A Prospective Natural History Study in Uveal Melanoma

The overall objective of this proposal is to develop and utilize a multicenter UM registry that will, in a longitudinal fashion, capture prospective data in order to characterize the natural history of UM and provide data that will be used to support the development of novel therapies for this disease. The care of patients with UM requires a multi-disciplinary team of physicians that commonly requires the involvement of both radiation oncology and interventional radiology, and is typically directed by an ophthalmologic oncologist at time of initial diagnosis of primary disease. Overall management is transitioned to a medical oncologist when distant recurrence is identified. In the case that a patient presents with metastasis at the time of diagnosis, a medical oncologist typically directs overall management. The management of surveillance for the development of metastasis following the treatment of primary disease is variable and, if performed at all, is managed by either an ophthalmologic oncologist or medical oncologist. Thus, the successful development of a registry that aims to capture the data regarding the full natural history of UM requires a collaborative effort including leaders from both the UM ophthalmologic oncology and medical oncology fields. To this end, the investigators have built an initial consortium of key ophthalmologic oncology and medical oncology leaders from multiple major UM centers in the United States.

Study Overview

• Status: Active, not recruiting
• Conditions: Uveal Melanoma

Detailed Description

Uveal melanoma (UM) is the most common primary intraocular malignancy in adults, accounting for 85% to 95% of ocular melanoma cases. However, UM represents only about 3% to 5% of all melanomas in the United States (US). UM most commonly arises from choroidal melanocytes (85-90%), but can also arise from the iris (3-5%) and ciliary body (5-8%). The median age of diagnosis is approximately 62; however, the peak range for diagnosis is between 70 and 79. Males have a 30% greater incidence than females. A variety of putative risk factors have been identified, including the presence of light eyes, fair skin, an inability to tan, ocular melanocytosis, dysplastic nevus syndrome, and germline BRCA1-associated protein 1 (BAP1) mutations.

Importantly, there are no recent or on-going multi-center natural history studies being conducted in this disease, and this effort is the only one to be launched with the goal of capturing the complete course of this disease, from diagnosis, initial management, surveillance, and treatment of recurrent disease in a national and international setting. This registry is especially important in providing such needed data.

• Study Type: Observational
• Enrollment (Actual): 122

Contacts and Locations

Study Locations

• Australia
  • Adelaide, Australia
    • Royal Adelaide Hospital
  • Chatswood, Australia
    • Chatswood Eye Specialists
  • East Melbourne, Australia
    • Royal Victorian Eve and Ear Hospital
  • Maribyrnong, Australia
    • Western Eye Specialists
  • North Adelaide, Australia
    • Pennington Eye Clinic
  • Subiaco, Australia
    • Perth Retina
  • Sydney, Australia
    • St. Vincent's Hospital
  • Sydney, Australia
    • Dr. Conway Private Rooms
• Canada
  • Toronto, Canada
    • Princess Margaret Cancer Center
• Germany
  • Erlangen, Germany
    • Erlangen
• United Kingdom
  • Birkenhead, United Kingdom
    • Clatterbridge Cancer Centre
  • Northwood, United Kingdom
    • Mount Vernon Cancer Centre
  • Sheffield, United Kingdom
    • Sheffield
• United States
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Massachusetts General Hospital
  • New York
    • New York, New York, United States, 10065
      • Memorial Sloan Kettering Cancer Center
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19107
      • Thomas Jefferson University Hospital
  • Texas
    • Houston, Texas, United States, 77030
      • MD Anderson Cancer Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

• Diagnosis of uveal melanoma
• Male/Female over the age of 18 years of age

Description

Inclusion Criteria

• Diagnosis of uveal melanoma
• Ability to provide written informed consent for participation in the prospective registry OR an institutional waiver by the IRB/ethics committee for retrospective data collection without written informed consent

Exclusion Criteria

*None

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
Uveal Melanoma; Diagnosis of uveal melanoma Ability to provide written informed consent for participation in the prospective registry OR an institutional waiver by the IRB/ethics committee for retrospective data collection without written informed consent

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Relapse-free Survival Rate	Document the relapse-free survival of patients with uveal melanoma from the time of diagnosis of primary disease	Up to Five years
Overall Survival Rate	document the overall survival of patients with uveal melanoma from the time of diagnosis of primary disease	Up to Five years
Overall Survival Rate of Patients with Uveal Melanoma	document the overall survival of patients with uveal melanoma from the time of development of metastatic disease	Up to Five years

Collaborators and Investigators

• Sponsor: Columbia University
• Investigators: Principal Investigator: Mariam El-Ashmawy, MD, PhD, Columbia University

Study record dates

Study Major Dates

• Study Start (Actual): May 6, 2021
• Primary Completion (Estimated): July 1, 2026
• Study Completion (Estimated): July 1, 2026

Study Registration Dates

• First Submitted: July 16, 2020
• First Submitted That Met QC Criteria: October 13, 2020
• First Posted (Actual): October 19, 2020

Study Record Updates

• Last Update Posted (Actual): August 22, 2025
• Last Update Submitted That Met QC Criteria: August 18, 2025
• Last Verified: August 1, 2025

More Information

Terms related to this study

• Keywords: Uveal Melanoma
• Additional Relevant MeSH Terms: Neoplasms by Site; Neoplasms; Neoplasms by Histologic Type; Eye Diseases; Skin Diseases; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms, Nerve Tissue; Neuroendocrine Tumors; Nevi and Melanomas; Skin Neoplasms; Eye Neoplasms; Uveal Diseases; Melanoma; Uveal Neoplasms
• Other Study ID Numbers: AAAR5976

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No