinoblastoma

Interventions

Melphalan

Condition Hierarchy (Ancestors)

Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue; Retinal Neoplasms; Eye Neoplasms; Neoplasms by Site; Eye Diseases, Hereditary; Eye Diseases; Retinal Diseases

Intervention Hierarchy (Ancestors)

Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phenylalanine; Amino Acids, Aromatic; Amino Acids, Cyclic; Amino Acids; Amino Acids, Peptides, and Proteins

Study Officials

• Margaret Reynolds, M.D.

  Washington University School of Medicine

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 8, 2020
• First Posted: April 13, 2020
• Study Start: August 11, 2020
• Primary Completion: January 4, 2023
• Study Completion: March 21, 2025
• Last Updated: May 11, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)