Intra-arterial Melphalan in Treating Younger Patients With Unilateral Retinoblastoma

NCT ID: NCT02097134

Last Updated: 2023-10-13

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 14 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2014-10-31
• Study Completion Date: 2023-09-30

Brief Summary

This pilot clinical trial studies whether unilateral group D retinoblastoma, or retinoblastoma affecting one eye that has spread to the inner jelly like part of the eye, can be treated with a new technique for delivering chemotherapy directly into the blood vessel that supplies the affected eye. This new technique is called intra-arterial injection. Giving melphalan via intra-arterial injection may make it less likely that children will need surgery to remove the eye and may reduce the amount of treatment side effects.

Detailed Description

PRIMARY OBJECTIVES:

I. To study the feasibility of delivering melphalan directly into the ophthalmic artery in children with newly diagnosed unilateral group D retinoblastoma, who would otherwise be considered for enucleation.

SECONDARY OBJECTIVES:

I. To estimate the ocular salvage rate after treatment with intra-arterial melphalan in children with newly diagnosed unilateral retinoblastoma with group D disease.

II. To evaluate the toxicities and adverse events associated with delivering multiple doses of intra-arterial chemotherapy.

III. To evaluate vision outcomes in children treated with intra-arterial chemotherapy.

IV. To monitor the rate of the development of metastatic disease while on protocol therapy.

TERTIARY OBJECTIVES:

I. To evaluate the effects of intra-arterial therapy on the histopathology of eyes enucleated for progression.

OUTLINE:

Patients receive melphalan intra-arterially (IA) on day 1. Treatment repeats every 28 days for up to 3 courses in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up every 3 months for 1 year, every 4 months for 1 year, every 6 months for 1 year, and then periodically for 2 years.

Conditions

Unilateral Retinoblastoma

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Treatment (melphalan)

Patients receive melphalan IA on day 1. Treatment repeats every 28 days for up to 3 courses in the absence of disease progression or unacceptable toxicity.

Group Type: EXPERIMENTAL

Melphalan

Intervention Type: DRUG

Given IA

Interventions

Melphalan

Given IA

Intervention Type: DRUG

Other Intervention Names

Alanine Nitrogen Mustard; CB-3025; L-PAM; L-Phenylalanine Mustard; L-Sarcolysin; L-Sarcolysin Phenylalanine mustard; L-Sarcolysine; Melphalanum; Phenylalanine Mustard; Phenylalanine Nitrogen Mustard; Sarcoclorin; Sarkolysin; WR-19813

Eligibility Criteria

Inclusion Criteria

• Newly diagnosed patients with unilateral group D retinoblastoma
• Magnetic resonance imaging (MRI) (or computed tomography [CT] if MRI is not available) of the brain must be performed within 14 days prior to study entry
• Diagnostic examination under anesthesia (EUA) must be performed within 14 days prior to study entry
• Rapid central review confirmation of group D disease based on RetCam images from diagnostic EUA must be obtained before starting treatment
• Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1 or 2
• Patients must have a life expectancy of >= 8 weeks
• Patients must have adequate renal function, defined as:

• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2 or
• A serum creatinine based on age/gender as follows:

• 1 month to < 6 months: 0.4 mg/dL
• 6 months to < 1 year: 0.5 mg/dL
• 1 to < 2 years: 0.6 mg/dL
• 2 to < 6 years: 0.8 mg/dL
• 6 to < 10 years: 1 mg/dL
• 10 to < 13 years: 1.2 mg/dL
• 13 to < 16 years: 1.5 mg/dL (male); 1.4 mg/dL (female)
• >= 16 years: 1.7 mg/dL (male); 1.4 mg/dL (female)
• Total bilirubin =< 1.5 x upper limit of normal (ULN) for age
• Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) < 2.5 x upper limit of normal (ULN) for age

Exclusion Criteria

• Patients with bilateral disease
• Unilateral retinoblastoma with group A, B, C, or E eyes
• Clinical or neuroimaging evidence of extraocular disease or orbital optic nerve involvement

• Minimum Eligible Age: 6 Months
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Cancer Institute (NCI)

NIH

Sponsor Role: collaborator

Children's Oncology Group

NETWORK

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Murali M Chintagumpala

Role: PRINCIPAL_INVESTIGATOR

Children's Oncology Group

Locations

Children's Hospital Los Angeles

Los Angeles, California, United States

UCSF Medical Center-Parnassus

San Francisco, California, United States

UCSF Medical Center-Mission Bay

San Francisco, California, United States

Yale University

New Haven, Connecticut, United States

University of Miami Miller School of Medicine-Sylvester Cancer Center

Miami, Florida, United States

Children's Healthcare of Atlanta - Egleston

Atlanta, Georgia, United States

Dana-Farber Cancer Institute

Boston, Massachusetts, United States

Duke University Medical Center

Durham, North Carolina, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center

Houston, Texas, United States

M D Anderson Cancer Center

Houston, Texas, United States

Countries

United States

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

NCI-2014-00618

Identifier Type: REGISTRY

Identifier Source: secondary_id

ARET12P1

Identifier Type: -

Identifier Source: secondary_id

ARET12P1

Identifier Type: OTHER

Identifier Source: secondary_id

ARET12P1

Identifier Type: OTHER

Identifier Source: secondary_id

U10CA180886

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U10CA098543

Identifier Type: NIH

Identifier Source: secondary_id

View Link

ARET12P1

Identifier Type: -

Identifier Source: org_study_id