Intra-arterial Melphalan in Treating Younger Patients With Unilateral Retinoblastoma

NCT02097134 | Completed Phase 1 Results DMC

• 4 other identifiers: ARET12P1NCI-2014-00618U10CA180886U10CA098543
• Study Type: interventional
• Target: 14
• Locations: 1 country
• Sites: 12

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.

Conditions

Unilateral Retinoblastoma

Outcome Measures

Primary Outcomes (1)

• Number of Patients Experiencing Feasibility Failure

  Feasibility failure is defined as a) interventional radiologist is unable to access the ophthalmic artery for the 1st chemotherapy administration for any reason; b) patient develops central retinal artery occlusion after the 1st or 2nd course that does not reopen by the time the next injection is due; or c) the patient cannot receive all three treatments because of Common Terminology Criteria for Adverse Events (CTCAE) complications grade III or IV that are considered possibly, probably or likely related treatment.

  Up to 4 months

Secondary Outcomes (4)

• Incidence of Grade 3 or Higher CTCAE Adverse Events Associated With Multiple Doses of IA Chemotherapy

  Up to 30 days after completion of study treatment

• Probability of Ocular Salvage

  2 years

• Rate of Metastases of Retinoblastoma

  Up to 2 years

• Vision Acuity, Assessed According to the Amblyopia Treatment Study Visual Acuity Testing Protocol

  1 year after therapy

Study Arms (1)

Treatment (melphalan)

EXPERIMENTAL

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.

Drug: Melphalan

Interventions

Melphalan DRUG

Given IA

Also known as: 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

Treatment (melphalan)

Eligibility Criteria

• Age: 6 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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:
• month to \< 6 months: 0.4 mg/dL
• months to \< 1 year: 0.5 mg/dL
• to \< 2 years: 0.6 mg/dL
• to \< 6 years: 0.8 mg/dL
• to \< 10 years: 1 mg/dL
• to \< 13 years: 1.2 mg/dL

You may not qualify if:

• 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

Sponsors & Collaborators

• Children's Oncology Group: lead
• National Cancer Institute (NCI): collaborator

Study Sites (12)

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

UCSF Medical Center-Parnassus

San Francisco, California, 94143, United States

Location

UCSF Medical Center-Mission Bay

San Francisco, California, 94158, United States

Location

Yale University

New Haven, Connecticut, 06520, United States

Location

University of Miami Miller School of Medicine-Sylvester Cancer Center

Miami, Florida, 33136, United States

Location

Children's Healthcare of Atlanta - Egleston

Atlanta, Georgia, 30322, United States

Location

Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center

Houston, Texas, 77030, United States

Location

M D Anderson Cancer Center

Houston, Texas, 77030, United States

Location

MeSH Terms

Interventions

Melphalan

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

Results Point of Contact

• Title: Results Reporting Coordinator
• Organization: Children's Oncology Group

resultsreportingcoordinator@childrensoncologygroup.org

626-447-0064

Study Officials

• Murali M Chintagumpala

  Children's Oncology Group

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

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

Study Record Dates

• First Submitted: March 25, 2014
• First Posted: March 26, 2014
• Study Start: October 31, 2014
• Primary Completion: June 30, 2017
• Study Completion: September 30, 2023
• Last Updated: October 13, 2023
• Results First Posted: June 26, 2018

Record last verified: 2023-10

Locations

US (12)